From Intellia

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2016 Financial Results

— First to demonstrate single dose, in vivo results, showing approximately 97 percent reduction in serum transthyretin protein levels
— Further established a comprehensive platform, including a proprietary lipid nanoparticle delivery system, to accelerate therapeutic development
— Executed partnering deal with Regeneron Pharmaceuticals, and continued to advance Intellia’s partnership with Novartis, to support Intellia’s emerging pipeline with six distinct programs in active research and development, spanning multiple types of genome editing
— Continued to deploy a diversified approach to maximize CRISPR potential, including pursuing CRISPR applications in Hepatitis B Virus and hematopoietic stem cells
— 2016 year-end cash position of $273 million

CAMBRIDGE, Mass., March 14, 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 fourth quarter and full year 2016.

“2016 was a phenomenal year for Intellia. We signed a multi-year research and development collaboration with Regeneron Pharmaceuticals, our second partner, and became a publicly traded company with an upsized offering. We also presented the first in vivo CRISPR liver editing data, and significantly grew our capabilities, laboratories, and team,” said Nessan Bermingham, Ph.D., chief executive officer and founder, Intellia Therapeutics. “I am extremely pleased with the progress since forming the company in May 2014. In 2017, we expect to make meaningful steps toward our goal of bringing revolutionary therapies to patients.”

Business Highlights

The company achieved numerous important milestones in 2016, including:

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 August 2016, Intellia became the first company to present preclinical in vivo liver editing data using its novel system of delivering CRISPR/Cas9 components by lipid nanoparticle (LNP) technology. Last week, the company reported an update on these results, demonstrating increased editing efficiency in vivo with a significant reduction of serum transthyretin (TTR) protein levels. This effect was durable for at least four months following a single administration in mice. In summary Intellia presented:
    • 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 (versus previously reported 60 percent);
    • Decreased serum TTR protein levels of up to approximately 97 percent (versus previously reported 80 percent); and
    • Undetectable Cas9 messenger RNA (mRNA) and guide RNA (gRNA) in the liver by 72 hours, post administration.

On March 22, 2017, the company will present additional data regarding its latest in vivo liver editing data at Le Studium Conference on Messenger RNA Therapeutics in Orleans, France.

Completed a successful initial public offering, securing a strong cash position, fueling the company’s pipeline and platform studies.

  • In May 2016, the company completed its initial public offering, upsizing the offer to $115.5 million and executing a concurrent private placement of $55 million. Through December 31, 2016, Intellia raised an aggregate of approximately $352.5 million to fund its operations, of which $97.0 million was through collaboration agreements, $170.5 million was from its initial public offering and concurrent private placements, and $85.0 million was from the sale of convertible preferred stock.

Announced a multi-year, multi-target research and development collaboration with Regeneron Pharmaceuticals, representing one of the largest deals in the CRISPR/Cas9 field.

  • Under the collaboration, Regeneron has the exclusive right to discover and develop CRISPR-based products against up to 10 targets, focused primarily on therapies for a broad range of diseases that may be treated by editing genes in the liver. Transthyretin amyloidosis (ATTR) is the first target to be jointly developed and potentially commercialized by the companies.
  • Intellia received access to the Regeneron Genetics Center, one of the world’s most comprehensive genetics databases, and proprietary mouse models for a limited number of its liver programs.
  • Intellia received a $75 million upfront payment and a $50 million equity investment and is eligible to receive significant milestone payments of up to $320 million per target, as well as high single-digit to low-teen royalties.

To advance the pipeline and explore the scope of genome edits with the CRISPR/Cas9 system, the company is working on four sentinel in vivo liver indications and two sentinel ex vivo programs employing different editing strategies.

In vivo

  • Transthyretin Amyloidosis (ATTR) program, being developed with Regeneron utilizing a gene knockout strategy. Candidate guides are being progressed to non-human primate studies in 2017.
  • Hepatitis B Virus (HBV) program, utilizing a knockout strategy to target covalently closed circular DNA (cccDNA). Over 400 candidate guides were characterized and evaluated, and advanced in vitro studies are underway using the most optimal guides identified.
  • Alpha-1 Antitrypsin Deficiency (AATD) program, utilizing either a gene knockout or a gene repair strategy, therapeutic outcome dependent. The company is performing activities for guide selection activities.
  • Primary Hyperoxaluria Type 1 (PH-1), our initial Inborn Errors of Metabolism (IEM) program, is in advanced guide selection, using a gene knockout strategy while we also characterize guides and edit types for several other IEMs of interest.

Ex vivo

  • Chimeric antigen receptor T Cell (CAR-T) and hematopoietic stem cell (HSC) applications in partnership with Novartis, where Intellia retains the right to develop and commercialize rights to certain HSC programs, advanced to late preclinical development stage.

