Category Archives: Genetic Therapy

BOSTON--(BUSINESS WIRE)--Akouos, a precision genetic medicine company developing gene therapies to potentially restore, improve and preserve hearing, announced today that data from its inner ear gene therapy platform will be presented during the 23rd American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, which will be held virtually May 12-15, 2020.

Two poster presentations will highlight Akouoss use of AAVAnc80 vector technology and its potential to address many forms of hearing loss. Presentation details are as follows:

Title:

Use of the Adeno-Associated Viral Anc80 (AAVAnc80) Vector for the Development of Precision Genetic Medicines to Address Hearing Loss

Date and Time:

Tuesday, May 12, 2020 5:30 PM - 6:30 PM (EST)

Title:

Enabling Temporal Control of Gene Expression in the Inner Ear after AAVAnc80 Vector Mediated Delivery

Date and Time:

Wednesday, May 13, 2020 5:30 PM - 6:30 PM (EST)

About Akouos

Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, the Company was founded in 2016 by world leaders in the fields of neurotology, genetics, inner ear drug delivery and AAV gene therapy. Akouos has strategic partnerships with Massachusetts Eye and Ear and Lonza, Inc. For more information, please visit http://www.akouos.com.

About AAVAnc Technology

The Ancestral AAV (AAVAnc) platform was developed in the laboratory of Luk Vandenberghe, Ph.D., director of the Grousbeck Gene Therapy Center at Harvard Medical School. AAVAnc technology uses computational and evolutionary methods to predict novel conformations of the adeno-associated viral particle. AAVAnc80, one of approximately 38,000 AAVAnc vectors, has demonstrated preliminary safety and effective gene delivery in both mice and non-human primates in preclinical studies.

Originally posted here:
Akouos to Present Data from Inner Ear Gene Therapy Platform at 23rd ASGCT Annual Meeting - Business Wire

Vertex is partnering with Affinia to develop gene therapies for DMD, among other conditions. Credit: Shutterstock. Vertex's campus in Boston, US. Credit: Shutterstock.

At the end of April, Vertex Pharmaceuticals and Affinia Therapeutics initiated a strategic research collaboration. Under the terms of the agreement, Affinias novel adeno-associated virus (AAV) capsid engineering expertise will be leveraged to develop gene therapies for Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF).

Affinia has created the proprietary AAVSmartLibrary, as well as associated technology, to create improved capsids, which are viral protein shells. AAV capsids are seen as a particularly promising delivery method for in vivo gene therapies, since AAVs are non-pathogenic and cannot be integrated into the genome of target cells, as explained by Harvard Universitys Wyss Institute.

According to the terms of the collaboration, Affinia will take responsibility for the discovery of the capsids, while Vertex will oversee the design and manufacturing of the gene therapies that incorporate these capsids, as well as clinical development and commercialisation.

This agreement gives Vertex an exclusive license to Affinias proprietary technology and intellectual property in DMD and DM1, as well as a right to license rights for CF and another undisclosed condition.

Affinia will be eligible for over $1.6bn in upfront payments and development, regulatory and commercial milestones $80m of which can be paid by Vertex during the research agreements term.

Why Affinia Therapeutics?

We performed a thorough landscape search for AAV technologies that could provide novel capsids with exceptional properties for the development of genetic therapies in multiple disease areas, explains Vertex spokesperson Heather Nichols. Affinias technology platform was very attractive because the libraries are designed rationally, resulting in a highly bioactive library that can be used as a starting point to optimise capsids with desired therapeutic profiles.

In addition, Nichols notes the scientific expertise that underlies Affinias work. Its technology was licensed from Lonza and Massachusetts Eye and Ear, and then further developed by Dr Luk Vandenberghe, a world-leading AAV expert who is a scientific co-founder of the company. Nicholls concludes: Together, the innovative technology and outstanding people made Affinia the clear choice for our partnership.

Affinia CEO Rick Modi stated: We are thankful for the scientific validation this partnership brings and look forward to working closely with [Vertex] to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases.

The small companys promise is also clear from its closing of an oversubscribed $60m Series A financing round in March 2020. This funding was co-led by F-Prime Capital and New Enterprise Associates (NEA), and supported by Atlas Venture, Alexandria Venture Investments, Partners Innovation Fund and Lonza.

NEA general partner Ed Mather noted: Affinia Therapeutics methodical process for designing and evaluating vectors is a differentiated approach to gene therapy, and the highly experienced leadership team will help carry these discoveries to the development, manufacturing and commercialisation of transformative medicines.

