From Intellia

Intellia Therapeutics to Present at Upcoming September Investor Healthcare Conferences

CAMBRIDGE, Mass., September 7, 2017 — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, will present at key September investor healthcare conferences. The Company also has appointed Lindsey Trickett as Vice President of Investor Relations.

Upcoming Conferences:

Tuesday, September 12, 2017
Rodman & Renshaw 19th Annual Global Investment Conference
Who: Nessan Bermingham, Ph.D., President, Chief Executive Officer and Founder
Location: New York, New York
Presentation Time: 2:35 pm EST

Tuesday, September 26, 2017
Cantor Fitzgerald 3rd Annual Healthcare Conference
Who: Tom Barnes, Ph.D., Senior Vice President, Innovative Sciences & eXtellia
Location: New York, New York
Presentation Time: 9:10 am EST

Wednesday, September 27, 2017
Leerink Partners Roundtable Series: Rare Disease & Immuno-Oncology
Who: Nessan Bermingham, Ph.D., President, Chief Executive Officer and Founder
Location: New York, New York
Presentation Time: 11:00 am EST

A live webcast of Intellia’s presentations will be accessible through the Events and Presentations page of the Investor Relations section of the company’s website at www.intelliatx.com. To access the webcasts, please log on to the Intellia website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Intellia’s website for 14 days following each conference.

Lindsey Trickett joins Intellia as Vice President, Investor Relations, and will be responsible for leading and overseeing all aspects of Investor Relations activities. Lindsey brings to Intellia more than 12 years of biopharmaceutical experience in varying roles of increasing responsibility at AstraZeneca and Medimmune, including Treasury, Capital Management and Operations Strategy. Most recently Lindsey was a Director of Investor Relations at AstraZeneca for the North American investor base focusing on immuno-oncology and cardiovascular and metabolic diseases. Lindsey earned a degree in International Economics at the University of Kentucky in Lexington and her Masters of Business Administration with a focus in Finance from Georgetown University in Washington, D.C.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

Intellia Contacts:

Media Contact:
Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com

Investor Contact:
Lindsey Trickett
Vice President, Investor Relations
+1 857-285-6211
lindsey.trickett@intelliatx.com

Intellia Therapeutics Announces Second Quarter 2017 Financial Results

  • In non-human primates, demonstrated robust green fluorescent protein expression throughout the liver 24 hours following a single, systemically delivered, intravenous dose, using our proprietary lipid nanoparticle delivery system
  • Using our proprietary lipid nanoparticle delivery system, demonstrated in vivo durable liver genome editing in mice through nine months, post-single-dose intravenous administration
  • $241 million cash and cash equivalents as of June 30 2017

CAMBRIDGE, Mass., August 1, 2017 (GLOBE NEWSWIRE) – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, announced financial and operational results for the second quarter of 2017.

The company has made significant progress using our proprietary lipid nanoparticle (LNP) delivery system throughout the quarter. We advanced into non-human primate studies, while first time data from rat studies, the second species we tested in vivo, confirmed the durable mouse data demonstrating high levels of sustained genome editing and serum transthyretin (TTR) reduction post-single-dose intravenous administration.

“We are very excited that our initial non-human primate data in vivo continue to validate our mRNA delivery technology. These preclinical data accelerate our momentum as we advance the development of potential therapies to treat patients with high unmet medical needs,” said Chief Executive Officer and founder Nessan Bermingham, Ph.D., Intellia Therapeutics. “During the quarter, we have further strengthened our company by advancing partnerships, including a research agreement with Ospedale San Raffaele in Milan for our ex vivo technology focused on novel engineered cell therapies using CRISPR, expanded our global intellectual property portfolio, and appointed two new members to our Board of Directors.”

Second Quarter 2017 Operational Highlights

The company achieved several key operational milestones during the second quarter of 2017, including:

  • Commenced non-human primate studies in support of our preclinical work relating to our lead  development program in TTR. We demonstrated robust green fluorescent protein expression throughout non-human primate liver 24 hours after a single systemically delivered administration dose in vivo, using our proprietary lipid nanoparticle delivery system.
  • Extended our data set beyond the mouse and delivered CRISPR/Cas9 in the rat. Specifically, initial data from rat models post-single-dose intravenous administration in vivo using our proprietary lipid nanoparticle delivery system showed up to 91 percent reduction in serum TTR protein levels and up to 66 percent editing at the target DNA site, demonstrating robust and dose-responsive gene editing of TTR. We presented these results at the American Society of Gene & Cell Therapy Annual Meeting in Washington D.C. in May 2017.
  • Provided an update on our ongoing durability study, demonstrating durable liver editing through nine-months post a single intravenous dose administration in mice using our proprietary lipid nanoparticle delivery system. Throughout the liver, the data continues to demonstrate durability and high editing efficiency of LNP-mediated editing of the TTR gene, showing 97 percent reduction in serum TTR protein levels and approximately 70 percent editing at the target DNA site. The intended transient nature of LNP delivery has been confirmed in rodents with undetectable Cas9 mRNA and gRNA levels in the liver by 72 hours post administration.
  • Entered into a three-year research collaboration, option, and license agreement to engineer optimized T cell cancer therapies with a leading scientific research institution, Ospedale San Raffaele SRL of Milan, Italy.
    • The collaboration aims to discover innovative targets against tough-to-treat cancers, leveraging Intellia’s proprietary CRISPR/Cas9 platform to generate next-generation      T cell therapies to address unmet needs in hematological and solid tumors.
    • The collaboration is the first partnership of Intellia’s eXtellia division, focused on advancing next generations of engineered cell therapies through unique and proprietary applications of CRISPR genome editing in areas including immuno-oncology and auto-immunity.
  • Continued to defend and enhance our CRISPR/Cas9 foundational and therapeutic intellectual property position through filing and prosecution of patent applications covering our internal, collaboration and in-licensed inventions. In relation to the foundational CRISPR/Cas9 genome editing intellectual property portfolio to which we have rights for human therapeutics and companion diagnostics, and which is co-owned by the Regents of the University of California, the University of Vienna, and Dr. Emmanuelle Charpentier (collectively “UC”), the following was achieved:
    • China’s State Intellectual Property Office granted to UC a patent broadly covering CRISPR/Cas9 single-guide genome editing methods and compositions. The patent includes claims that cover methods for editing DNA in non-cellular and cellular settings, including in eukaryotic cells such as human and mammalian cells. It also includes CRISPR/Cas9 composition of matter and system claims for use in any setting, including claims covering the use of CRISPR/Cas9 in producing medicines for treating disease.
    • UC submitted its opening brief July 25, 2017 to the U.S. Court of Appeals for the Federal Circuit in their appeal from a February 15, 2017 decision by the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (“PTAB”) in an interference proceeding relating to the CRISPR/Cas9 genome editing technology. In the appeal, UC requests the reversal of the PTAB’s decision to terminate the interference between certain CRISPR/Cas9 patent claims owned by UC and patents and patent applications owned by the Broad Institute, Massachusetts Institute of Technology, the President and Fellows of Harvard College and the Rockefeller University.
  • Named Moncef Slaoui, Ph.D. and Frank Verwiel, M.D. to its Board of Directors. In addition the Board established a Science & Technology Committee:
    • Moncef Slaoui, Ph.D. and Frank Verwiel, M.D. joined the Board of Directors. Drs. Slaoui and Verwiel began Board responsibilities on July 25, 2017. Intellia’s Board of Directors also established a Science and Technology Committee, which will be chaired by Dr. Slaoui. Additionally, Carl L. Gordon, Ph.D., CFA, General Partner, OrbiMed Advisors, LLC, informed the company of his resignation from the Intellia Board of Directors effective at the close of business on July 25, 2017.

