Category Archives: Genetic Therapy

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...

Amber Freeds singular mission is to help scientists develop a treatment for her son. But the pandemic has put the success of that mission into question.

Freed is a mother to twins Riley and Maxwell, who just turned 3 this year. Riley is a healthy toddler. But Maxwell has a rare genetic disease that has led to delays in his development. Hes nonverbal, and he has a movement disorder. The condition responsible for these symptoms is called SLC6A1, after the gene thats affected, and Maxwell was only the 34th person in the world to get this diagnosis. if left untreated, the disease could soon cause debilitating epilepsy.

When doctors broke the news of the diagnosis to Freed, they told her there was no treatment or cure for her son, and that her family would have to prepare to live with Maxwells condition. But Amber didnt accept that.

Every instinct in my body said, Youll have your whole lifetime to cry for yourself, Freed said. At this exact moment, you put your feelings and sadness aside. This isnt about you. This is about Maxwell. And you fight like the third monkey on the loading deck to Noahs Ark, and its starting to rain.

This is how Freeds quest began. She taught herself microbiology, so she could understand her sons condition. She lobbied scientists to take up the research, which wasnt on their radar given how rare the disease is. She convinced scientists in China to make genetically engineered mice that could be sent to the US and act as a model of Maxwells disease. And she fundraised a necessary step given the high cost of the gene therapy research.

All told, shes raised more than $1 million. What were doing will be the building blocks of all gene therapies to come after us, Freed told Arielle Duhaime-Ross on an episode of Reset in January.

Even though she was racing against time, she was optimistic that Maxwell would be treated this year, as experiments on the genetically modified mice were about to begin.

Then the pandemic hit.

Covid-19 has brought most non-coronavirus medical research to a halt, and closed labs.

I never anticipated or thought something like this could happen, Freed said, but also it was a state of devastation like I felt when Maxwell was originally diagnosed that lightning hit us. The unthinkable happened. And here, lightning has hit again. A black swan event for the world that no one could have anticipated.

Freed came back to Reset to talk about the impact of the pandemic on Maxwell and her family as well as rare disease research as a whole. Listen to the full episode below.

The episode also features a conversation with BuzzFeed science reporter Dan Vergano, who first wrote about Ambers story and who talks about how the closure has impacted thousands of other kids with rare diseases, as well as clinical trials for other types of medical research.

Listen and read more:

Listen to the original Reset story from January

Dan Verganos reporting on this story for BuzzFeed

Back in January, Dans piece on how the mice were being used to develop a treatment

Milestones for Maxwell, Amber Freeds website

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A treatment for this toddlers rare genetic condition was in sight. Then the pandemic hit. - Vox.com

ALLENDALE, NJ., USA and MUNICH, Germany, May 11, 2020 / B3C newswire / -- Hitachi Chemical Advanced Therapeutics Solutions, LLC (HCATS) and apceth Biopharma GmbH (apceth), both subsidiaries of Hitachi Chemical Co., Ltd. (Hitachi Chemical) today announced that they have expanded their relationship with bluebird bio (NASDAQ: BLUE) with long-term development and manufacturing services agreements for clinical and commercial supply for multiple therapies, including:

These agreements are the latest in a long-standing partnership between bluebird bio and Hitachi Chemical. In 2011, HCATS, which represents the North America region of Hitachi Chemicals global regenerative medicine business, and bluebird bio entered into their first clinical services agreement. A commercial drug product manufacturing service agreement was also established between bluebird bio and apceth (which represents the Europe region of Hitachi Chemicals global regenerative medicine business) in 2016. In January 2020, apceth announced its readiness to begin commercial manufacturing of ZYNTEGLO with bluebirds announcement of the launch in Germany.

"With three products in our severe genetic disease franchise to potentially launch between now and 2022, securing long-term commercial drug product manufacturing capacity is critical to our ability to deliver for patients, " said Nick Leschly, chief bluebird. "Our partnership with Hitachi Chemical is a significant example of our continued progress on this front and we believe Hitachi Chemicals recent expansion will help support our growing commercial needs. We are pleased to benefit from their expertise as well as their footprint in both the US and Europe as we work to bring transformative therapies to patients in need."