Becoming part of Vertexs toolkit

This deal builds on Vertexs strong history in treating the genetic, underlying causes of serious diseases. The company has been a major player in the CF field since its founding over three decades ago; it currently has four drugs approved that target the underlying genetic basis of CF.

However, over the past few years, Vertex has committed to expanding its expertise to treating other serious diseases caused by genetic mutations or defects. It has been committed to building a toolkit of technologies and capabilities that will allow us to pursue the diseases were interested in from all angles including genetic therapies, Nichols adds.

Affinia Therapeutics innovative approach to the discovery and design of AAV capsids brings yet another tool to ourVertexCell and Genetic Therapies toolkit, and were excited to partner with them to bring together their technology platform with our research and development expertise, said Vertex executive vice-president and chief of cell and gene therapies Bastiano Sanna.

Other members of Vertexs tool kit focusing on the DMD and DM1 field specifically include CRISPR Therapeutics and Exonics Therapeutics.

In June last year, Vertex expanded an existing four-year collaboration with CRISPR to develop new treatments for genetic causes of human diseases. The initial focus of the agreement was transfusion-dependent beta thalassemia and sickle cell disease; in November, the partners announced positive interim data from their investigational therapy CTX001 in Phase I/II studies in these two patient groups.

Through the expanded collaboration with CRISPR and the acquisition of Exonics, we are bringing together the intellectual property, technologies, and scientific expertise needed to establish a leading gene editing platform for DMD and DM1, stated Vertex CEO, chairman and president Jeffrey Leiden.

Other therapeutic areas also on Vertexs gene-focused radar are type 1 diabetes through a deal with Semma signed in September last year.

Impact on Vertexs share price

Vertex is headquartered in Boston, Massachusetts, and is listed on the New York Stock Exchange.

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Vertex-Affinia partnership: expanding the gene therapy toolkit - Pharmaceutical Technology

BOSTON and WUHAN, China, May 11, 2020 /PRNewswire/ -- Neurophth Therapeutics, Inc., a clinical-stage biotechnology company discovering and developing innovative therapies for ocular and other genetic disease, today announced that the results of two clinical studies utilizing NFS-01 (rAAV2-ND4) in the treatment of Leber's Hereditary Optic Neuropathy (LHON) will be presented in May 2020 at the American Society of Gene and Cell Therapy (ASGCT) 23rd Annual Meeting and the Association for Vision and Ophthalmology (ARVO) Annual Meeting.

Founder Bin Li, M.D., Ph.D., professor of ophthalmology at the Tongji Medical College of Huazhong University of Science and Technology (HUST), initiated the first China gene therapy clinical trial in 2011, evaluating an adeno-associated virus serotype 2 (AAV2)-based gene therapy in nine Leber's Hereditary Optic Neuropathy (LHON) patients with the G11778A (ND4) mutation. Patients have been followed for over 7 years, several demonstrating significant visual acuity improvement and none reporting severe adverse events.

"The exciting results of the first study (NCT01267422) have been instrumental in developing the largest international clinical study (NCT03153293) in 159 LHON patients, who have been followed for 12 months," said Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer of Neurophth. "Our results from NFS-01 demonstrate the potential of gene therapy to be both effective and safe for the treatment of LHON and further validate Neurophth's platform which is being applied across a growing pipeline of pre-clinical and clinical programs in gene therapy to treat a wide range of inherited diseases."

ASGCT: American Society of Gene and Cell Therapy Virtual Annual Meeting 2020Presentations can be accessed through ASGCT's website at http://www.asgct.org on May 12, 2020.

First China International Gene Therapy Study in Leber's Hereditary Optic Neuropathy (Abstract #1307)Oral presentation by Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer, NeurophthSession Date/Time: Friday, May 15, 10:45AM EDTSession Title:AAV Vectors - Clinical Studies I

Multiyear Follow-up of AAV2-ND4 Gene Therapy for Leber's Hereditary Optic Neuropathy (Abstract #621)Poster presentation by Su Xiao, Ph.D., Co-Founder and Chief Technical Operations Officer, NeurophthSession Date/Time: Wednesday, May 13, 5:30PM EDTSession Title:AAV Vectors - Clinical Studies

ARVO: Association for Vision and Ophthalmology The ARVO 2020 Annual Meeting has been modified to a virtual format and presentations have been published online on May 6, 2020.

First China International Gene Therapy Study in Leber's Hereditary Optic Neuropathy (Abstract #5182)Oral Presentation by Alvin Luk, Ph.D., M.B.A., C.C.R.A., Chief Executive Officer, NeurophthNarrated presentation will be available onlineScientific Section: Eye Movements/Strabismus/Amblyopia/Neuro-Ophthalmology

About Neurophth Therapeutics, Inc.

