Gene Therapy: Page 5


  • A photo of Bluebird bio signage in a corporate lobby
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    Bluebird, playing catch-up, gets decision date for sickle cell gene therapy

    The FDA will issue a decision on lovo-cel by Dec. 20, roughly two weeks after a verdict is expected on a rival treatment from Vertex and CRISPR Therapeutics. 

    By Kristin Jensen • June 21, 2023
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    Permission granted by Landmark Bio
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    State of Play

    Cell and gene therapy manufacturing: the next generation of startups

    Joining larger CDMOs like Lonza and Catalent, a new group of companies aims to capitalize on persistent bottlenecks in producing complex genetic treatments.

    By June 20, 2023
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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
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    metamorworks via Getty Images
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    Sponsored by Center for Breakthrough Medicines

    A right-first-time approach to development

    Avoid costly delays, don't let development oversights derail your advanced therapy commercialization. Read the article.

    June 20, 2023
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    Sarah Silbiger via Getty Images
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    Ferring, with manufacturing update, takes a step towards first gene therapy sales

    The FDA has cleared Ferring’s planned scale-up process for its bladder cancer treatment Adstiladrin, which the company plans to make available in the U.S. later this year.

    By June 13, 2023
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    Christopher Furlong via Getty Images
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    AstraZeneca teams up with startup Quell, joining ‘Treg’ cell therapy hunt

    The pharma is the latest large drugmaker to show interest in the emerging technology, following similar deals by Bristol Myers and Regeneron.

    By June 9, 2023
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    Dr_Microbe via Getty Images
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    Using twin viruses, startup AAVantgarde aims to extend gene therapy’s reach

    The biotech has raised about $65 million to test two ways to deliver larger genes into the body, which could help gene therapy treat more diseases.

    By June 6, 2023
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    fpm via Getty Images
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    Sponsored by Bio-Rad

    Overcoming common pitfalls in lentiviral cell and gene therapies

    See how ddPCR technology can take your lentiviral cell and gene therapies to the next level.

    By Marwan Alsarraj • May 30, 2023
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    Alvarez via Getty Images
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    PTC lays off staff, fires CFO as neuromuscular drug study fails

    The biotech is giving up on early-stage gene therapy research as part of a restructuring meant to cut costs and redirect resources toward therapies most likely to succeed.

    By Kristin Jensen • May 24, 2023
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    Courtesy of Sarepta
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    FDA delays decision on Duchenne gene therapy, considers narrower approval

    The agency will extend its review of Sarepta’s treatment by one month as it weighs limiting an initial OK to children with Duchenne aged 4 to 5 years old.

    By Updated May 24, 2023
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    Sarah Silbiger/Getty Images via Getty Images
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    FDA approves Krystal gene therapy for rare wound disorder

    Cleared to treat dystrophic epidermolysis bullosa, Vyjuvek is the sixth gene therapy for an inherited disease to gain clearance in the U.S.

    By May 22, 2023
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    Novartis
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    Novartis buys rare disease gene therapy from Avrobio

    The pharma will pay nearly $90 million to acquire Avrobio’s treatment for cystinosis, an inherited condition caused by the toxic buildup of an amino acid.

    By May 22, 2023
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    Mustang pares research, sells manufacturing plant to save money

    The biotech joins a score of other cell and gene therapy developers that, on the heels of a historic market downturn, have cut costs.

    By May 18, 2023
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    Dr_Microbe via Getty Images
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    Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy

    Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.”

    By May 17, 2023
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    Lydia Polimeni, National Institutes of Health
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    Gene therapy consortium targets eight rare diseases for clinical trials

    Backed by nearly $100 million, the public-private consortium aims to create a standard development roadmap for gene therapies using AAV viral vectors.

    By Updated May 16, 2023
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    Courtesy of UniQure
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    UniQure sells royalty rights to hemophilia gene therapy

    The Dutch biotech sold a portion of royalties owed by CSL Behring on sales of Hemgenix, adding to a cash pile it has built since out-licensing the treatment.

    By May 15, 2023
  • A photograph of a Sarepta building in Boston, MA
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    Courtesy of Sarepta
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    FDA panel narrowly backs approval of Sarepta’s Duchenne gene therapy

    Shares in Sarepta rose by 25% Monday following a close advisory committee vote supporting accelerated approval of the biotech’s treatment, despite uncertainty around its benefit.

    By , Updated May 12, 2023
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    Courtesy of Sarepta
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    FDA staff skeptical of Sarepta’s Duchenne gene therapy, documents show

    Sarepta has not provided “unambiguous evidence” its treatment can help patients, agency scientists wrote in documents released ahead of a crucial Friday advisory committee meeting.

    By Updated May 10, 2023
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    Permission granted by Susan and Chris Finazzo
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    Deep Dive

    ‘The luckiest of the unlucky’: A Duchenne gene therapy brings hope to families — and tests the FDA

    The FDA in June approved a treatment that patient advocates and doctors believe to be a breakthrough for a deadly disease, but hasn’t yet proven that it works. 

    By , May 7, 2023
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    After delay, Bluebird submits sickle cell gene therapy for FDA approval

    Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

    By April 24, 2023
  • Gene therapy of the DNA cell, low-poly design of a human hand with a syringe and a spiral-shaped chromosome. Blue background.
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    Ilya/Stock.adobe.com

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    French biotech pulls EMA application for eye disease gene therapy

    With cash running low and a search for a buyer underway, GenSight wants advice from regulators on the type of data that would support an approval.

    By April 21, 2023
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    Function Oncology raises $28M to use CRISPR to tailor cancer diagnosis and treatment

    The startup says its genomics platform can improve on conventional sequencing by uncovering drug target vulnerabilities directly in tumor samples.

    By Nick Paul Taylor • April 13, 2023
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    Copyright © 2022 Miltenyi Biotec B.V. & Co. KG. All rights reserved.

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    Century Therapeutics changes leadership as CEO steps down

    The resignation of top executive Lalo Flores comes three months after a restructuring, marking the latest change of direction for the once high-flying cell therapy startup.  

    By April 12, 2023
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    Dr_Microbe via Getty Images
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    A new biotech wants to ease a bottleneck in cell and gene therapy production

    Founded by University of Pennsylvania researchers, VintaBio will manufacture the viral vectors that are essential in cell and gene therapy production, but which have been in shorter supply as more companies entered the space.

    By April 11, 2023
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    GSK outlines deal to send cell therapies back to Adaptimmune

    The pharma, which recently retreated from cell therapy research, will pay Adaptimmune about $37 million as part of an agreement to return rights to two cancer treatments.

    By April 11, 2023
  • A photo of laboratory equipment at Ginkgo Bioworks
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    Courtesy of Ginkgo Bioworks
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    Ginkgo grows its gene therapy offerings with StrideBio deal

    The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.

    By April 5, 2023