Gene Therapy


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    Jose Luis Calvo Martin, Jose Enrique Garcia-Maurino Muzquiz via Getty Images
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    Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision

    The failure of another trial adds to uncertainty around the benefits of the gene-based treatments, though industry analysts still expect Sarepta’s Elevidys’ to be unaffected.

    By June 13, 2024
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    Can robots break the cell therapy bottleneck?

    A partnership between cell therapy delivery specialist Portal Biotechnologies and precision robotics maker Multiply Labs could address manufacturing hurdles, the CEOs said.

    By Michael Gibney • May 29, 2024
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    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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    Ermath, Michael. (2020). "Individualized Therapies Workshop" [Photograph]. Retrieved from Flickr.
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    With Duchenne decision ahead, FDA’s Marks pushes for speedy gene therapy approvals

    But the head of the FDA’s CBER office didn’t tip where the agency stands on potentially broadening use of Sarepta’s Duchenne gene therapy Elevidys.

    By May 24, 2024
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    Courtesy of Millipore Sigma via Business Wire
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    Merck KGaA to buy gene therapy tools maker for $600M

    The German drugmaker is bolstering its MilliporeSigma business with a unit of Gamma Biosciences that specializes in so-called transfection reagents.

    By Kristin Jensen • May 23, 2024
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    Regeneron gene therapy improves hearing in two children

    The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness. 

    By Kristin Jensen • Updated May 8, 2024
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    Patient dies in Pfizer study of Duchenne gene therapy

    Pfizer said the patient, a young boy who was treated earlier last year, died suddenly from cardiac arrest. The company is working with trial researchers to investigate further.

    By Updated May 8, 2024
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    Bluebird, Vertex prep for first commercial use of sickle cell gene therapies

    One patient has started the treatment process for Bluebird's Lyfgenia, while five others have done the same for Vertex's Casgevy.

    By Updated May 7, 2024
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    AstraZeneca ups stake in Cellectis in latest cell therapy bet

    The British drugmaker, which has made genetic medicine a larger priority of late, added $140 million to a deal to develop up to 10 gene and cell therapies.

    By May 6, 2024
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    Moderna ends gene editing alliance with Metagenomi

    Metagenomi said the companies’ decision to part ways followed a “strategic prioritization” undertaken by Moderna, which has made several investments in gene editing research in recent years.

    By Kristin Jensen • May 2, 2024
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    Astellas adds to ‘off-the-shelf’ cell therapy capabilities with Poseida deal

    The partnership follows Astellas’ investment in the biotech last year and adds to its portfolio of donor-derived cellular medicines.

    By May 1, 2024
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    Prime gets FDA green light to begin first trial test of ‘prime editing’

    The company will soon start a Phase 1/2 study of its treatment for chronic granulomatous disease, a milestone for the search-and-replace gene editing technology.

    By April 29, 2024
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    Brillianata via Getty Images
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    Pfizer hemophilia gene therapy arrives in US to uncertain future

    The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition struggle to gain traction.

    By April 26, 2024
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    BioMarin drops drug programs in pipeline cull

    The cuts of four pipeline prospects come as BioMarin resets its priorities following the slow launch of its hemophilia gene therapy Roctavian.

    By Kristin Jensen • April 25, 2024
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    Gene editing

    Regeneron expands in gene editing with Mammoth deal

    “With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.

    By April 25, 2024
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    FDA rejects Abeona cell therapy, asks for more manufacturing data

    The complete response letter for Abeona’s treatment is one of several manufacturing setbacks for cell and gene therapy developers in recent years.

    By April 23, 2024
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    Bristol Myers taps startup to boost cell therapy production

    A partnership with Cellares, worth up to $380 million, is meant to help Bristol Myers speed and scale manufacture of CAR-T treatments for cancer.

    By April 22, 2024
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    Image courtesy of Ultragenyx.

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    Ultragenyx says Angelman therapy is working, but safety questions remain

    Treatment appeared to result in functional and cognitive gains in people with the neurological disorder. Three participants experienced lower extremity weakness, however.

    By April 15, 2024
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    Roche, Adaptimmune part ways on cell therapy research

    The two companies have ended a collaboration that was struck in 2021 and focused on “off-the-shelf” cell therapies for cancer.

    By April 12, 2024
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    Verve pauses base editing trial, shifts strategy after treatment side effect

    Lab abnormalities in one study participant led the company to halt enrollment and prioritize a different version of its genetic medicine for heart disease.

    By April 2, 2024
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    Orchard sets out to sell world’s priciest gene therapy

    Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25 million list price, the highest of any genetic medicine to come to market. 

    By Kristin Jensen • March 20, 2024
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    Capstan heats up ‘in vivo’ cell therapy chase with $175M fundraise

    The high-profile startup, co-founded by CAR-T innovator Carl June, will use the funds to bring an autoimmune disease treatment into proof-of-concept studies. 

    By March 20, 2024
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    libre de droit via Getty Images
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    Orchard follows buyout with FDA approval of rare disease gene therapy

    The U.S. clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.  

    By Kristin Jensen • March 19, 2024
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    Bristol Myers cell therapy wins first-of-its-kind approval

    Sold as Breyanzi, the therapy is now cleared for use in certain adults with hard-to-treat forms of relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma.

    By March 15, 2024
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    Design Cells via Getty Images
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    J&J, Novo support Asgard’s push to make ‘in vivo’ cell therapies

    The Swedish biotech will use the investment to advance a personalized cancer therapy that reprograms tumor cells inside the body.

    By March 14, 2024
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    FDA to focus on ‘early deaths’ in meeting on broader CAR-T use in myeloma

    Briefing documents posted ahead of Friday’s advisory panel zeroed in on increased rates of early death in tests of cell therapies from Bristol Myers Squibb and Johnson & Johnson.

    By March 13, 2024