Neurophth Therapeutics, Inc. ("Neurophth") is China's first gene therapy company for ophthalmic diseases. Founded in 2016 by Professor Bin Li, M.D., Ph.D., the clinical-stage biotechnology company is dedicated to discovering and developing gene therapies for ocular and other genetic diseases. Currently, Neurophth is advancing several clinical-stage and pre-clinical programs in ophthalmology. In April 2020, Neurophth successfully raised $18.4 million USD in a Series A round led by Fosun's InnoStar Venture and Sequoia Capital China. The company plans to bring the lead assets to patients globally and build a GMP gene therapy manufacturing facility for global supply.

About NFS-01

NFS-01 is an AAV construct comprising with promoter, mitochondrial targeting sequence (MTS), correct ND4 gene sequence, and an untranslated region flanked by two inverted terminal repeats (ITRs). In 2011, Neurophth initiated the world's first investigational NFS-01 (AAV2-ND4) gene therapy study (NCT01267422) of a single intravitreal injection into one of the eyes of patient with ND4 mutation associated with Leber's Hereditary Optic Neuropathy (LHON). The corrected ND4 gene is transferred into the cell to be expressed and eventually produces the missing functional protein to restore mitochondrial function of the respiratory chain. After nearly 8 years of long-term follow-up, the treatment displayed good safety profiles and patients have maintained significant visual acuity improvement to-date. These results demonstrated long-term safety and durability of clinical application of gene therapy technology and were published in Ophthalmology 2020. Encouraged by these results, Professor Li's team has completed an international gene therapy clinical trial (NCT03153293) during 2017-2018 in 159 LHON patients in China and Argentina, which is the world's largest clinical trial for LHON gene therapy.

About Leber's Hereditary Optic Neuropathy (LHON)

Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited mitochondrial disorder which often manifests as bilateral visual loss. The most common mutation is the mitochondrial DNA at nucleotide position of 11778G>A, which is located within the NADH ubiquinone oxidoreductase subunit-4 (ND4) gene. This mutation causes impairment of the respiratory chain, adenosine triphosphate deficiency, and oxidative stress in retinal ganglion cells, leading to apoptosis. There is currently no effective treatment or cure for LHON.

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Neurophth Therapeutics Announces Presentations at ASGCT and ARVO Annual Meetings - Woburn Daily Times

(PRESS RELEASE) CHICAGO In order to help patients with Inherited Retinal Diseases (IRDs) receive an accurate diagnosis through genetic testing, Prevent Blindness, the nations oldest volunteer eye health organization, is embarking on a new awareness initiative to educate the public on IRDs, a group of genetic disorders that can cause severe vision loss or total blindness. A recent editorial noted that IRDs are the leading cause of vision loss in persons between 15 and 45 years of age- impacting one in every 2,000 people.

To continue the Prevent Blindness mission of preventing blindness and saving sight, the group has declared May 17-23, 2020 as Inherited Retinal Disease Genetic Testing Week. As part of this initiative, Prevent Blindness has created a dedicated webpage (to go live on May 15, 2020) with no-cost educational resources on IRDs, providing detailed information on risk factors, therapy and research options, financial assistance services, the importance of genetic testing, and more. Shareable social media graphics on IRDs are also available. Development of these new resources was supported by a donation from Spark Therapeutics, a gene therapy company striving to challenge the inevitability of genetic disease.

The most common types of IRDs include:

All those diagnosed with IRDs should work with their healthcare professional to develop a disease management plan tailored to the patients needs.

Patients with suspected IRDs are encouraged to participate in genetic testing, even those who were tested more than five years ago and did not receive a definitive result. According to the National Institutes of Health, genetic testing is a type of medical test that identifies changes in chromosomes, genes, or proteins. The results of a genetic test may provide a detailed diagnosis which confirms or rules out most suspected genetic conditions and the test can help determine a persons chance of developing or passing on a genetic disorder to their children. Free genetic testing is now available from select organizations and commercial businesses, including through the ID YOUR IRD program.

Inherited retinal diseases can have a significant impact on the quality of life for patients, including affecting the ability to work and live independently, said Jeff Todd, president and CEO of Prevent Blindness. Fortunately, today there are more therapies being researched than ever before. We encourage patients and their caregivers to educate themselves on steps that can be taken today to save sight for tomorrow.