We are excited to partner with bluebird bio through our new U.S. facility, utilizing our state-of-the-art capabilities and systems for late-stage clinical testing and ultimately commercial production once all applicable regulatory approvals are granted, said Robert Preti, Ph.D., Chief Strategy Officer, Hitachi Chemical Life Science Business Headquarters. It is our honor to support bluebird bio in the manufacture of their potentially transformative gene therapies, to the benefit of patients in both the United States and Europe, as the foundation for our collaboration to address this devastating disease

We are very happy to deepen our trustful and productive strategic partnership with bluebird bio, commented Dr. Christine Guenther, Deputy General Manager of the Hitachi Chemical Regenerative Medicine Business Sector and CEO of apceth Biopharma GmbH. The apceth team is proud to be part of bluebird bios most exciting pioneering work for the advancement of cell and gene therapies and to supply patients suffering from severe genetic illnesses with potentially life-changing treatments.

This medicinal product is subject to additional monitoring.

About Hitachi Chemicals Regenerative Medicine BusinessHitachi Chemical provides cell and gene therapy contract development and manufacturing organization (CDMO) services at current Good Manufacturing Practices (cGMP) standards, including clinical manufacturing, commercial manufacturing, and manufacturing development. The global footprint of the business is over 200,000 square feet and includes operations in North America (Allendale, New Jersey and Mountain View, California), Europe (Munich, Germany), and Japan (Yokohama). The business leverages two decades of experience exclusively focused on the cell therapy industry.

For more information on North America services, please visit http://www.pctcelltherapy.comFor more information on Europe services, please visit http://www.apceth.comFor more information on Japan services, please visit http://www.hitachi-chem.co.jp/english

Contacts

Hitachi Chemical Advanced Therapeutics SolutionsEric PowersDirector, Marketing and CommunicationsThis email address is being protected from spambots. You need JavaScript enabled to view it.

apceth Biopharma GmbHAlmut WindhagerManager, Business Development and CommunicationsThis email address is being protected from spambots. You need JavaScript enabled to view it.

Keywords: Humans; Regenerative Medicine; Thalassemia; HLA Antigens; Hematopoietic Stem Cell Transplantation; Anemia, Sickle Cell; Hematopoietic Stem Cells; Genetic Therapy

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Hitachi Chemical Advanced Therapeutics Solutions and apceth Biopharma GmbH Enter into Strategic Clinical and Commercial Manufacturing Agreements with...

Chicago hedge fund firm Citadel is known for buying and selling stocks, bonds and derivatives across public markets. But lately it's been pumping millions of dollars into private biotech companies.

The firm's Surveyor Capital fund has invested about $150 million in 17 such companies, mainly in the biopharmaceutical field, since February 2018.

That venture investing is a small portion of billionaire Ken Griffin's $30 billion Citadel business, but it's part of a striking and expanding departure from his 30-year focus on public markets. Placing capital with young biotech companies can be risky, but Surveyor has captured big gains in initial public offerings. Those types of returns have increasingly attracted investors to the biotech sector over the past decade, and that wave of interest is likely to keep climbing with the new hunt for coronavirus treatments.

"We're really at a significant growth phase in biotech in terms of new treatments hitting the market," says Sean Nolan, the former CEO of Chicago-based AveXis, a gene therapy company that combats a rare genetic disease in children. "The need created by the COVID virus is going to spur opportunities in biotech to develop treatments and vaccines."

While Surveyor isn't in coronavirus-specific startups, Seattle research firm Pitchbook has detailed the breadth and depth of Citadel's investing in the area, echoing press statements from fledgling biotech companies that have received the money. Citadel spokeswoman Megan Ingersoll declines to comment.

Surveyor, with 175 investment professionals, is one of a handful of Citadel funds investing worldwide, pursuing equity strategies to deliver returns for Griffin, clients and firm employees. So far this year, Citadel's flagship Wellington fund has achieved a 10% gain through April, according to a person familiar with the results, despite the market rout.