Second Quarter 2017 Financial Results

Collaboration Revenue

Collaboration revenue was $5.9 million for the second quarter of 2017, compared to $4.2 million for the second quarter of the prior year. The increase in collaboration revenue in 2017 was primarily driven by amounts recognized under our collaboration agreement with Regeneron Pharmaceuticals, Inc. (Regeneron), which was entered into in April 2016.

Through June 30, 2017, the company received $104.1 million in funding under its collaborations with Novartis Institutes for BioMedical Research, Inc. (Novartis) and Regeneron, excluding amounts received for equity investments, and recorded accounts receivable of $2.8 million. Excluding the $2.6 million of the upfront payment received from Novartis, which was allocated to the purchase of equity securities, we recognized $34.7 million in collaboration revenue under these agreements through June 30, 2017, and had remaining deferred revenue of $69.6 million as of June 30, 2017.

Operating Expenses

Research and development expenses increased $8.2 million to $15.6 million during the second quarter 2017, compared to $7.4 million during the same period of 2016. This increase was driven primarily by greater support for the advancement of our early-stage research programs and includes laboratory supplies and research materials. Additionally, salary and related headcount-based expenses increased as the company grew to 110 research and development employees as of June 30, 2017, from 56 research and development employees as of June 30, 2016.

General and administrative expenses increased $2.7 million to $6.4 million during the second quarter of this year, compared to $3.7 million in the second quarter of 2016. This increase was driven primarily by increased salary and related headcount-based expenses as the company grew to 33 general and administrative employees as of June 30, 2017, from 19 general and administrative employees as of June 30, 2016, to support our public company compliance and administration obligations. The company also incurred increased corporate insurance, legal, and other professional expenses related to its expanding operations since becoming a public company in May 2016.

Our net loss was $15.6 million for the second quarter 2017, compared to $6.9 million for the second quarter of 2016.

Balance Sheet

Cash and cash equivalents at June 30, 2017, were $241 million, compared to $301 million for the same quarter in 2016. The base period cash and cash equivalents were primarily attributable to $115.5 million in proceeds from our initial public offering, $55 million in concurrent private placements, and a $75 million upfront payment from Regeneron in April 2016. The year-over-year change is attributed to cash used in ongoing operations.

Financial Guidance

Our primary uses of capital will continue to be research and development programs, laboratory and related supplies, compensation and related expenses, legal and other regulatory expenses, patent prosecution, filing and maintenance costs for our licensed intellectual property, and general overhead costs.

During 2017, the company expects expenses to continue to increase compared to prior periods relating to our ongoing activities, particularly as research and development and preclinical activities gather further momentum toward human clinical trials, and we spend a full year occupying our new office and laboratory facility, which we began to occupy in the fourth quarter of 2016.

As of June 30, 2017, the company had an accumulated deficit of $81.8 million. We expect our losses to increase as we continue to incur significant research and development and other expenses related to the advancement of our therapeutic programs and our ongoing operations. Based on our research and development plans and expectations related to the progress of the company’s programs, we expect that the cash and cash equivalents as of June 30, 2017, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2019, excluding any potential milestone payments or extension fees received under our collaboration agreements with Novartis and Regeneron.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products, as well as our CRISPR/Cas9 intellectual property portfolio; our ability to achieve stable liver editing; effective genome editing with a single treatment dose; the potential timing and advancement of our preclinical studies, including continuing non-human primate studies, and clinical trials; the potential development of the ex vivo cell therapeutics through our eXtellia division, including the development of next-generation T cell therapies that address unmet needs in hematological and solid tumors, immuno-oncology and auto-immunity; the intellectual property position and strategy of Intellia’s licensors; actions by government agencies; the impact of our collaborations with Ospedale San Raffaele, Novartis and Regeneron on our development programs; the potential timing of regulatory filings regarding our development programs; the potential commercialization opportunities, including value and market, for product candidates; our expectations regarding our uses of capital, expenses, future accumulated deficit and other 2017 financial results; and our ability to fund operations through mid-2019. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain our intellectual property position; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

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Intellia Contacts:

Media Contact:

Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com

Investor Contact:
Graeme Bell
Executive Vice President, Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com

Intellia’s Statement on Genome Editing of Human Embryos by U.S. Researchers

Intellia is aware of the recent reports of U.S. researchers editing the DNA of human embryonic cells. We cannot speculate on the specifics of the study.

The focus of Intellia’s work is, and will continue to be, on finding treatments that help patients who suffer today. To that end, we are committed to: using genome editing technologies, including CRISPR/Cas9, only for patient benefit; focusing on treating serious diseases of high unmet need; refraining from directly modifying germline cells, including sperm, egg or embryonic tissue, or developing any clinical applications of germline genome editing.

We acknowledge that some applications of genome editing raise important ethical issues, specifically when used for non-therapeutic purposes or for modifying the genomes of germline cells with the intention of affecting future generations.

