Gene Therapy


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    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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    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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    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

    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

    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
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    Permission granted by Ed Shipman for Mass Eye and Ear
    Deep Dive // Gene therapy safety

    Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy

    Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it.

    By Nov. 15, 2021
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    Courtesy of Sanofi

    Eye-focused gene therapy startup gets $60M cash infusion from Sanofi

    Shortly after postponing a $135 million IPO, Gyroscope has found another way to back a treatment for a disease that's become a top target of multiple drugmakers.  

    By Nov. 8, 2021
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    Artur Plawgo via Getty Images

    Beam gets green light to begin first clinical test of base editing

    Alongside the milestone, the gene editing biotech also announced John Maraganore, who will soon step down as Alnylam's CEO, will join its board.

    By Nov. 8, 2021
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    Kena Betancur via Getty Images

    Blackstone continues biotech push with up to $250M cell therapy investment

    The investment in CAR-T maker Autolus Therapeutics adds to a string of biotech deals the private equity firm has made since 2018.

    By Nov. 8, 2021
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    Jeenah Moon via Getty Images

    Pfizer gene therapy research delayed by trial changes, safety questions

    Data readouts for Pfizer's hemophilia gene therapies are now expected later than previously forecast, while the company also works through modifying a trial of its Duchenne treatment.

    By Nov. 3, 2021
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    Mario Tama via Getty Images

    FDA extends review of J&J, Legend's cell therapy for multiple myeloma

    The three-month delay could give an advantage to Bristol Myers Squibb, which holds the first approval for a CAR-T therapy for the blood cancer.

    By , Nov. 2, 2021
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    Maddie Meyer via Getty Images

    Moderna, teaming with a startup, wades further into gene editing

    The biotech has identified gene editing as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions.

    By Nov. 2, 2021
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    Lydia Polimeni, National Institutes of Health

    NIH, FDA spearhead broad partnership to speed gene therapy research

    The two institutions will work with drugmakers and nonprofit groups on a wide-ranging initiative meant to make the complex treatments easier and less costly to produce. 

    By Kristin Jensen • Oct. 28, 2021