Beyond providing the company multiple potential product opportunities, each of these programs potentially enable CRISPR/Cas9 translation into a broader set of disease indications requiring the same types of DNA edits.

Gained exclusive access to two leading genetic database centers that will bring critical disease insights to Intellia’s preclinical and clinical process.

  • Through its agreement with Regeneron, the company has access to the Regeneron Genetics Center, one of the world’s most comprehensive genetic databases, which pairs sequenced exomes with anonymized electronic health records.
  • Intellia joined Genomics England’s GENE Consortium as the first dedicated genome editing company to participate in the 100,000 Genome Project. Participation in the GENE Consortium, alongside leading global biotech and pharmaceutical companies, gives Intellia access to important data to better understand the basis of rare genetic diseases, which could lead to identifying novel targets and possible treatment approaches.

Enhanced our CRISPR/Cas9 foundational and delivery intellectual property position.

  • Executed an invention management agreement (IMA) with the owners (University of California, University of Vienna, and Emmanuelle Charpentier) and other key licensees of the CRISPR/Cas9 foundational intellectual property, thus ensuring alignment in the prosecution, defense and enforcement of, as well as global freedom to operate regarding this intellectual property.
  • Secured issuance in the United Kingdom of two critical CRISPR/Cas9 patents, which are the first two issued patents from the UCal/Vienna/Charpentier CRISPR/Cas9 foundational patent application family.
  • Filed and prosecuted patent applications covering the company’s intellectual property, including the company’s LNP delivery system, treatment and analytical methods, and chemical modifications to guide RNA.
  • Worked with collaborators to protect jointly developed and licensed intellectual property including RNA guide improvements and disease specific applications.

Expanded our highly qualified expert leadership team by augmenting the Board of Directors and company management, while significantly growing R&D operations and laboratories, in preparation for clinical studies.

  • Perry Karsen, formerly CEO of Celgene’s Cellular Therapeutics Division, was appointed Chairman of the Board, bringing decades of biopharmaceutical C-suite management and board experience.
  • Graeme Bell, formerly CFO of Anacor Pharmaceuticals (acquired by Pfizer in 2016), was named Executive Vice President and Chief Financial Officer, bringing extensive financial and operational experience and strong relationships with the investment community.
  • Andrew Schiermeier, formerly Senior Vice President and Global Head of Merck KGaA’s Oncology, was named Senior Vice President of eXtellia, responsible for the strategic direction and oversight of all operations within the division. eXtellia is the company’s division focused on delivering ex vivo therapies in the areas of immuno-oncology and autoimmune/inflammatory diseases.
  • In December 2016, Intellia opened a new state-of-the-art headquarters facility in Cambridge, Massachusetts, increasing the company’s total laboratory and office space to more than 80,000 sq. ft.

Full-Year 2016 Financial Results

Cash and cash equivalents at December 31, 2016, were $273.1 million, compared to $75.8 million at December 31, 2015. The increase in cash and cash equivalents was primarily attributable to proceeds from our initial public offering and concurrent private placements in May 2016, as well as a $75.0 million upfront payment from Regeneron in April 2016, partially offset by cash used in operations.

The company is not profitable and has incurred losses in each period since our inception. Our net loss was $31.6 million for the year ended December 31, 2016, and $10.6 million for the fourth quarter of 2016, compared to $12.4 million for the year ended December 31, 2015, and $5.2 million for the fourth quarter of 2015.

Collaboration Revenue

Collaboration revenue was $16.5 million for the year ended December 31, 2016, and $5.6 million for the fourth quarter of 2016, as compared to $6.0 million for the year ended December 31, 2015, and $1.7 million for the fourth quarter of 2015. The increase in collaboration revenue in 2016 was primarily driven by amounts recognized under our collaboration agreement with Regeneron, which was entered into in April 2016.

Through December 31, 2016, the company received $97.0 million in funding under our collaborations with Novartis and Regeneron, excluding amounts received for equity investments, and recorded accounts receivable of $6.5 million as of December 31, 2016. Excluding the $2.6 million of upfront payment received from Novartis that was allocated to the purchase of equity securities, we recognized $22.5 million in collaboration revenue under these agreements through December 31, 2016, and had remaining deferred revenue of $78.3 million as of December 31, 2016.

Operating Expenses

Research and development expenses increased $20.6 million to $31.8 million during the year ended December 31, 2016, as compared to $11.2 million during the year ended December 31, 2015, and increased by $6.9 million to $11.3 million during the fourth quarter of 2016, as compared to $4.4 million during the fourth quarter of 2015.
This increase was primarily driven by our growth to 77 research and development employees as of December 31, 2016, from 38 research and development employees as of December 31, 2015, and the advancement of our early-stage research programs collectively resulting in increases in salaries and related compensation expenses, as well as laboratory supplies and research materials.