We take our ethical and social responsibility to not only patients, but also humanity as a whole very seriously, and will continue to develop the technology in an appropriate and ethical way. We are aligned with the U.S. National Academies of Sciences, Engineering and Medicine, that cautioned in its February 2017 Report on Human Genome Editing,“heritable germline editing is not ready to be tried in humans,” and “should only be permitted in clinical trials to prevent serious diseases or disabilities if there is stringent oversight in the healthcare system.”

Intellia’s joint position on germline editing with CRISPR Therapeutics can be found here: http://www.intelliatx.com/germline-gene-editing-joint-statement/.

 

 

CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomics Announce Appellate Brief Seeking Reversal of U.S. Patent Board Decision on CRISPR/Cas9 Gene Editing

• Appeal seeks reversal of Patent Trial and Appeal Board decision terminating interference without determining priority of inventorship of CRISPR/Cas9 gene editing
• Brief asserts that the Board failed to properly apply controlling U.S. Supreme Court and Federal Circuit precedents, and ignored evidence of multiple groups readily applying CRISPR/Cas9 gene editing to eukaryotic cells following teachings of Charpentier-Doudna team

ZUG, Switzerland; CAMBRIDGE, Massachusetts; BERKELEY, California; DUBLIN, Ireland; July 25, 2017 (GLOBE NEWSWIRE) – CRISPR Therapeutics (NASDAQ:CRSP), Intellia Therapeutics, Inc. (NASDAQ:NTLA), Caribou Biosciences, Inc. and ERS Genomics, Ltd. announced that The Regents of the University of California, the University of Vienna, and Dr. Emmanuelle Charpentier (collectively “UC”), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome engineering, today submitted an appellate brief to the U.S. Court of Appeals for the Federal Circuit (the “Federal Circuit”) seeking reversal of a decision by the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (“PTAB”) in an interference proceeding relating to CRISPR/Cas9 gene editing technology. In the appeal, UC requests reversal of the PTAB’s decision terminating the interference between certain CRISPR/Cas9 patent claims owned by UC and claims of the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively, “Broad”).

In its brief to the Federal Circuit (Case No. 17-1907), UC asserts that the PTAB’s February 15, 2017 determination that the UC patent claims did not make the Broad’s patent claims obvious is based on a misapplication of controlling legal standards established by U.S. Supreme Court and Federal Circuit precedent. In its decision, the PTAB had concluded that UC’s claims covering CRISPR/Cas9 single guide gene editing technology and its application in any cellular or non-cellular setting did not make obvious Broad’s claims covering application of the same technology limited to use in eukaryotic cellular settings.

In its brief, UC sets forth multiple errors in the PTAB’s holding that the use of CRISPR/Cas9 in eukaryotes is separately patentable as alleged by Broad, including the following:

• With respect to the obviousness of applying the technology to eukaryotic cells, U.S. Supreme Court and Federal Circuit precedents clearly mandate that obviousness determinations be based on an “expansive and flexible approach” including consideration of “the inferences and creative steps that a person of ordinary skill in the art would employ.” In contrast, the PTAB applied a narrow and restrictive approach that ignored certain key evidence, including the steps actually employed by those of skill in the art at the time, and also effectively required a “guarantee” that UC’s CRISPR/Cas9 invention would work in eukaryotic cells, when well-established case law requires only a “reasonable expectation of success.”
• With respect to the reasonable expectation of success standard that establishes obviousness, the PTAB effectively ignored U.S. Supreme Court and Federal Circuit case law, which emphasize that an invention can be obvious if it is “obvious to try,” even if success is not guaranteed, “[w]hen there is a design need or market pressure to solve a problem,” “there are a finite number of identified, predictable solutions,” and experimentation leads to “the anticipated success.”
• The PTAB ignored U.S. Supreme Court and Federal Circuit precedent highlighting that the occurrence of what appear to be simultaneous “inventions” arising close together in time is itself strong evidence of their obviousness, and accordingly requiring that such evidence be considered in any obviousness analysis. In contrast, in terminating the interference, the PTAB essentially dismissed as “irrelevant” the evidence that six different laboratories successfully applied UC’s claimed CRISPR/Cas9 invention in eukaryotic cells using conventional techniques within months after UC publicly disclosed the invention – some of them prior to Broad’s first filing.
• The PTAB effectively ignored that Broad’s own eukaryotic application of UC’s CRISPR/Cas9 invention had simply utilized conventional prior art techniques. As a consequence, it failed to consider that Broad’s alleged invention did not reflect any significant innovation on Broad’s part, an important issue under applicable precedent.
• The PTAB failed to hold Broad to its burden of proving, among other things, the effective priority date for its patent claims, as required by the PTAB’s own rules and Federal Circuit precedent. By not requiring the Broad to meet its burden of proof, and by merely assuming that all of the Broad’s claims were entitled to their earliest possible filing date despite Broad’s failure of proof, the PTAB improperly ignored relevant intervening art that made the Broad’s claims obvious.

As explained in UC’s brief, application of the correct legal standards to the case is believed to require reversal of the PTAB’s decision. For these reasons, UC requests that the Federal Circuit instruct the PTAB to reinstate the interference proceeding so that it can properly determine priority of inventorship, as previously requested by UC. The PTAB’s failures to consider pertinent evidence and apply appropriate legal standards should at the very least require the matter to be remanded so that the PTAB can properly consider the evidence related to obviousness and Broad’s no-interference-in-fact motion using appropriate legal standards.

In the PTAB’s February decision terminating the interference proceeding prematurely, it had not yet considered the teachings of UC’s own prior-filed patent application with respect to using CRISPR/Cas9 in eukaryotic cells. Instead, the PTAB only addressed the threshold question of whether use in eukaryotic cells can be separately patentable from use in all settings as covered by UC’s claims. However, determinations on the underlying substantive matters have recently been made in parallel prosecution before the U.S. Patent & Trademark Office (“USPTO”). The USPTO has rejected a series of patent applications filed by Broad that are directed to uses of CRISPR/Cas9 technology in eukaryotic cells as being non-novel in view of UC’s prior-filed patent application, which the USPTO examiners considered to have effectively taught use of the CRISPR/Cas9 technology in eukaryotic cells. In addition, patent applications filed by Sigma-Aldrich and Toolgen that similarly claim use of CRISPR/Cas9 in eukaryotic cells (both of which filed applications before Broad’s application) have likewise recently been rejected as being either non-novel or obvious in view of the prior-filed UC patent application with specific respect to its teachings regarding application of the invention to use in eukaryotic cells.