As a leading gene therapy company with a strong commitment to genetic testing for the IRD community, Spark Therapeutics is pleased to support the Prevent Blindness inaugural IRD Genetic Testing Week, said Dan Chung, DO, MA, ophthalmology therapeutic area leader, Spark Therapeutics. We encourage patients living with IRDs and caregivers to pursue genetic testing in order to receive a true, genetic diagnosis.

For more information on IRDs and genetic testing for vision issues, please call Prevent Blindness at (800) 331-2020 or visit here. For a listing of vision care financial assistance programs in English or Spanish, visit here.

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Prevent Blindness Offers New Educational Resources on IRD's, Importance of Genetic Testing and Services - InvisionMag

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, May 11, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.

The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy, said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for such disease or condition. Similar to Breakthrough Therapy designation, RMAT designation provides the benefits of intensive FDA guidance on efficient drug development, including the ability for early interactions with FDA to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.

In addition to RMAT designation, CTX001 has received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

About CTX001CTX001 is an investigational ex vivo CRISPR gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and painful and debilitating sickle crises for SCD patients. CTX001 is the most advanced gene-editing approach in development for beta thalassemia and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic partnerships with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of RMAT designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, 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: the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://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 Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the information provided regarding the status of, and expectations with respect to, the CTX001 clinical development program and related global regulatory approvals, and expectations regarding the RMAT designation. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that the development of CTX001 may not proceed or support registration due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-961-0534orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001 for the...

CAMBRIDGE, Mass., May 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology bothin vivoandex vivo,is presenting three oral presentations and two poster presentations at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place virtually from May 12-15, 2020. Intellia researchers are presenting new data in support of NTLA-5001, the companys engineered cell therapy candidate for the treatment of acute myeloid leukemia (AML). Intellia is also providing an update on NTLA-2002, its newest development candidate for the treatment of hereditary angioedema (HAE).

At Intellia, we are applying our CRISPR/Cas9 technology to develop new processes that can produce enhanced engineered cell therapies to treat severe cancers, such as AML, that traditional approaches cannot address. Our proprietary platform provides a powerful tool to generate more potent TCR-directed cells, that can treat blood cancers initially and potentially solid tumors. The data being presented today validate Intellias approach of reducing AML tumor cell blasts, and our plans to enter the clinic with NTLA-5001 next year, said Intellia President and CEO John Leonard, M.D. We are also pleased to present data that support our recently announced HAE development candidate, NTLA-2002, Intellias second systemic therapy employing our in vivo knockout approach and modular delivery platform.

Data Presentations on Intellias First Engineered Cell Therapy Development Candidate, NTLA-5001 for the Treatment of AML, and Proprietary Cell Engineering Process

NTLA-5001 is Intellias first engineered T cell receptor (TCR) T cell therapy development candidate, which targets the Wilms Tumor 1 (WT1) intracellular antigen for the treatment of AML. NTLA-5001 is being developed in collaboration with Chiara Boninis team at IRCCS Ospedale San Raffaele to treat AML patients regardless of the genetic subtype of a patients leukemia. AML is a cancer of the blood and bone marrow that is rapidly fatal without immediate treatment and is the most common type of acute leukemia in adults(Source:NIH SEER Cancer Stat Facts: Leukemia AML).

Intellias proprietary process is a significant improvement over standard engineering processes commonly used to introduce nucleic acids into cells. Intellias process enabled multiple gene edits using CRISPR/Cas9, while maintaining cell products with high expansion potential and minimal undesirable chromosomal translocations. CRISPR/Cas9 was used to insert a WT1-directed TCR in locus, while eliminating the expression of the endogenous TCRs, with the goal of producing homogeneous T cell therapies like NTLA-5001.

Intellias novel approach with NTLA-5001 can overcome the challenges of standard T cell therapy, including risks of reduced specificity associated with mixed expression and mispairing of endogenous and transgenic TCRs (tgTCRs); graph-versus-host disease (GvHD) risks, which could lead to an attack on the patients healthy cells; and reduced efficacy tied to lower tgTCR expression per T cell. Intellias unprecedented process is expected to streamline cell engineering and manufacturing, yielding a homogenous product comprising WT1-targeted T cells with high anti-tumor activity. Data highlights from todays presentation include the following:

Intellias cell engineering efforts are focused on its initial clinical investigation of NLTA-5001 on AML, while continuing preclinical studies exploring the potential for targeting WT1 in solid tumors. The company confirmed plans last week to submit an IND or IND-equivalent for NTLA-5001 for the treatment of AML in the first half of 2021.