General and administrative expenses increased by $8.5 million to $16.8 million during the year ended December 31, 2016, as compared to $8.3 million during the year ended December 31, 2015, and increased by $2.3 million to $5.1 million during the fourth quarter of 2016, as compared to $2.8 million during the fourth quarter of 2015.

This increase was primarily related to increased salary and related headcount-based expenses, including equity-based compensation expenses, as the company grew to 27 general and administrative employees as of December 31, 2016, from 14 general and administrative employees as of December 31, 2015, as well as increased corporate insurance, legal, and other professional expenses related to our operations as a public company beginning in May 2016.

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 increase compared to prior periods in connection with our ongoing activities, particularly as research and development and preclinical activities continue, 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 December 31, 2016, the company had an accumulated deficit of $53.6 million. We expect our losses to increase as we continue to incur significant research and development and other expenses related to our ongoing operations, seek regulatory approvals for our future product candidates, scale-up manufacturing capabilities, maintain, expand and protect our intellectual property portfolio, and hire additional personnel to support the development of our product candidates and to enhance our operational, financial and information management systems. 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 December 31, 2016, 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

The company will participate in the following upcoming investor conferences:

  • Oppenheimer Healthcare Conference, March 22, 2017, New York City; and
  • Needham Healthcare Conference, April 5, 2017, 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” 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 our ability to advance CRISPR/Cas9 into therapeutic products for severe and life-threatening diseases; the potential timing and advancement of our clinical trials; the impact of our collaborations with Novartis and Regeneron on our development programs; the impact of our research relationships with Regeneron and the GENE Consortium on our ability to potentially identify disease targets and possible treatments; the future effects of our invention management agreement on our intellectual property strategy and position; the potential indications we may pursue, including our sentinel indications; the potential timing of regulatory filings regarding our development programs; potential commercialization opportunities for product candidates; our expectations regarding our uses of capital, expense, future accumulated deficient and other 2017 financial results; and our expectations regarding our ability to fund operations through mid-2019. 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, the risk that any one or more of our product candidates will not be successfully developed and commercialized, the risk that positive results from a preclinical or clinical study may not necessarily be predictive of the results of future preclinical or clinical studies, the risk of cessation or delay of any of the ongoing or planned clinical trials and/or our development of our 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, the risk that our collaboration with Novartis or Regeneron will not continue or will not be successful, and risks related to our ability to protect and maintain our intellectual property position.

For a discussion of other 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 subsequent filings with the Securities and Exchange Commission, including our upcoming Annual Report on Form 10-K for the year ended December 31, 2016. 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 Therapeutics Demonstrates Pioneering CRISPR/Cas9 Genome Editing Efficiency Data Using Lipid Nanoparticle Delivery Technology

– First to demonstrate single dose, in vivo results, showing approximately a 97 percent reduction in serum transthyretin protein levels
– Durability data show stable liver editing for at least four months
– Increased liver editing efficiency reported to date at 70 percent, following a single dose

CAMBRIDGE, Mass., March 8, 2017 (GLOBE NEWSWIRE) – Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapies, today reported updated data showing increased levels of genome editing efficiency in vivo and durability results with its CRISPR/Cas9 technology, following a single administration. Using its lipid nanoparticle (LNP) technology, Intellia achieved approximately a 97 percent reduction in serum transthyretin (TTR) protein driven by 70 percent gene editing efficiency in the mouse liver.

“These results are extremely promising, as they demonstrate compelling activity with lipid nanoparticles observed in the liver following a single dose,” said Executive Vice President, R&D John Leonard, M.D. “We are excited by the extent of the effect, which confirms the power of CRISPR/Cas9 for potential therapeutic uses. These high levels of gene editing are the result of Intellia’s effort to identify highly efficient delivery of CRISPR/Cas9 using lipid nanoparticles. The data advance our efforts as we look to transform the way we treat disease.”

The data are being presented for the first time at the Cowen and Company 37th Annual Health Care Conference in Boston on Wednesday, March 8, 2017 at 8:40 am ET. Complete data are being presented on March 22, 2017 at the Le Stadium Conference on Messenger RNA Therapeutics in Orleans, France. Data showed robust editing and sustained results including:

• Progress in achieving 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 (versus previously reported 60 percent);
• An associated decrease in serum TTR protein levels of up to approximately 97 percent (versus previously reported 80 percent);
• Undetectable Cas9 mRNA and guide RNA (gRNA) in the liver for 72 hours post administration; and
• Durable and stable liver editing for at least four months following a single administration.