Consistent with the substantive determinations reached by the USPTO regarding the broad teachings of the UC patent application, UC’s corresponding cases covering use of CRISPR/Cas9 in all settings, including in eukaryotic cells, have been advanced to grant or decisions to grant in numerous jurisdictions worldwide, including in the United Kingdom, the nearly 40 other countries that are members of the European Patent Convention, and more recently in other jurisdictions such as Australia, New Zealand, Singapore and China.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The Company’s multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Additionally, CRISPR Therapeutics has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in diseases with high unmet need. The foundational CRISPR/Cas9 patent estate for human therapeutic use was licensed from the Company’s scientific founder Dr. Emmanuelle Charpentier. CRISPR Therapeutics is headquartered in Zug, Switzerland, with business offices in London, United Kingdom, and R&D operations based in Cambridge, Massachusetts. For more information, please visit www.crisprtx.com.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

About Caribou Biosciences, Inc.

Caribou is a leading company in CRISPR genome engineering founded by pioneers of CRISPR/Cas9 biology based on research carried out in the Doudna Laboratory at the University of California, Berkeley. Caribou’s tools and technologies provide transformative capabilities to therapeutic development, agricultural biotechnology, industrial biotechnology, and basic and applied biological research. For more information, including information about obtaining research and commercial licenses as well as collaborations, visit www.cariboubio.com and follow the Company @CaribouBio. “Caribou Biosciences” and the Caribou logo are trademarks of Caribou Biosciences, Inc.

About ERS Genomics

ERS Genomics was formed to provide broad access to the foundational CRISPR/Cas9 intellectual property held by Dr. Emmanuelle Charpentier. Non-exclusive licenses are available for research and sale of products and services across multiple fields including: research tools, kits, reagents; discovery of novel targets for therapeutic intervention; cell lines for discovery and screening of novel drug candidates; GMP production of healthcare products; production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology. For additional information please visit www.ersgenomics.com.

CRISPR’s Forward-Looking Statement

Certain statements set forth in this press release constitute “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, but not limited to, statements concerning: the intellectual property protection of our technology and therapies, the intellectual property positions of third parties, and the therapeutic value, development, and commercial potential of CRISPR/Cas-9 gene editing technologies and therapies. You are cautioned that forward-looking statements are inherently uncertain. Although the company believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, the forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: uncertainties regarding the intellectual property protection for our technology and intellectual property belonging to third parties; uncertainties inherent in the initiation and completion of preclinical studies for the Company’s product candidates; availability and timing of results from preclinical studies; whether results from a preclinical trial will be predictive of future results of the future trials; expectations for regulatory approvals to conduct trials or to market products; and those risks and uncertainties described under the heading “Risk Factors” in the company’s most recent annual report on Form 10-K, and in any other subsequent filings made by the company with the U.S. Securities and Exchange Commission (SEC), which are available on the SEC’s website at www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. The information contained in this press release is provided by the company as of the date hereof, and, except as required by law, the company disclaims any intention or responsibility for updating or revising any forward-looking information contained in this press release.

Intellia’s Forward-Looking Statement

This press release contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward looking statements include, but are not limited to, express or implied statements regarding the intellectual property position and strategy of Intellia’s licensors; and Intellia’s ability to advance CRISPR/Cas9 into therapeutic products for severe and life-threatening diseases and its CRISPR/Cas9 intellectual property portfolio. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to Intellia’s ability to protect and maintain its intellectual property position, risks related to the ability of Intellia’s licensors to protect and maintain their intellectual property position, the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized, the risk of cessation or delay of any of the ongoing or planned clinical trials and/or development of Intellia’s product candidates, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, and the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

CRISPR CONTACTS
Media:
Jennifer Paganelli
WCG for CRISPR
+1 347-658-8290
jpaganelli@wcgworld.com
Investors:
Chris Brinzey
Westwicke Partners for CRISPR
+ 1 339-970-2843
chris.brinzey@westwicke.com
INTELLIA CONTACTS
Media:
Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+ 1 857-706-1071
jenn.smoter@intelliatx.com
Investors:
Graeme Bell
Executive Vice President, Chief Financial Officer
+ 1 857-706-1081
graeme.bell@intelliatx.com
CARIBOU CONTACT
Greg Kelley
Feinstein Kean Healthcare
+ 1 404-836-2302
gregory.kelley@fkhealth.com
ERS GENOMICS CONTACTS
MacDougall Biomedical Communications
Mario Brkulj or Dr. Stephanie May
+49 89 2420 9345 or +48 89 2420 9344
mbrkulj@macbiocom.com or smay@macbiocom.com

Intellia Therapeutics Names Moncef Slaoui, Ph.D. and Frank Verwiel, M.D. to Its Board of Directors and Establishes a Science and Technology Committee

CAMBRIDGE, MASS. – July 24, 2017 (GLOBE NEWSWIRE) – Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, announced today the appointment of Moncef Slaoui, Ph.D. and Frank Verwiel, M.D. to its Board of Directors. Drs. Slaoui and Verwiel will begin Board responsibilities effective immediately. Intellia’s Board of Directors also is establishing a Science and Technology Committee, which will be chaired by Dr. Slaoui. Additionally, Carl L. Gordon, Ph.D., CFA, General Partner, OrbiMed Advisors, LLC, has informed the Company of his resignation from the Intellia Board of Directors effective at that close of business on July 25, 2017.

“We are very pleased to welcome Drs. Slaoui and Verwiel to our Board of Directors. They bring decades of scientific, operational, business and strategic experience in the pharmaceutical and biotech industries to Intellia. We look forward to their guidance as we continue to advance our genome editing technology to patients,” said Perry Karsen, Chair of the Board of Directors, Intellia Therapeutics. “On behalf of the Board of Directors, I want to thank Carl Gordon for his leadership in establishing Intellia and for all of his contributions to the Company.”