The presentation titled, Enhanced tgTCR T Cell Product Attributes Through Process Improvement of CRISPR/Cas9 Engineering, will be made today by Aaron Prodeus, Ph.D., senior scientist, Cell Therapy, and can be found here, on the Scientific Publications & Presentations page of Intellias website. These data were a follow-on to the study presented at Keystone Symposias Engineering the Genome Conference from this past February.

In Vivo Data Supports Intellias Novel TCR Candidate

A second presentation on engineered cell therapy progress, in collaboration with IRCCS Ospedale San Raffaele, showed in vivo data demonstrating the potential of TCR-edited T cells to effectively target WT1 tumor cells in AML. In addition to the previously disclosed results of effective in vitro recognition of primary AML tumor cells by edited WT1-specific cytotoxic T cells (CD8 T cells), new data indicate that the selected TCR also enables T helper cells (CD4 T cells) to react to WT1-expressing tumor cells, providing cytokine support. This distinguishes Intellias TCR from other therapeutic TCR candidates, which either exclusively activate toxic CD8 T cells or require the co-transfection of CD8 into CD4 T cells to render them functional.

Using a mouse model carrying disseminated human primary AML, researchers observed a significant therapeutic effect, including decreased AML tumor burden. In addition, no signs of GvHD were observed in mice treated with the WT1-specific T cells. The data show that tgTCR-engineered cells have targeted anti-cancer activity in a challenging model of systemic AML, demonstrating the therapeutic potential of Intellias engineered TCR T cell approach.

The presentation titled, Exploiting CRISPR-Genome Editing and WT1-Specific T Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid Leukemia, will be given today by Eliana Ruggiero, Ph.D., Experimental Hematology Unit, Division of Immunology, Transplantation and Infectious Diseases, IRCCS Ospedale San Raffaele, Italy. Notably, ASGCT meeting organizers selected this presentation as one of six to receive the ASGCT Excellence in Research Award this year.

Continued Progress on Intellias Second In Vivo Development Candidate, NTLA-2002 for the Treatment of HAE

Intellia is presenting development data updates on its potential HAE therapy, NTLA-2002, which utilizes the companys systemic in vivo knockout approach, including its proprietary lipid nanoparticle (LNP) system. HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating or even fatal in certain cases. NTLA-2002 aims to prevent unregulated production of bradykinin by knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.

The KLKB1 gene knockout in an ongoing non-human primate (NHP) study resulted in a sustained 90% reduction in kallikrein activity, a level that translates to a therapeutically meaningful impact on HAE attack rates(Source: Banerji et al., NEJM, 2017). This kallikrein activity reduction was sustained for at least six months, demonstrating the same high level of efficacy and durability seen in earlier rodent studies.

The short talk titled, CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat Hereditary Angioedema, will be given by Jessica Seitzer, director, Genomics, Intellia on Fri., May 15, 2020, when it will be made available here, on the Scientific Publications & Presentations page of Intellias website. The presented data include results from ongoing collaborations with researchers at Regeneron, and the program is subject to an option by Regeneron to enter into a Co/Co agreement, in which Intellia would remain the lead party. Intellia expects to submit an IND or IND-equivalent to initiate a Phase 1 trial for NTLA-2002 in the second half of 2021.

About Intellia Therapeutics

Intellia Therapeuticsis a leading genome editing company focused on developing proprietary, 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, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more aboutIntellia Therapeuticsand CRISPR/Cas9 atintelliatx.comand follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) 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 Intellias beliefs and expectations regarding its: planned submission of an investigational new drug (IND) application or similar clinical trial application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020 and its planned dosing of first patients in the second half of 2020; plans to submit an IND application for NTLA-5001, its first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program in the first half of 2021; plans to submit an IND or similar clinical trial application for its hereditary angioedema (HAE) program in the second half of 2021; plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program and HAE programs, and other animal studies supporting other in vivo and ex vivo programs, including its AML program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion; further development of its proprietary cell engineering process for multiple sequential editing; presentation of additional data at upcoming scientific conferences, and other preclinical data in 2020; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR, AML, and HAE programs, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Novartis or Regeneron Pharmaceuticals, Inc., and Regenerons ability to enter into a co-development and co-promotion agreement for the HAE program; statements regarding the timing of regulatory filings regarding its development programs.

Any forward-looking statements in this press release are based on managements 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 Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Novartis or Regeneron or its other ex vivo collaborations 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 Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917 lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Reports Progress on CRISPR/Cas9 AML Cancer Therapy Using Proprietary Cell Engineering Process at the 23rd Annual Meeting of the...