Study Background

The ongoing, preclinical editing studies were designed to explore the use of lipid nanoparticles for delivery of CRISPR/Cas9 components to the liver in mice and to mediate editing of target DNA within hepatocytes. For the LNPs in the studies, Cas9 mRNA was co-formulated with chemically synthesized gRNAs targeting the mouse TTR gene, and administered via one intravenous tail vein injection. Additional studies were performed to evaluate the impact of editing of variables in guide format, degree of guide chemical modification, and dose response on editing efficiency. The durability of the liver editing was evaluated through a four-month time period, and pharmacokinetic (PK) parameters for Cas9 mRNA and sgRNA were measured.

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. These forward looking statements include, but are not limited to, express or implied statements regarding our ability to advance CRISPR/Cas9 into therapeutic products for severe and life-threatening diseases; the potential timing and advancement of our clinical trials; the potential targets or indications we may pursue; and potential commercialization opportunities for product candidates. 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, the risk that any one or more of our product candidates will not be successfully developed and commercialized, the risk that positive results from a preclinical or clinical study may not necessarily be predictive of the results of future preclinical or clinical studies, risks related to our ability to protect and maintain our intellectual property position, the risk of cessation or delay of any of the ongoing or planned clinical trials and/or our development of our 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 our collaboration 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 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 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
Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com

Intellia Therapeutics to Present at March Investor Healthcare Conferences

CAMBRIDGE, Mass., March 1, 2017 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, announced that company senior leaders will present at key March investor healthcare conferences, including the Cowen and Company 37th Annual Health Care Conference, the Goldman Sachs Third Annual Innovation Symposium, and the Oppenheimer 27th Annual Healthcare Conference.

Details of the upcoming presentations are as follows:

Wednesday, March 8th
Cowen and Company 37th Annual Health Care Conference
Who: Nessan Bermingham, Ph.D., chief executive officer & founder
Location: Boston, Massachusetts
Presentation Time: 8:40 am ET

Wednesday, March 15th
The Goldman Sachs Third Annual Innovation Symposium
Gene Editing and CRISPR Panel
Who: Nessan Bermingham, Ph.D., chief executive officer & founder
Location: New York, New York
Panel Time: 10:15 am ET

Wednesday, March 22nd
The Oppenheimer 27th Annual Healthcare Conference
Who: John Leonard, M.D., executive vice president, R&D
Location: New York, New York
Presentation Time: 2:45 pm ET

-more-

Individuals may access the presentations for available conferences via live webcast on the Intellia website at www.intelliatx.com under “Events & Presentations” in the “Investor Relations” section. When available, replays of these webcasts will be available on the site for 90 days following the live events.

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 THERAPEUTICS TO HOLD CONFERENCE CALL TO ADDRESS PATENT INTERFERENCE PROCEEDINGS

CAMBRIDGE, Mass., Feb. 15, 2017 (Globewire) – Intellia Therapeutics, Inc. (Nasdaq: NTLA) will hold a conference call with investors on Thursday, February 16, 2017 at 8 a.m., EST. Investors are invited to dial into the conference call hosted by Nessan Bermingham, Ph.D., chief executive officer and founder, Jose E. Rivera, executive vice president and general counsel, and Graeme Bell, chief financial officer.

To participate on the day of the call, dial 844-882-7840 and use Conference ID# 74650186, approximately five minutes before the call. International callers, please dial +1 574-990-9827 and use Conference ID# 74650186.

The call will be available for replay from February 16 to March 18, 2017. The replay number is 855-859-2056, Conference ID# 74650186. International callers, please dial +1 404-537-3406, Conference ID# 74650186.

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.

CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomics Provide Update on CRISPR/Cas9 U.S. Patent Interference Proceedings and Grants of Corresponding Patents in the U.K.

 

  • UC’s patent application covering the use of CRISPR/Cas9 genome editing technology with a single-guide RNA format in any non-cellular or cellular setting, including human and other eukaryotic cells, will be released from the interference absent an appeal, and may then be prosecuted to potential issuance
  • Additional legal channels are available to recognize the priority of the University of California/University of Vienna/Charpentier intellectual property covering eukaryotic cells
  • US. Patent Trial & Appeal Board did not make a determination regarding which party in the interference is the first inventor of the use of the CRISPR/Cas9 genome editing technology in eukaryotes
  • UK. Intellectual Property Office grants patents to CRISPR/Cas9 genome editing technology in any cellular setting – including in eukaryotes – to University of California/University of Vienna/Charpentier

 

