Gene Therapy: Page 2


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    Amicus Therapeutics

    Amicus' SPAC deal unravels, leading to layoffs and cost cuts

    The biotech is calling off plans to spin out its gene therapy business via a merger with a blank check company, citing unfavorable market conditions and a "challenging environment."

    By Kristin Jensen • Feb. 24, 2022
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    cgtoolbox via Getty Images

    Allogene, slipping in 'off-the-shelf' CAR-T race, prepares for key trials

    Allogene, once the clear leader in development of a more convenient option to personalized cancer cell therapies, now is competing with CRISPR Therapeutics and other fast-emerging rivals.

    By Feb. 24, 2022
  • Trendline

    Gene Therapy

    Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, researchers and drugmakers still face major challenges. 

    By BioPharma Dive staff
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    Permission granted by Spark Therapeutics

    Spark CEO Marrazzo, who led company to historic gene therapy approval, to step down

    Jeffrey Marrazzo has led Spark since its founding in 2013, through the historic FDA approval of Luxturna and the company's $4.3 billion buyout by Roche.

    By Feb. 23, 2022
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    DKosig via Getty Images

    BioMarin says gene therapy hold will endure as FDA questions cancer risk

    According to BioMarin, the regulator has requested additional data to assess the "theoretical" cancer risk to the treatment, information that will take several quarters to collect. 

    By Feb. 18, 2022
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    National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.

    Intellia follows CRISPR peers with deal to explore 'natural killer' cell therapy

    By partnering with ONK Therapeutics, Intellia joins CRISPR Therapeutics and Editas Medicine in striking deals that marry gene editing with a fast-emerging form of cancer immunotherapy.

    By Feb. 15, 2022
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    Solskin via Getty Images
    Sponsored by First Republic Bank

    Private equity powers digital healthcare revolution

    Read how private equity investment helps scale the push towards healthcare digitization.  

    By First Republic Bank • Feb. 7, 2022
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    Getty / Edited by BioPharma Dive

    In England, Orchard secures rare agreement on gene therapy coverage

    Gene therapy developers have struggled to secure reimbursement in Europe, making Orchard's deal with the National Health Service in England and Wales a notable accomplishment.

    By Feb. 4, 2022
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    Courtesy of Sarepta

    Sarepta reveals gene editing alliance with biotech exploring alternative delivery methods

    The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering gene editing tools into the body, dates back to December 2020, but has recently begun to advance.

    By Feb. 1, 2022
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    Sarah Silbiger via Getty Images

    Bluebird faces longer wait for FDA verdict on experimental gene therapies

    The FDA has extended by three months its review of Bluebird's treatments for beta thalassemia and a rare brain disorder, the latest delay in the company's lengthy road to market in the U.S.

    By Jan. 18, 2022
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    Danielle Ternes/BioPharma Dive

    5 questions facing gene therapy in 2022

    As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines. 

    By Jan. 13, 2022
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    Courtesy of Sarepta

    Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval

    The biotech will take new Phase 2 data to U.S. regulators, CEO Doug Ingram said Monday, though it still expects to need data from an ongoing late-stage trial to win approval. 

    By Jan. 10, 2022
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    cgtoolbox via Getty Images

    Allogene cleared by FDA to resume 'off the shelf' cancer cell therapy trials

    A "chromosomal abnormality" that led regulators to halt Allogene's trials was judged to be unrelated to its technology, a finding with important implications for the field of donor-derived cell therapies.

    By Jan. 10, 2022
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    ismagilov via Getty Images

    BioMarin plans return to FDA with updated data on hemophilia gene therapy

    Two-year results from a Phase 3 study of Roctavian show treatment prevented bleeding, potentially giving BioMarin the data it needs to resubmit an application for approval. 

    By Updated Jan. 10, 2022
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    Dan Kitwood via Getty Images
    CRISPR gene editing

    Pfizer expands into gene editing with Beam research deal

    Pfizer's work with mRNA vaccines led it to explore other applications of the technology, resulting in a multiyear partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases.

    By Jan. 10, 2022
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    Artur Plawgo via Getty Images

    Sanofi cuts ties with Sangamo, sharpening focus on 'off-the-shelf' cell therapy

    The French pharma cited a "change in strategic direction" for ending a long-running pact focused on genetic medicines for blood diseases, led by a sickle cell treatment in early testing.

    By Jan. 6, 2022
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    Novartis

    Novartis ups investment in gene therapy for the eye with $800M buyout

    The deal for Gyroscope Therapeutics, worth up to $1.5 billion, gives the Swiss pharma access to a treatment for a type of age-related blindness as well as new means of delivering drugs into the eye.

    By Dec. 22, 2021
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    Dr_Microbe via Getty Images

    Duchenne patient dies in Pfizer gene therapy study

    The tragic news follows changes Pfizer recently made to the design of another study testing the therapy due to side effects seen in some participants. 

    By Dec. 21, 2021
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    Artur Plawgo via Getty Images

    Bluebird hit with another hold for sickle cell gene therapy

    Study enrollment and treatment of sickle cell patients under 18 will be paused for safety reasons, another hurdle in a series of setbacks for the company.

    By Dec. 20, 2021
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    Spark Therapeutics

    Spark expands in Philadelphia, with new facility meant to be Roche's gene therapy hub

    The Luxturna developer, now owned by Roche, will invest $575 million to build a huge research and development center on Drexel University's campus.

    By Dec. 17, 2021
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    Jeenah Moon via Getty Images

    Pfizer opens gene therapy plant in $800M North Carolina expansion

    The new facility cost nearly $70 million to build, and is part of a major push by the pharma giant to become a leader in genetic medicine manufacturing.

    By Kristin Jensen • Dec. 16, 2021
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    ismagilov via Getty Images

    Rebounding from setbacks, Sanofi reveals promising data for hemophilia drug

    Fitusiran, which Sanofi licensed from Alnylam, could finally get to market after trial delays and safety concerns slowed its progress. But gene therapies and other new medicines might provide competition.

    By Dec. 14, 2021
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    ismagilov via Getty Images

    Fresh data show UniQure's hemophilia gene therapy appears to hold up

    The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.

    By Dec. 9, 2021
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    Getty / Edited by BioPharma Dive

    A high-profile gene therapy biotech takes aim at Huntington's

    Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's.

    By Dec. 8, 2021
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    Seth Babin/BioPharma Dive
    Deep Dive // CRISPR gene editing

    Heart attacks struck Sek Kathiresan's family. He's devoted his life to stopping them.

    After several family members had early heart attacks, Kathiresan vowed to understand why they happen. His research journey has changed medical practice and led to a new biotech startup, Verve Therapeutics, that seeks to prevent them.

    By Nov. 29, 2021
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    libre de droit via Getty Images

    Bluebird, after delays, gets speedy FDA review for beta thalassemia gene therapy

    The regulator will decide whether to approve Bluebird's treatment by next May, but its evaluation is beginning more than a year later than the biotech hoped.

    By Nov. 22, 2021