Gene Therapy


  • An illustration of an adeno-associated virus.
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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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    Mario Tama 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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    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
  • A photograph of a Sarepta building in Boston, MA
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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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    Maximusnd via Getty Images
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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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    Emerging biotech

    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
  • A photograph of a Sarepta building in Boston, MA
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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

    FDA advisers voted 8-6 in support of the agency approving a treatment that patient advocates and doctors believe to be a breakthrough for a deadly disease.

    By , May 7, 2023
  • A photo of Bluebird bio signage in a corporate lobby
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    Permission granted by Bluebird bio
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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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    libre de droit via Getty Images
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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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    Emerging biotech

    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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    Warren Little via Getty Images
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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
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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
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    Dr Microbe via Getty Images
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    Freeline cuts more jobs and halts Fabry gene therapy work

    The U.K. biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.

    By April 4, 2023
  • An illustration of CRISPR-cas9 gene editing
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    del Aguila III, Ernesto. (2018). "CRISPR Cas9" [Illustration]. Retrieved from Flickr.
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    Gene editing

    Vertex, CRISPR finish US filing for gene editing drug approval

    The approval application is the first in the U.S. for a CRISPR-based medicine and puts the partners ahead of a rival therapy from Bluebird bio. 

    By April 3, 2023
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    BillionPhotos via Adobe Stock 

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    Sponsored by Advanced Clinical

    Changing cell & gene therapy landscape and implications on clinical trials

    Cell & gene therapy clinical research may help treat diseases and change how we approach healthcare.

    By Elizabeth Dugan, Director, Business Development • April 3, 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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    Emerging biotech

    Gene therapy startup Vedere to close two years after launch

    The startup, a successor to a biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.

    By Updated April 2, 2023
  • Electron microscope image of T regulatory cells interacting with antigen-presenting cells
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    NIAID. (2019). "T Regulatory Cells" [Microscope image]. Retrieved from Flickr.
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    Regeneron bets on ‘Treg’ cell therapy with Sonoma deal

    The biotech will work with Sonoma, a well-funded startup run by prominent immunologist Jeffrey Bluestone, to develop cell-based medicines for ulcerative colitis and Crohn’s disease.

    By March 28, 2023