BASEL, Switzerland, CAMBRIDGE, Mass., BERKELEY, California, DUBLIN, Ireland, Feb. 15, 2017 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ:CRSP), Intellia Therapeutics, Inc. (NASDAQ:NTLA), Caribou Biosciences, Inc., and ERS Genomics Limited provide an update on the Patent Trial & Appeal Board (“PTAB”) of the U.S. Patent and Trademark Office (“USPTO”) decision on the motions filed by the University of California, the University of Vienna and Dr. Emmanuelle Charpentier (collectively, “UC”), on one hand, and the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively, “Broad”), on the other, in the interference proceeding relating to CRISPR/Cas9 genome editing technology (“CRISPR/Cas9 Technology”).  The PTAB discontinued the current interference finding that the claim sets presented by the two parties were considered “patentably distinct” from each other because UC’s current claims are broader in scope in that they are not restricted to use in eukaryotic cells, whereas Broad’s claims are all limited to use in eukaryotic cells. As a result of the decision, UC’s broader case, which was previously considered allowable but for the interference, is now released from the interference and may be prosecuted to potential issuance by UC, while a new interference can be sought with respect to eukaryote claims, currently pending in a separate UC patent application once they are deemed allowable. Alternatively, UC could appeal the current decision, which is currently under consideration. In parallel cases, the United Kingdom’s Intellectual Property Office (UK IPO) granted patents to foundational CRISPR/Cas9 genome editing technology in any non-cellular or cellular setting (including in human cells) to UC.

 

The prosecution and enforcement of UC’s foundational intellectual property covering CRISPR/Cas9 Technology, such as this patent application, is governed by a global cross-consent and invention management agreement between the co-owners of the intellectual property – the Regents of the University of California, Emmanuelle Charpentier, and the University of Vienna – as well as their key licensees and sublicensees – CRISPR Therapeutics, ERS Genomics, Caribou Biosciences, and Intellia Therapeutics.

 

U.S. Interference Proceeding

The written decisions and associated documents relating to U.S. patent interference 106,048 are publicly available at https://acts.uspto.gov/ifiling/PublicView.jsp.

 

  • UC’s earliest patent application describing the CRISPR/Cas9 genome editing technology was filed on May 25, 2012, and Broad’s earliest patent application was filed on December 12, 2012.

 

  • The interference was based on (i) UC’s claims directed to the use of the CRISPR/Cas9 Technology with the widely used single-guide RNA format (in which the two key RNA molecules, tracrRNA and crRNA, are fused into a single molecule) in any setting – including but not limited to eukaryotic cells, and (ii) Broad’s claims covering the use of the CRISPR/Cas9 Technology in eukaryotic cells only. UC’s claims, which USPTO examiners previously deemed in condition for allowance subject to the interference, are now released from the interference, and UC may pursue them to potential issuance.

 

  • The current proceeding was terminated as a “threshold” matter based on uses in eukaryotic cells being considered separately patentable from the use of the CRISPR/Cas9 Technology in all cellular and non-cellular settings (i.e. prokaryotes, eukaryotes and in vitro). Although UC’s and Broad’s claims both encompass uses in eukaryotic cells, the PTAB decided that the interference should not proceed with the current claim sets because UC’s claims are broader in scope in that they are not limited to use in the eukaryotic setting, whereas Broad’s claims are all limited to uses in eukaryotes. Under patent interference rules, the PTAB applies a “2-way” test requiring that the parties’ claims define the “same patentable invention.”  In its decision, the PTAB concluded that, although they overlap, the respective scope of UC and Broad’s claims did not define the same patentable invention.

 

  • The PTAB’s holding ends the current proceeding based on the patent claims before it, and the PTAB’s decision is not intended to nor does it establish which party actually invented first with respect to use of the CRISPR-Cas9 Technology in eukaryotic cells. UC is free to seek a new interference with Broad’s patents based on existing applications UC already has before the PTO that include claims limited to use of the CRISPR-Cas9 Technology in eukaryotes. If a new interference is sought and declared, it would then begin with a motions phase related to the newly-designated claims.

 

  • The PTAB did not rule on substantive motions, including whether the parties are entitled to rely on their earliest patent applications for priority benefit. UC’s earliest application was filed on May 25, 2012, and Broad’s was filed on December 12, 2012. However, determinations on certain substantive matters have recently been made in parallel prosecution before the 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 in the claims involved in the interference) as being “non-novel” in view of the UC’s prior-filed patent application (which the USPTO examiners considered to have provided an enabling disclosure that effectively taught use of the CRISPR/Cas9 Technology in eukaryotic cells). These Broad cases include USSN 14/105,031, USSN 14/105,035, USSN 14/523,799, and USSN 14/703,511, all of which stand rejected by the USPTO.  In rejecting Broad’s applications, the USPTO concluded that the UC’s priority application “discloses methods of, and compositions and CRISPR-Cas systems for interfering with a target DNA sequence in both prokaryotic and eukaryotic cells using CRISPR RNA (crRNA) and a CRISPR-associated (cas) protein/nucleic acid.” The USPTO also determined that Broad’s attempt to antedate or “swear behind” the earlier-filed UC patent applications using inventor declarations (as was done in obtaining many of the issued Broad patents) is improper because the UC case “clearly discloses each of the claimed limitations in the earliest two priority applications.” (See Final Rejection of Broad application USSN 14/523,799, August 29, 2016, and similar rejections made against all of the other above-referenced Broad cases.)