Dr. Moncef Slaoui brings more than 30 years of experience in pharmaceutical drug research and development, new product launches and business development to Intellia’s Board of Directors

Dr. Moncef Slaoui recently retired as Chairman of Vaccines at GlaxoSmithKline (GSK), where he also served on the company’s executive team and board of directors for eleven years. During his nearly 30-year career at GSK, he amassed significant expertise in drug discovery and development, business development, new product development and commercial organizations. Dr. Slaoui led the global integration of Novartis’ vaccine unit into GSK. Dr. Slaoui was chairman of GSK’s global R&D organization for eight years, bringing improved productivity with more than 30 phase 3 programs, and re-organizing drug discovery to accommodate 38 focused discovery performance units in the company. Additionally, Dr. Slaoui was responsible for GSK’s venture capital arm, SR One, and other venture capital partnerships.

Dr. Slaoui led the company’s bioelectronics R&D strategy, including the launch of Galvani Bioelectronics, a company where he remains Chairman of the board of directors.

Dr. Slaoui has authored more than 100 scientific papers and presentations and served on the board of directors for the Pharmaceutical Research and Manufacturers of America’s Foundation, the Biotechnology Innovation Organization’s board of directors, and was a member of the National Institutes of Health’s Advisory Committee until 2016. Currently, he is a member of the Advisory Board for the Qatar Foundation.

Dr. Slaoui received his Ph.D. in Molecular Biology and Immunology from the Université libre de Bruxelles Belgium and completed post-doctoral studies at Harvard Medical School and Tufts University of Medicine in Boston.

Dr. Frank Verwiel brings more than 25 years of strategic, operational and international expertise in the biotechnology and pharmaceutical industries and over a decade of industry board experience

Dr. Verwiel possesses significant biotech and pharmaceutical experience, having been the president and CEO of Aptalis Pharma Inc., prior to its acquisition in 2014 by Forest Laboratories. Dr. Verwiel also was a vice president at Merck & Co., where he led the global hypertension franchise. He also served as the general manager of Merck Sharpe Dohme’s operations in the Netherlands. He held numerous leadership positions in the commercial organization at Servier Laboratories.

Dr. Verwiel currently serves as chairman of the board of directors of ObsEva SA, a biopharmaceutical company focused on women’s reproductive health. Dr. Verwiel holds membership and committee chair positions on the boards of AveXis, Inc., Achillion Pharmaceuticals, Inc. and Bavarian Nordic A/S. Previously, he served on the board of directors of Intermune, until its acquisition by Roche in 2014, and the Biotechnology Innovation Organization.

Dr. Verwiel received a doctorate in medicine from Erasmus University in Rotterdam, Netherlands. He received a Masters of Business Administration from the Institut Européen d’Administration des Affaires (INSEAD) in Fontainebleau, France.

New Science and Technology Committee Supports Intellia’s R&D Strategy

In recognition of the company’s advances and opportunities in applying the CRISPR/Cas technology platform, Intellia’s Board of Directors is creating a new committee to the Board, the Science and Technology Committee. This committee will support the Board of Directors in its oversight and advisement of the Company’s research and development activities and scientific strategy. The Science and Technology Committee will be chaired by Dr. Slaoui, given his extensive operational, scientific and industry experience. 

“Both Drs. Slaoui and Verwiel have served as board directors for key organizations and were top executives at companies that delivered innovative medicines to patients,” said Nessan Bermingham, Ph.D., Founder and Chief Executive Officer, Intellia Therapeutics. “We value their comprehensive industry knowledge and previous board experiences, which along with the formation of the new Science & Technology Committee, will help shape Intellia as we move forward on our path to the clinic. We thank Carl Gordon for his partnership and OrbiMed’s support of Intellia that enabled our achievements and successes thus far.”

Intellia’s Board of Directors, committees, chairpersons and members include:

• Chair of the Board: Perry Karsen
• Board Members: Perry Karsen, Nessan Bermingham, Caroline Dorsa, Jean-François Formela, John Leonard, Moncef Slaoui and Frank Verwiel
• Audit Committee: Caroline Dorsa (Chair), Jean-François Formela, and Frank Verwiel
• Compensation Committee: Jean-François Formela (Chair), Caroline Dorsa, Perry Karsen, and Moncef Slaoui
• Nominating & Corporate Governance Committee: Perry Karsen (Chair), Caroline Dorsa and Frank Verwiel
• Science & Technology Committee: Moncef Slaoui (Chair), Jean-François Formela and John Leonard

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. For a discussion of risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent quarterly report on Form 10-Q filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in our other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

Intellia Contacts:
Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
857-701-1071
jenn.smoter@intelliatx.com

Graeme Bell
Executive Vice President, Chief Financial Officer
857-701-1081
graeme.bell@intelliatx.com

Intellia Therapeutics Announces Patent for CRISPR/Cas Genome Editing in China

– Patent covers CRISPR/Cas9 gene editing methods and compositions for use in any setting, including human and other eukaryotic cells 

CAMBRIDGE, Mass., June 19, 2017 (GLOBE NEWSWIRE) — Intellia Therapeutics (NASDAQ: NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, today announced that China’s State Intellectual Property Office (“SIPO”) has indicated that it will grant a patent broadly covering CRISPR/Cas9 single-guide gene editing methods and compositions.  The patent includes claims covering methods for editing DNA in non-cellular and cellular settings, including in eukaryotic cells such as human and mammalian cells.  It also includes CRISPR/Cas9 composition of matter and system claims for use in any setting, including claims covering the use of CRISPR/Cas9 in producing medicines for treating disease.

“SIPO’s decision further expands our IP portfolio, and is further global recognition that Jennifer Doudna, Emmanuelle Charpentier and their team are the pioneers in the application of CRISPR/Cas9 in all cell types,” said Intellia Therapeutics Chief Executive Officer and President, Nessan Bermingham, Ph.D. “Intellia continues to build on preclinical work and to focus on the development of our pipeline of novel human therapeutics that will potentially transform the lives of patients with genetic diseases.”

The European Patent Office and the United Kingdom’s Intellectual Property Office have previously issued patents from the same underlying international patent application.  This international patent application is based on the same U.S. priority applications that were filed starting in May 25, 2012 by the Regents of the University of California, the University of Vienna and Dr. Emmanuelle Charpentier (collectively, “the UC Intellectual Property”).  Intellia has rights to the UC Intellectual Property, including the European and UK patents, for human therapeutic, prophylactic, and palliative uses (including companion diagnostics), excluding anti-fungal and anti-microbial applications. Intellia obtained these rights through a 2014 license agreement with Caribou Biosciences, Inc., which is the exclusive licensee of the University of California and University of Vienna, two of the co-owners of the intellectual property.  In the United States, certain patent claims from the UC Intellectual Property were involved in an interference proceeding with patents and patent applications owned by the Broad Institute et al. before the U.S. Patent Trial and Appeal Board (“the PTAB”), and the PTAB decision to terminate the interference is currently on appeal at the U.S. Court of Appeals for the Federal Circuit.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Intellia’s combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; follow us on Twitter @intelliatweets.

