Gene Therapy


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    Popartic via Getty Images
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    UniQure stock surges on data for Huntington’s gene therapy

    The biotech’s stock rose more than 60% as fresh data appeared to alleviate some investor concerns about the therapy’s effectiveness and reliability.

    By July 9, 2024
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    Meletios Verras via Getty Images
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    Cartesian data add to progress, questions for CAR-T in autoimmune disease

    The company claimed Phase 2 study results prove the therapy’s promise treating myasthenia gravis. But shares lost more than a quarter of their value amid questions about the data.

    By July 2, 2024
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    iStock via Getty Images
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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
  • Employees of biotechnology company UniQure work in a laboratory.
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    Courtesy of UniQure
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    UniQure, with sale of plant, outsources Hemgenix manufacturing

    The gene therapy developer, which cut jobs in October, expects the sale of a Lexington, Massachusetts, facility to Genezen to lower its cash burn.

    By July 1, 2024
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    Marcin Klapczynski via Getty Images
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    With revised IPO pitch, Artiva touts ‘natural killer’ cell therapy for autoimmune disease

    Like many of its cell therapy peers, Artiva, which originally sought an IPO in 2021, has shifted its strategy to ride a wave of investor interest in autoimmune disease research.

    By July 1, 2024
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    luismmolina via Getty Images
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    Sponsored by Bio-Rad

    Are you meeting safety standards for residual host cell DNA?

    Ensure the safety of gene therapies with accurate HEK293 DNA measurement using Vericheck ddPCR kits.

    July 1, 2024
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    Sarah Silbiger via Getty Images
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    Rocket gene therapy rejected by FDA over manufacturing

    The complete response letter follows other recent manufacturing-related rejections, including one for a cell therapy from Abeona Therapeutics.

    By June 28, 2024
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    del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
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    First-of-its-kind Intellia data suggest CRISPR drug could be given more than once

    The findings provide early proof that multiple doses of a gene editing medicine can be safely administered with additive effects, overcoming a key constraint of the complex therapies.  

    By June 26, 2024
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    Olena_T via Getty Images
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    Emerging biotech

    Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine

    The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.

    By June 25, 2024
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    Permission granted by Accredo
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    Sponsored by Accredo by Evernorth

    The role of a specialty pharmacy in enhancing patient access to rare and gene therapies

    Four critical components in which the right specialty pharmacy partner can bring rare and gene therapy manufacturers the scale, expertise and capabilities needed to drive enhanced patient access.

    June 24, 2024
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    Courtesy of Sarepta
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    Sarepta Duchenne gene therapy wins broader use from FDA

    The approval makes Elevidys available to most Duchenne patients at least 4 years of age, despite mixed trial results that have led to skepticism about its effectiveness. 

    By Updated June 21, 2024
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    Syncona melds two gene therapy biotechs for better shot at new nervous system treatments

    The new company, Spur Therapeutics, is getting another $50 million from Syncona to support its broadened pipeline, which targets Parkinson’s disease as well as heart conditions.

    By June 17, 2024
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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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    Getty Images via Getty Images
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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
  • A photo of FDA CBER Director Peter Marks delivering remarks at a public workshop on March 3, 2020.
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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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    libre de droit via Getty Images
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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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    David Dee Delgado via Getty Images
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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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    Permission granted by Bluebird bio
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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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    Christopher Furlong via Getty Images
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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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    Maddie Meyer via Getty Images
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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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    JuSun via Getty Images
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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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    alvarez via Getty Images
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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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    Permission granted by Regeneron
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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