 

  • Regarding Staphylococcus aureus Cas9, the PTAB did not decide Broad’s substantive motion to “de-designate” (i.e. remove from the interference) claims directed to use of S. aureus Cas9 (essentially based on Broad’s arguments that use of the aureus Cas9 orthologue was non-obvious in view of the S. pyogenes Cas9 and therefore separately patentable). However, in substantive examination of an UC patent application, the USPTO recently determined as follows: “It would have been obvious to one of ordinary skill in the art to have modified the method of Jinek [a June 2012 publication co-authored by UC scientists] by replacing the S. pyogenes Cas9 protein with the Staphylococcus aureus Cas9 protein because it would have merely amounted to a simple substitution of one known Cas9 protein for another to yield predictable results.” (See USPTO Official Action in USSN 14/942,782, January 4, 2017.)

 

 

U.K. Patent Grants

Outside the United States, a UC application directed broadly to the single-guide CRISPR/Cas9 genome editing system (i.e. not limited by cellular or non-cellular setting) was examined by the United Kingdom’s Intellectual Property Office and, despite multiple evidentiary “observations” filed by third parties including the Broad, was granted as UK Patent No. 2518764. A second UK patent application, which is directed to chimeric CRISPR/Cas9 systems, was also the subject of third-party observations, and was granted as a patent on February 7, 2017 (UK Patent No. 2537000). Corresponding applications are being prosecuted in the European Patent Office and in other regional and national offices covering approximately 80 jurisdictions worldwide. Granted patents can be subject to proceedings challenging their grant, validity or scope.

 

Almost all jurisdictions worldwide are “first-to-file” systems, which recognize the first patent applicant(s) as the legal inventor(s) and do not permit the filer of a later patent application to antedate the earlier filings of others.  In the case of the CRISPR-Cas9 Technology, UC filed its first priority application on May 25, 2012, and Broad filed more than six months later on December 12, 2012.  In the United States, with respect to patent applications filed prior to March 2013, a subsequent filer could claim to have invented before an earlier filer by filing a declaration in the USPTO, which is what the Broad did and led to the interference proceeding discussed herein.

 

Broad’s related European patents have been opposed by numerous parties on procedural as well as substantive grounds, and are now the subject of proceedings challenging their validity and issuance at the Opposition Division of the European Patent Office.

 

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 Emmanuelle Charpent

ier, Ph.D. CRISPR Therapeutics is headquartered in Basel, Switzerland with its 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 developer of cellular engineering and analysis solutions based on CRISPR technologies. The Company was 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, visit www.cariboubio.com and follow the Company @CaribouBio. “Caribou Biosciences” and the Caribou logo are registered 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 Forward-Looking Statements

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 therapeutic value, development, and commercial potential of CRISPR/Cas-9 gene editing technologies and therapies and the intellectual property protection of our technology 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 in Item 1A under the heading “Risk Factors” in the company’s most recent quarterly report on Form 10-Q, 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 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, statements regarding Intellia’s ability to advance CRISPR/Cas9 into therapeutic products for severe and life-threatening diseases and its CRISPR/Cas9 intellectual property portfolio, and statements regarding the intellectual property position and strategy of Intellia’s licensors. 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 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 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.

 

Disclaimer Regarding Third Party Websites

The announcing companies are providing the above reference to the USPTO’s website as a third-party source of additional factual information relating to U.S. patent interference 106,048, and none of the announcing companies adopt any information contained or found in such reference. Any information in this press release accessible through such reference is as of the date of the release, and the announcing companies undertake no duty to update this information unless required by law.

 

CRISPR CONTACTS

Media Contact:
Jennifer Paganelli
W2O Group for CRISPR
+1 347-658-8290
jpaganelli@w2ogroup.com

Investor Contact:
Chris Brinzey
Westwicke Partners for CRISPR
+1 339-970-2843
chris.brinzey@westwicke.com

INTELLIA CONTACTS

Media Contact:
Jennifer Mound Smoter
SVP, 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

CARIBOU CONTACTS

Greg Kelley
Feinstein Kean Healthcare
+1 404-836-2302
gregory.kelley@fkhealth.com

ERS GENOMICS CONTACTS

MacDougall Communications
Mario Brkulj or Dr. Stephanie May
Direct:  +49 89 2420 9345 or +48 89 2420 9344
E-Mail:  mbrkulj@macbiocom.com or smay@macbiocom.com

Intellia Therapeutics to Present at Leerink Partners 6th Annual Global Healthcare Conference

CAMBRIDGE, Mass., February 1, 2017 – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR/Cas9 technology, announced that Chief Executive Officer and Founder Nessan Bermingham, Ph.D., will present at the Leerink Partners 6th Annual Global Healthcare Conference, Wednesday, February 15, 2017.

