From Intellia

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

Intellia Therapeutics and CRISPR Therapeutics Announce U.S. Patent Covering CRISPR/Cas9 Ribonucleoprotein Complexes

CAMBRIDGE, Mass., Basel, Switzerland, April 26, 2017, (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and CRISPR Therapeutics AG (NASDAQ:CRSP), two leading genome editing companies focused on the development of potentially curative therapies, announced that the United States Patent and Trademarks Office (“USPTO”) is expected to issue a CRISPR/Cas9 genome editing patent to Vilnius University (“Vilnius”). Intellia and CRISPR are nonexclusive sublicensees for a defined field of human therapeutic, prophylactic, and palliative uses (including companion diagnostics), excluding anti-fungal and anti-microbial applications.

The Vilnius patent claims are directed to CRISPR/Cas9 complexes assembled in vitro and used for site-specific modification of target DNA sequences. CRISPR/Cas9 complexes, referred to as CRISPR ribonucleoproteins or “RNPs,” are contemplated for use in a number of ex vivo applications in which cells, such as blood cells, may be corrected or edited outside of the body before being returned to a patient as a potential therapeutic. The patent is expected to issue on May 2, 2017 as U.S. Patent No. 9,637,739.

This new patent, together with the companies’ respective rights to foundational CRISPR/Cas9 intellectual property co-owned by The Regents of the University of California, University of Vienna and Dr. Emmanuelle Charpentier, provide CRISPR and Intellia with complementary rights to inventions claimed by the earliest developers in the discovery and application of CRISPR/Cas9 technology.

Intellia has a non-exclusive, royalty-free, worldwide sublicense to the Vilnius intellectual property through a 2014 license agreement with Caribou Biosciences, Inc., under which Intellia has an exclusive, worldwide sublicense to certain of Caribou’s developed or in-licensed CRISPR/Cas9 technology intellectual property for a defined field of human therapeutic, prophylactic, and palliative uses (including companion diagnostics), excluding anti-fungal and anti-microbial applications. Caribou has certain rights to Vilnius University’s intellectual property through a cross-license agreement with the DuPont Company.

CRISPR acquired rights to this patent as a result of a cross-option and license agreement with Intellia which was completed in connection with the global agreement on foundational intellectual property for CRISPR/Cas9 gene editing that both companies jointly announced with the co-owners and licensors, as well as another licensee, on December 16, 2016. Under the cross-option and license agreement, CRISPR has a royalty-free worldwide sublicense to Intellia’s rights to the Vilnius intellectual property.

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 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 Intellia on Twitter @intelliatweets.

About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases using the 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 crisprtx.com.

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’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 coverage and positions of the company, its licensors and 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.

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

CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomics Announce Appeal of CRISPR/Cas9 U.S. Patent Board Decision

  • Appeal to the U.S. Court of Appeals for the Federal Circuit seeks review and reversal of the Patent Trial and Appeals Board’s decision to terminate CRISPR/Cas9 interference
  • In parallel, the companies and their licensors plan to pursue additional patents in the U.S. and worldwide covering the CRISPR/Cas9 technology and its use in cellular and non-cellular settings, including eukaryotic cells

BASEL, Switzerland; CAMBRIDGE, Massachusetts; BERKELEY, California; DUBLIN, Ireland; April 13, 2017 (GLOBE NEWSWIRE) – CRISPR Therapeutics (NASDAQ:CRSP), Intellia Therapeutics (NASDAQ:NTLA), Caribou Biosciences and ERS Genomics announced today 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, have appealed to the U.S. Court of Appeals for the Federal Circuit (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 (collectively, “Broad”).

In the appeal, UC is seeking review and reversal of the PTAB’s February 15, 2017 decision, which terminated the interference without determining which inventors actually invented the use of the CRISPR/Cas9 genome editing technology in eukaryotic cells. In its decision, the PTAB concluded that, although the claims overlap, the respective scope of UC and Broad’s claim sets as presented did 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. UC is asking the Federal Circuit to review and reverse the PTAB’s decision.

In parallel with the appeal, UC is pursuing applications in the U.S. and other jurisdictions worldwide to obtain patents claiming the CRISPR/Cas9 technology and its use in non-cellular and cellular settings, including eukaryotic cells. Corresponding patents have already been granted in the United Kingdom, and the European Patent Office is also granting a patent to UC, which will issue on May 10, 2017. UC’s earliest patent application describing the CRISPR/Cas9 genome editing technology and its use was filed on May 25, 2012, while the Broad’s earliest patent application was filed more than six months later, on December 12, 2012.

The law firm of Munger, Tolles & Olson LLP will be handling the appeal, with Don Verrilli, former Solicitor General of the United States, as lead counsel.

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 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 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 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 Announces European Patent Office’s Decision to Grant CRISPR/Cas9 Genome Editing Technology Patent

– Patent covers foundational CRISPR/Cas9 inventions that Intellia sublicensed for use in human therapeutics
– Patent covers compositions of CRISPR single guide RNA technology for use in non-cellular and cellular settings, including eukaryotic cells

CAMBRIDGE, Mass., April 11, 2017 (GLOBEWIRE) – Intellia Therapeutics (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, announced that the European Patent Office (EPO) has decided to grant 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 and cellular setting, including eukaryotic cells such as human or mammalian cells, as well as for use in human therapeutics.

According to the EPO, the patent will formally grant on May 10, 2017. The EPO’s decision to grant this patent follows its March 24, 2017 notice of intent to issue the patent, which was not challenged by any third party. This European patent will be nationalized in, and cover, approximately forty European countries, including Germany, Italy, France, Spain and the Netherlands. As provided by relevant European legislation, third parties will have nine months from the issue date to oppose the patent in the EPO.

In addition to the EPO decision, earlier this year, the United Kingdom’s Intellectual Property Office granted national UK patents on the CRISPR/Cas9 genome editing system. The UK patents cover the single guide RNA for uses in both non-cellular and cellular settings, as well as chimeric CRISPR/Cas9 systems in which the Cas9 protein is modified to provide alternative DNA-modulating activities. The underlying international patent application is based on a U.S. application, which was filed on May 25, 2012, by the University of California on its own behalf and on behalf of the University of Vienna and Dr. Emmanuelle Charpentier. In the U.S., the corresponding application has been involved in an interference proceeding with the Broad Institute, Harvard University and the Massachusetts Institute of Technology, which was terminated without a decision on which sets of inventors were the first to discover the application of the CRISPR/Cas9 technology to eukaryotic cells.

“We are extremely pleased with this EPO outcome as it recognizes Jennifer Doudna, Emmanuelle Charpentier and their team as CRISPR/Cas9 pioneers, and also acknowledges the breadth of their original patent application,” said Intellia Therapeutics CEO and Founder, Nessan Bermingham, Ph.D. “Intellia continues to build on the compelling preclinical data we have generated and to focus on the development of our pipeline of novel human therapeutics that will potentially transform the lives of patients with genetic diseases.”
Intellia has rights to this intellectual property estate, 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.

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.

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 the formal issue date of the European patent and the nationalization of the European patent in approximately forty countries; 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.

 

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 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.

Screen Shot 2017-03-13 at 10.48.46 PM

Screen Shot 2017-03-13 at 10.48.54 PM

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.