Surveyor bought AveXis shares at $20 apiece in a February 2016 initial public offering and shared in its windfall less than two years later when industry giant Novartis bought AveXis for $8.7 billion, or $218 a share, in April 2018. "Surveyor, at least in the AveXis instance, had a long view and was a very good investor along the way," Nolan says.

Now a different Surveyor team is getting into biotech companies pre-IPO, even earlier in the attempt to move a research idea through clinical trials to federal regulatory approvals to production. There are bigger gains to be had from investing at lower valuations before companies go public, but there are also more chances to stumble. "The venture capitalists are there earlier because they're willing to take more risk," Nolan says.

Surveyor always invests as part of a group that doesn't disclose how much each investor contributes, but based on the group amounts and dynamics, Surveyor is likely in for about $5 million to $15 million per company.

So far this year, Surveyor joined with other investors to place $170 million with Cambridge, Mass., gene and cell therapy accelerator ElevateBio; $105 million with Boston-based hearing loss therapy company Akouos; $100 million with South San Francisco-based Pliant Therapeutics, which is developing treatments to fight fibrotic diseases; and $88 million with San Francisco-based Spruce Biosciences, targeting treatments to battle endocrine disorders.

At that rate, Surveyor is on track to exceed the eight investments it made last year and the five in 2018. The dollar amounts of Surveyor's group investments have also mainly climbed, perhaps reflecting the arena's rising allure.

The IPO typically gives a 30% boost to investments, says Keith Crandell, whose Chicago-based investment firm, Arch Venture Partners, has carved out a niche in biotech venture investing. "Generally, you see the values pop, so there's talk about arbitraging that private-public step-up," says Crandell, an Arch managing director.

More investment funds are crossing over from public to private markets to grab that upside, he says. The closer companies get to approval for starting trials on humans, the more capital they require and the bigger sums they attract. "If you have a success in the clinic, it's worth a lot of money," he says, but "none of these are certain things."

Still, it takes only a couple of big wins to make a pack of investments successful. Surveyor's $85 million group investment in New York-based cancer treatment company Zentalis Pharmaceuticals in December delivered big returns last month when the company went public. Its shares, priced at $18 in the IPO, jumped as much as 50% on their first day of trading before closing at $23.20 and have since climbed to $31.93.

Surveyor's pre-IPO investment in Beltsville, Md.-based NextCure in November 2018 also panned out. In the May 2019 first-time stock sale, shares priced at $15 apiece and have more than doubled a year later, closing at $35.89 on May 7.

Crandell observes that a significant portion of Surveyor's investments are in gene and cell therapy and in cancer-fighting treatments. Those therapies have "performed well," particularly in oncology, "because the science has proved out," he says.

Surveyor may be increasing its odds by backing startups that have already landed "top-tier" venture capitalists in earlier stages of fundraising, he says, noting prominent firms like Third Rock Ventures and Canaan Partners. Surveyor's group co-investors aren't slouches, either, including Fidelity Management & Research and Franklin Templeton Investments.

Nonetheless, some of Surveyor's picks have declined. It invested in Sutro Biopharma in July 2018 before the company's September 2018 IPO, which priced at $15 a share. The shares have since sunk by more than a third to $9.62 on May 7.

All in all, Surveyor is still doing what Citadel does best, hedging its bets, but with more investor interest in biotech, the biggest gains may be harder to come by.

This story first appeared in our sister publication, Crain's Chicago Business.

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$30 billion hedge fund's next bet is on biopharmaceuticals - ModernHealthcare.com

The smid-cap biotech earnings deluge hit Wall Street in the week ended May 9, positively impacting the stocks in the sector. The iShares NASDAQ Biotechnology Index (NASDAQ: IBB) gained about 6% for the week.

Large-cap pharma names AstraZeneca plc (NYSE: AZN) and Novartis AG (NYSE: NVS) received FDA nods for their heart failure and lung cancer therapies, respectively. After receiving emergency use authorization from the FDA in the U.S., Gilead Sciences, Inc.'s (NASDAQ: GILD) remdesivir obtained full regulatory approval in Japan.