Details of the presentation are as follows:

 Date: Wednesday, February 15, 2017

Location: Lotte New York Palace, New York, New York

Time: The presentation will be at 10:00 am ET

Webcast: Available live during the presentation, and through replay on the company’s website immediately following. To participate in the webcast go to Leerink registration.

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 Therapeutics Joins Genomics England’s Industry Consortum

CAMBRIDGE, Mass., and LONDON – January 11, 2017 – Intellia Therapeutics has joined the Genomics England Genomics Expert Network for Enterprises (GENE) Consortium, as the first, dedicated genome editing company to participate in the 100,000 Genomes Project.

The GENE Consortium, established in March 2015, is the 100,000 Genomes Project’s industry partnership. Intellia will join twelve other companies who are working together in a pre-competitive trial. The collaboration aims to identify the most effective and secure way of bringing industry expertise into the 100,000 Genomes Project to realise future potential benefits for patients affected by rare diseases or cancers.  Members of the consortium are granted controlled access to aggregated, de-identified genome and health data of participants. They work alongside experts that specialise in data analysis, so that the project can benefit from cutting edge advances in handling Big Data.

Genomics can greatly improve our understanding of health and disease, unlocking new treatments or repurposing existing treatments based an individual’s genomic makeup; so-called personalised medicine.

Sir John Chisholm, Executive Chairman, Genomics England, said: “The potential for genomics to transform healthcare, from better diagnoses to new drugs and treatments, is extraordinary. We are delighted to welcome Intellia Therapeutics to our GENE Consortium. The UK is a global leader in population sequencing and it’s important for the future of medicine that we continue to attract and collaborate with the most innovative emerging technologies in this space.”

“Access to genomics information is critical as Intellia looks to better understand the basis of disease and to develop potential genome-editing treatments,” said Intellia’s Chief Executive Officer and Founder, Nessan Bermingham, Ph.D., “We look forward to actively participating in the GENE Consortium, as Genomics England is enabling scientific exploration and key medical insights that ultimately will benefit patients.”

 

About Genomics England

Genomics England is a company owned by the Department of Health and was set up to deliver the 100,000 Genomes Project. This flagship project will sequence 100,000 whole genomes from National Health Service patients and their families. Genomics England has four main aims:

  • to bring benefit to patients,
  • to create an ethical and transparent programme based on consent,
  • to enable new scientific discovery and medical insights,
  • to kickstart the development of a UK genomics industry.

The project is focusing on patients with rare diseases, and their families, as well as patients with common cancers.

For more information visit our website www.genomicsengland.co.uk or follow us on Twitter @genomicsengland.

 

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 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.

 Genomics England Contact:

Katrina Nevin-Ridley

Director of Communications

0207 882 6493

katrina.nevin-ridley@genomicsengland.co.uk

 

CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomics Announce Global Agreement on the Foundational Intellectual Property for CRISPR/Cas9 Gene Editing Technology

BASEL, Switzerland, CAMBRIDGE, Mass., BERKELEY, California, DUBLIN, Ireland, December 16, 2016 (GLOBE NEWSWIRE)   – CRISPR Therapeutics (NASDAQ:CRSP), Intellia Therapeutics (NASDAQ:NTLA),  Caribou Biosciences, and ERS Genomics announced that the companies and their licensors have entered into a global cross-consent and invention management agreement for the foundational intellectual property covering CRISPR/Cas9 gene editing technology. The parties to the agreement include the co-owners of the intellectual property – the Regents of the University of California, Emmanuelle Charpentier, and the University of Vienna – as well as key licensees and sublicensees – CRISPR Therapeutics, ERS Genomics, Caribou Biosciences, and Intellia Therapeutics.

Under the agreement, the parties commit to maintain and coordinate the prosecution, defense and enforcement of the CRISPR/Cas9 foundational patent portfolio worldwide, and each of the co-owners of the intellectual property grants cross-consents to all existing and future licenses and sublicenses based on the rights of another co-owner.

“We are pleased that we have come to this global agreement with Intellia, Caribou, ERS and the co-owners and other licensees of this foundational CRISPR/Cas9 technology IP,” said Dr. Rodger Novak, CEO of CRISPR Therapeutics.  “We believe that the Charpentier-University of California-Vienna IP estate constitutes the foundational IP in the CRISPR/Cas9 editing space. Intellia, CRISPR Therapeutics,  Caribou, and ERS view this agreement as enhancing the efforts to protect our shared intellectual property rights and support the ongoing development of our product candidates, as well as those of our corresponding partners and licensees.”