Intellia’s Forward-Looking Statement

This press release contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward looking statements include, but are not limited to, express or implied statements regarding the intellectual property position and strategy of Intellia’s licensors; and Intellia’s ability to advance CRISPR/Cas9 into therapeutic products for severe and life-threatening diseases and its CRISPR/Cas9 intellectual property portfolio. Any forward-looking statements in this press release are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, risks related to Intellia’s ability to protect and maintain its intellectual property position, risks related to the ability of Intellia’s licensors to protect and maintain their intellectual property position, the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized, the risk of cessation or delay of any of the ongoing or planned clinical trials and/or development of Intellia’s product candidates, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, and the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful. For a discussion of other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

Intellia Therapeutics
Media Contact:
Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com

Investor Contact:
Graeme Bell
Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com

Intellia’s Response to Nature Methods Article on CRISPR/Cas9

Recently Schaefer et al. (1) reported the presence of more than a thousand genome differences between mice that had been edited with S. pyogenes Cas9 at the zygote stage, and a control mouse of the same strain. Given the overlap of genetic differences between the two edited mice compared to the control mouse, the authors concluded that these differences arose from a Cas9-dependent activity. Flaws in the study design and data analysis, however, make this conclusion inappropriate. Based on the information available on the mouse study, the more plausible conclusion is that the genetic differences reflect a normal level of variation between individuals in a colony.

These alternative interpretations can be resolved by establishing when the reported differences arose in the lineage of these mice. The authors assume, given the pattern of homo- and heterozygosity, that they occurred in a burst in either the 1- or 2-cell stage even though the intended Cas9 repair occurred at a later stage given the mosaicism of these edited animals (2). Unfortunately, the authors did not sequence the parents, nor do we know if the edited animals (F03 and F05) are true sibs or simply closely related. There is also no information given on how closely related the colony control animal was to the parents used in this study. This missing evidence is critical to support their conclusion. Notably, in an unrelated study, WGS of unedited inbred mouse littermates showed 985 sites of variation between individuals (3), a number similar to that found in the authors’ dataset (696 SNVs and indels). Furthermore, a second unrelated study directly examined the effects of Cas9 editing (with intact Cas9 cleavase or Cas9D10A nickase) using WGS (4). These authors saw significant variation between individuals, but after comparison with relevant control parental and sib genomes, demonstrated the opposite result, namely that Cas9 induced no unexpected mutations. Absent the parental DNA sequence, the Schaefer authors could also have either looked at more than 2 F0 animals, or examined the degree of overlap between the control genome and either edited mouse, which they did not do (they only reported on variant sites in the edited mice absent from the control genome). This complete pairwise analysis could directly support or refute their claim.

Beyond this missing evidence, the authors’ explanation requires activities that Cas9 is not known to possess. First, the authors show that none of the edited sites has sufficient target homology to the guide (5) to support Watson-Crick gRNA-DNA base pairing. There was no genome-wide unbiased off-target analysis done with the chosen guide RNA (2), so we have no baseline for understanding its specificity. Second, 13/30 sites presented (43%) lack a -GG- PAM within 4 bases of the variant, the only known pre-requisite for Cas9 to engage with DNA (6). Third, there is no known mechanistic basis for Cas9 to induce SNVs, nor did the authors propose one. Indeed the preponderance of SNVs reported is consistent with their being caused by spontaneous deamination, a major driver of sequence drift in genomes (7).

In summary, we feel that the principal conclusions of the paper (that the variation seen was unexpected and that it was Cas9-mediated) have not been demonstrated, and a simpler alternative explanation was not explored.

References

  1. Schaefer, K. et al. Unexpected mutations after CRISPR-Cas9 editing in vivo. Nat. Methods 14, 548-549 (2017).
  2. Wu, W.-H. et al. CRISPR Repair Reveals Causative Mutation in a Preclinical Model of Retinitis Pigmentosa. Mol. Ther. 24, 1388-1394.
  3. Oey, H. et al. Genetic and epigenetic variation among inbred mouse littermates: identification of inter-individual differentially methylated regions. Epigenetics & Chromatin 8, 54-65 (2015)
  4. Iyer, V. et al. Off-target mutations are rare in Cas9-modified mice. Nat. Methods 12, 479 (2015).
  5. Doensch, J.G. et al. Optimized sgRNA design to maximize activity and minimize off-target effects of CRISPR-Cas9. Nat. Biotechnol. (2016) 34, 184-191.
  6. Anders, C. et al. Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease. Nature (2014) 513, 569-573.
  7. Tubbs, A and Nussenzweig, A. Endogenous DNA damage as a source of genomic instability in cancer. Cell 168, 644-656.

Intellia Therapeutics and San Raffaele University and Research Hospital to Combine CRISPR/Cas9 Genome Editing with Enhanced Cell Therapies to Fight Cancer

  • Research collaboration harnesses powerful CRISPR/Cas9 genome editing to engineer improved T-cell therapies targeting unmet needs in cancer
  • Agreement builds on Intellia’s ex vivo approach through its division, eXtellia

CAMBRIDGE, Mass., and MILAN, Italy, June 6, 2017 (GLOBEWIRE) – Intellia Therapeutics (NASDAQ:NTLA), a leading genome editing company, and San Raffaele University and Research Hospital, a leading scientific institution, have entered into a three-year research collaboration, option and license agreement to engineer optimized T cell cancer therapies. The goal of the collaboration is to discover innovative tools to target tough-to-treat cancers, while leveraging Intellia’s propietary CRISPR/Cas9 platform to generate next-generation T cell therapies that will address unmet needs in both hematological and solid tumors. Professor Chiara Bonini, Head of San Raffaele’s Experimental Hematology Unit and Deputy Director of the Division of Immunology, Transplantation and Infectious Diseases, will lead the scientific work at San Raffaele.