Moderna Inc (NASDAQ: MRNA) received the OK toproceedwith the Phase 2 trial of its mRNA coronavirus vaccine candidate mRNA-1273.

The following are the key events and catalysts that biotech investors need to watch in the coming week.

Clovis Oncology Inc (NASDAQ: CLVS) awaits the FDA nod for an expanded indication for its cancer therapy Rubraca. The sNDA seeks approval of Rubraca as a monotherapy treatment for patients with BRCA1/2-mutant recurrent, metastatic castrate-resistant prostate cancer.

Abeona Therapeutics Inc (NASDAQ: ABEO) is due to present updated interim results from the Transpher A and Transpher B studies, Phase 1/2 trials of ABO-102 and ABO-101, respectively, in mucopolysaccharidosis type IIIA, aka as Sanfilippo syndrome type.

Rocket Pharmaceuticals Inc (NASDAQ: RCKT will present updated data from Phase 1/2 FANCOLEN-I study, which is evaluating the safety and efficacy of infusion of autologous CD34 + cells transduced with a lentiviral vector carrying the FANCA gene in patients with Fanconi anemia subtype A, and updates from the Phase 1 LAD-I study that is evaluating its investigational gene therapy RP-L201 to treat severe Leukocyte Adhesion Deficiency-I.

Avrobio Inc (NASDAQ: AVRO) is scheduled to make an oral presentation on new data from the Phase 2 trial of AVR-RD-01 for Fabry (Wednesday). The company will also make an oral presentation of new data from the collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04 in cystinosis. Another oral presentation on new data from a preclinical research program for a gene therapy for Pompe disease is also scheduled for Wednesday.

Ultragenyx Pharmaceutical Inc (NASDAQ: RARE) is due to present updated data from the first three cohorts of a Phase 1/2 study of DTX301 in treating ornithine transcarbamylase deficiency (Wednesday). The company will also present updated data from the confirmatory cohort from a Phase 1/2 study of DTX401 in glycogen storage disease Type 1a (Friday).

Pfizer Inc. (NYSE: PFE) will present Phase 1b data for PF-06939926 in Duchenne muscular dystrophy on Friday.

Krystal Biotech Inc (NASDAQ: KRYS) will present a poster on KB407, an HSV-1 based gene therapy vector, for the treatment of cystic fibrosis.

See Also: Gilead Works To 'Maximize Global Supply' Of Coronavirus Candidate Remdesivir Amid Threat Of Patent Loss

Genocea Biosciences Inc (NASDAQ: GNCA) will host a KOL symposium with a live Q&A for analysts and investors to reflect on the progress of the T cell therapy landscape and provide an in-depth profile of GEN-011 Genocea's neoantigen cell therapy. (Tuesday)

Krystal Biotech is due to present at the SID meeting withresults froma Phase 1/2 study of in vivo gene therapy KB105 for treating autosomal recessive congenital ichthyosis as well as results of a Phase 1/2 trial that is evaluating in vivo correction of dystrophic epidermolysis bullosa by direct cutaneous COL7A1 gene replacement.

Caladrius Biosciences Inc (NASDAQ: CLBS) will present at the SCAI meeting Thursday with full data from the ESCaPE-CMD study of CLBS16 for the treatment of coronary microvascular dysfunction.

Constellation Pharmaceuticals Inc (NASDAQ: CNST) said abstracts of a presentation due at the June 11-14 European Hematology Association meeting will be made available Thursday. The abstract pertains to an interim update from the MANIFEST Phase 2 study that is evaluating CPI-0610 in myelofibrosis.

Auris Medical Holding Ltd (NASDAQ: EARS) is due to release top-line data in early May from the Phase 1b trial that is evaluating AM-201 in healthy volunteers. AM-201 is the company's investigational drug for the prevention of antipsychotic-induced weight gain and somnolence.

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ORIC Pharmaceuticals Inc (NASDAQ: ORIC)

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The Week Ahead In Biotech: Virtual Conference Presentations, Clovis PDUFA Date In The Spotlight - Benzinga