“Through this agreement, we are ensuring alignment in our efforts to protect and prosecute the foundational CRISPR/Cas9 discoveries made by Dr. Doudna, Dr. Charpentier, and their teams, which have transformed the genomics field and unleashed new therapeutic possibilities,” said Nessan Bermingham, CEO and founder, Intellia Therapeutics. “This strengthens Intellia’s IP position as we continue forging ahead with the discovery and development of therapies for patients worldwide.”

Rachel Haurwitz, President and CEO of Caribou Biosciences, added, “We appreciate the efforts of the co-owners and licensees to finalize this agreement and are pleased to move forward as each of our companies develops products using this breakthrough CRISPR/Cas9 foundational IP.”

“This broadly enabling technology will be transformative across such a wide range of areas,” said Eric Rhodes, CEO of ERS Genomics, “and we are thrilled to now be able to offer worldwide access to this important technology.”

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 Emmanuelle Charpentier, Ph.D. CRISPR Therapeutics is headquartered in Basel, Switzerland with its 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 developer of cellular engineering and analysis solutions based on CRISPR technologies. The Company was 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, 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 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 therapeutic value, development, and commercial potential of CRISPR/Cas-9 gene editing technologies and therapies and the intellectual property protection of our technology 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 in Item 1A under the heading “Risk Factors” in the company’s most recent quarterly report on Form 10-Q, 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, statements regarding 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, 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, the risk that Intellia’s collaborations with Novartis or Regeneron will not continue or will not be successful, and risks related to Intellia’s ability to protect and maintain its intellectual property position. 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 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 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
W2O Group for CRISPR
347-658-8290
jpaganelli@w2ogroup.com

Investors:
Chris Brinzey
Westwicke Partners for CRISPR
339-970-2843
chris.brinzey@westwicke.com

CARIBOU CONTACTS

Greg Kelley
Feinstein Kean Healthcare
404-836-2302
gregory.kelley@fkhealth.com 

ERS GENOMICS CONTACTS

MacDougall Biomedical Communications
Mario Brkulj or Dr. Stephanie May
Direct:  +49 89 2420 9345 or +48 89 2420 9344
E-Mail:  mbrkulj@macbiocom.com or smay@macbiocom.com

Intellia Therapeutics Names Graeme Bell Chief Financial Officer

CAMBRIDGE, Mass., December 13, 2016 – Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on developing potentially curative therapeutics using CRISPR/Cas9 technology, has appointed Graeme Bell as its Chief Financial Officer. Mr. Bell succeeds Sapna Srivastava, Ph.D., who is transitioning to a senior advisory role within Intellia.

Mr. Bell joins Intellia from his role as Chief Financial Officer at Anacor Pharmaceuticals, Inc., a publicly traded biopharmaceutical company acquired by Pfizer, Inc. in June 2016. At Anacor, he led the finance, human resources, business development, IT, and facilities functions. Prior to Anacor, Mr. Bell spent more than 20 years at Merck & Co, Inc., where he held numerous finance and accounting senior-level positions including: CFO of U.S. operations, Controller, Global Pharmaceutical Franchises, CFO United Kingdom subsidiary, and head of investor relations. He is a Fellow of the Institute of Management Accountants with an MBA from the University of Durham, UK.

Mr. Bell will relocate to Intellia’s headquarters in Cambridge, Massachusetts, overseeing all financial functions including financial planning and analysis, financial reporting, treasury, tax, accounting, and finance. Additionally, Mr. Bell will be responsible for investor relations.

Dr. Srivastava has decided to transition from the Chief Financial and Strategy Officer role to a senior advisor primarily due to her personal commitments in New York City. Dr. Srivastava joined Intellia in April 2015, and played an integral role in helping start up Intellia’s financial operations and strategic direction, the Series B private funding, and the initial public offering. Dr. Srivastava is committed to ensuring a smooth transition of her responsibilities to Mr. Bell. At the conclusion of her advisory role, Dr. Srivastava will transition from Intellia.

“I want to thank Sapna for her significant contributions to ensuring that Intellia’s business strategy and public offering were successful,” said Chief Executive Officer and Founder Nessan Bermingham, Ph.D. “We welcome Graeme to his new role as he joins us for the next phase of growth in our company. We are excited about his extensive financial and operational experience and existing relationships with the investment community.”

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. These forward looking statements include, but are not limited to, statements regarding our growth as a company and the anticipated contribution of executives to our operations and progress. 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, the risk that any one or more of our 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 our development of our 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, the risk that our collaboration with Novartis or Regeneron will not continue or will not be successful, risks related to the competitive landscape, and risks related to our ability to protect and maintain our intellectual property position. For a discussion of other 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 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.