The collaboration marks the first external partnership of Intellia’s eXtellia division. eXtellia’s long-term strategy is focused on advancing new generations of engineered cell therapies through the unique and proprietary applications of CRISPR genome editing. eXtellia was established in 2016, and has identified its initial areas of focus as immuno-oncology and auto-immunity, where genome-edited cell therapy offers a potentially powerful and differentiated therapeutic modality. The agreement also includes options and licenses to key technologies for production of engineered cell therapies developed at San Raffaele.

“Through this collaboration, eXtellia aims to apply CRISPR/Cas9 genome editing in a multi-faceted way to modulate the fundamental properties of engineered immune cells and amplify their anti-cancer properties far beyond current applications,” said Andrew Schiermeier, Ph.D., Senior Vice President, eXtellia. “San Raffaele and Dr. Bonini are recognized globally as leaders in cell therapy and immuno-oncology, with excellent track records in translating innovative research into approved therapies. We aspire to one day cure cancer in patients who are fighting everyday with few or no treatment options.”

“T cell therapy has recently produced impressive results in clinical trials in specific cancer types. However, to realize the full potential of T cell therapy, including in a broader set of cancer types, next generation cellular products are needed,” said Professor Chiara Bonini. “Intellia’s leadership in genome editing will be critical to achieve this goal and shape this new class of ‘living drugs’.”

About San Raffaele University and Research Hospital

Ospedale San Raffaele is a research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. Since 2012, it is part of San Donato Hospital Group, the leading hospital group in Italy. San Raffaele is a multi-speciality center with over 50 clinical specialties and over 1,300 beds. Research focuses on integrating basic, translational and clinical activities to provide the most advanced care to its patients. For further information, visit: hsr.it.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products; our ability to achieve stable liver editing; effective genome editing with a single treatment dose; and the potential timing and advancement of our preclinical studies and clinical trials. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain our intellectual property position; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

Contacts:

Intellia Therapeutics

Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com

Graeme Bell
Executive Vice President, Chief Financial Officer +1 857-706-1081
graeme.bell@intelliatx.com

Ospedale San Raffaele – University and Research Hospital

Press office:
Gea Gardini + 39 02.2643.3004 ufficio.stampa@hsr.it www.hsr.it

Intellia Therapeutics Announces Progress with CRISPR/Cas9 at the American Society of Gene & Cell Therapy Annual Meeting

  • First-time data validates high levels of gene editing and reduction in serum transthyretin protein levels in rat models
  • First to demonstrate single-dose, in vivo results using proprietary lipid nanoparticle delivery system in mice showing: approximately 97 percent reduction in serum transthyretin protein levels; 70 percent liver editing efficiency following a single dose; six months post-single-dose durability of stable liver editing

WASHINGTON, May 13, 2017 (GLOBE NEWSWIRE) – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, presented an update on its long-term mouse genome editing and delivery studies and shared new, first-time data in rat models demonstrating consistent dose-dependent editing, at the American Society of Gene & Cell Therapy’s Annual Meeting (ASGCT).

These data, featured in a platform presentation on Saturday, May 13 at ASGCT showed:

  • Six-month mouse study data demonstrating both durability and high editing efficiency in vivo, with approximately 70 percent editing at the target DNA site with a single intravenous dose. A 97 percent reduction of serum transthyretin (TTR) protein levels also was sustained.
  • In addition, robust, dose-responsive lipid nanoparticle (LNP)-mediated editing of the TTR gene in rat livers following single intravenous administration; up to 66 percent editing at the target DNA site and up to 91 percent reduction in serum TTR protein levels.
  • Both studies were conducted using Intellia’s proprietary LNP delivery system, providing high levels of liver delivery and rapid clearance.

“Data from the additional rat study further validates the in vivo CRISPR/Cas9 platform using Intellia’s proprietary LNP delivery system,” said David Morrissey, Ph.D., senior vice president, Platform and Delivery Technology. “In both species, we saw unprecedented in vivo liver editing results and consistent delivery of CRISPR/Cas9 with systemic administration using LNPs, while also showing the ability to expand our studies in larger species.”

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products; our ability to achieve stable liver editing; effective genome editing with a single treatment dose; and the potential timing and advancement of our preclinical studies and clinical trials. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain our intellectual property position; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

 

Intellia Contacts

Media Contact:

Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com

Investor Contact:
Graeme Bell
Executive Vice President, Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com

Intellia Therapeutics Announces First Quarter 2017 Financial Results

  • First to demonstrate single dose, in vivo results using a proprietary lipid nanoparticle delivery system showing: approximately 97 percent reduction in serum transthyretin protein levels; 70 percent liver editing efficiency following a single dose; six months post-single-dose durability of stable liver editing
  • Appeal of U.S. Patent Trial and Appeal Board decision on CRISPR/Cas9 interference involving Intellia-licensed patent family filed by the University of California and other co-owners
  • European Patent Office announced decision to grant CRISPR/Cas9 genome editing technology patent and the United Kingdom Intellectual Property Office granted national chimeric CRISPR/Cas9 patent 
  • First quarter 2017 cash position of $258 million

CAMBRIDGE, Mass., May 2, 2017 (GLOBE NEWSWIRE) – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, announced financial results for the first quarter 2017.

“The first quarter of 2017 has been highly productive for Intellia as we continue to advance our preclinical programs toward developing life-transforming therapies. Building on our earlier results showing 97 percent in vivo reduction of serum transthyretin protein levels, our durability data also continue to demonstrate stable liver editing and support our vision that genome editing could be effective with just a single dose,” said Nessan Bermingham, Ph.D., chief executive officer, and founder, Intellia Therapeutics. “We expect to advance  preclinical studies significantly this year by progressing our technology in non-human primate studies and look toward its potential broad clinical applications to help treat patients with unmet medical needs.”

Business Highlights

During the first quarter of 2017, the company achieved numerous important milestones:

Significantly advanced the genome editing field with unprecedented liver editing and lipid nanoparticle delivery data using CRISPR/Cas9 as a potential one-time treatment.  

  • In March, the company reported editing efficiency in vivo with a significant reduction of serum transthyretin (TTR) protein levels. More recent durability study data show stable liver editing in mice for at least six months following a single administration.
    • Enhanced in vivo gene editing in the TTR locus, with an efficiency of approximately 70 percent in the total mouse liver at the target DNA site, after a single intravenous administration;
    • Decreased serum TTR protein levels of up to approximately 97 percent; and
    • Achieved undetectable Cas9 messenger RNA (mRNA) and guide RNA (gRNA) in the liver by 72 hours, post administration.

Defended and enhanced our CRISPR/Cas9 foundational and therapeutic intellectual property position.

  • The European Patent Office (EPO) decided to grant to co-owners the Regents of the University of California, University of Vienna, and Dr. Emmanuelle Charpentier (collectively, UC) a patent broadly covering the CRISPR/Cas9 genome editing technology. The patent includes claims covering compositions of the widely adopted CRISPR single guide RNA technology for use in any non-cellular or cellular setting, including eukaryotic cells such as human or mammalian cells, as well as for use in human therapeutics. This European patent, which is expected to grant on May 10, 2017, will be nationalized in, and cover, approximately forty European countries that are member states of the European Patent Organisation.
  • In addition to the EPO decision, earlier this year, the United Kingdom’s (U.K.) Intellectual Property Office granted to UC a second national patent on the CRISPR/Cas9 genome editing system. This February 7, 2017 U.K. patent covers chimeric CRISPR/Cas9 systems in which the Cas9 protein is modified to provide alternative DNA-modulating activities while the first U.K. patent obtained by UC covers the single guide RNA for uses in both non-cellular and cellular settings.
  • In April 2017, UC appealed to the U.S. Court of Appeals for the Federal Circuit the decision by the Patent Trial and Appeal Board (PTAB) to terminate the interference between certain CRISPR/Cas9 patent claims owned by UC and patents and patent applications owned by the Broad Institute, Harvard University and the Massachusetts Institute of Technology. UC is seeking review and reversal of the PTAB’s February 15, 2017 decision, which concluded that, although the claims overlap, the respective scope of UC and Broad’s claim sets do not define the same patentable invention and, accordingly, terminated the interference without deciding which party first invented the use of the CRISPR/Cas9 technology in eukaryotic cells.

First Quarter 2017 Financial Results

Collaboration Revenue

Collaboration revenue was $6.2 million for the first quarter of 2017, compared to $1.8 million for the first quarter of the prior year. The increase in collaboration revenue in 2017 was primarily driven by amounts recognized under our collaboration agreement with Regeneron Pharmaceuticals, Inc (Regeneron), which was entered into in April 2016.

Through March 31, 2017, the company received $103.1 million in funding under its collaborations with Novartis Institutes for BioMedical Research, Inc. (Novartis) and Regeneron, excluding amounts received for equity investments, and recorded accounts receivable of $2.4 million. Excluding the $2.6 million of the upfront payment received from Novartis which was allocated to the purchase of equity securities, we recognized $28.8 million in collaboration revenue under these agreements through March 31, 2017 and had remaining deferred revenue of $74 million as of March 31, 2017.

Operating Expenses

Research and development expenses increased $8.2 million to $13.4 million during the first quarter 2017, compared to $5.2 million during the same period of 2016. This increase was driven primarily by increased salary and related headcount-based expenses, including equity-based compensation expenses, as the company grew to 88 research and development employees as of March 31, 2017, from 44 research and development employees as of March 31, 2016. This increase supported the advancement of our early-stage research programs including, consumption of laboratory supplies and research materials.

General and administrative expenses increased $2.5 million to $5.7 million during the first quarter of this year, compared to $3.2 million in the first quarter of 2016. This increase was driven primarily by increased salary and related headcount-based expenses, including equity-based compensation expenses, as the company grew to 31 general and administrative employees as of March 31, 2017, from 17 general and administrative employees as of March 31, 2016. The company also incurred increased corporate insurance, legal, and other professional expenses related to its expanding operations since becoming a public company in May 2016.

Our net loss was $12.6 million for the first quarter 2017, compared to $6.7 million for the first quarter of 2016.

Balance Sheet

Cash and cash equivalents at March 31, 2017, were $258 million, compared to $64 million for the same quarter in 2016. The increase in cash and cash equivalents was primarily attributable to $115.5 million in proceeds from our initial public offering, $55 million in concurrent private placements and a $75 million upfront payment from Regeneron in April 2016, partially offset by cash used in operations.

Financial Guidance

Our primary uses of capital will continue to be research and development services, compensation and related expenses, laboratory and related supplies, legal and other regulatory expenses, patent prosecution, filing and maintenance costs for our licensed intellectual property, and general overhead costs.

During 2017, the company expects expenses to continue to increase compared to prior periods in connection with our ongoing activities, particularly as research and development and preclinical activities gather momentum, and we spend a full year occupying our new office and laboratory facility, which we began to occupy in the fourth quarter of 2016.

As of March 31, 2017, the company had an accumulated deficit of $66.2 million. We expect our losses to increase as we continue to incur significant research and development and other expenses related to our ongoing operations. Based on our research and development plans and expectations related to the progress of the company’s programs, we expect that cash and cash equivalents as of March 31, 2017, as well as technology access and research funding from Novartis and Regeneron, will enable Intellia to fund operating expenses and capital expenditures through mid-2019, without giving effect to any potential milestone payments or extension fees received under our collaboration agreements with Novartis and Regeneron.

Upcoming Events During the Second Quarter 2017

The company expects to make presentations at the following upcoming scientific and investor conferences:

  • The American Society of Gene & Cell Therapy, May 13, Washington
  • The TechConnect World Innovation Conference, May 16, Washington
  • Jefferies Healthcare Conference, June 6, New York City
  • JMP Securities Life Sciences Conference, June 20, New York City
  • Goldman Sachs Third Annual Innovation Symposium, June 27, New York City

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains “forward-looking statements” of Intellia within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellia’s ability to advance and expand the CRISPR/Cas9 technology to develop into human therapeutic products, as well as our CRISPR/Cas9 intellectual property portfolio; our ability to achieve stable liver editing; effective genome editing with a single treatment dose; the potential timing and advancement of our preclinical studies, including non-human primate studies, and clinical trials; the intellectual property position and strategy of Intellia’s licensors; actions by government agencies; the impact of our collaborations with Novartis and Regeneron on our development programs; the potential timing of regulatory filings regarding our development programs; the potential commercialization opportunities, including value and market, for product candidates; our expectations regarding our uses of capital, expenses, future accumulated deficit and other 2017 financial results; and our ability to fund operations through mid-2019. Any forward-looking statements in this press release are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain our intellectual property position; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.

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Intellia Contacts

Media Contact:

Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com

Investor Contact:
Graeme Bell
Executive Vice President, Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com