Gene Therapy: Page 6


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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
  • Employees of biotechnology company UniQure work in a laboratory.
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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
  • Colorful DNA double helix. Explore the Trendline
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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
  • 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
  • Two young boys are seen posing for a photograph with a snake.
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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
  • 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
  • A 3D illustration of an adeno-associated virus.
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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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    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
  • 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
  • A 3D illustration showing swollen neurons with lamellar inclusions due to accumulation of gangliosides in lysosomes.
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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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    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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    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
  • A 3D rendering of the CRISPR-cas9 gene editing system.
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    Meletios Verras via Getty Images
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    Vertex pays CRISPR to use its gene editing tech for diabetes drugs

    Vertex will license CRISPR technology to develop insulin-producing islet cells that are more resistant to immune rejection, adding to other efforts directed at Type 1 diabetes.

    By March 27, 2023
  • A view of Moderna headquarters on May 08, 2020 in Cambridge, Massachusetts.
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    Maddie Meyer via Getty Images
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    Moderna inks second genetic medicine deal in a month

    The biotech plans to use Generation Bio’s nonviral delivery technology to get nucleic acid-based therapies to immune cells and into the liver.

    By March 23, 2023
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    Justin Sullivan via Getty Images
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    Gilead’s Yescarta extends survival in lymphoma study

    The results, the first for a CAR-T therapy, are further evidence supporting the use of the complex therapies earlier in a patient’s disease course. 

    By March 21, 2023
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    LaylaBird via Getty Images
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    Sponsored by MilliporeSigma

    Addressing manufacturing challenges in gene therapy development

    As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

    By Kate Silver • March 20, 2023
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    Evgenii Kovalev via Getty Images
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    Switch Therapeutics launches with $52M to capitalize on RNA medicine ‘era’

    The company’s name comes from its goal of developing drugs that only “switch” on in target cells, potentially enabling more precise therapies.

    By March 14, 2023
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    Grigorii Yalukov via Getty Images
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    BioMarin, as expected, sees delay to FDA review of hemophilia gene therapy

    The agency is considering three-year follow-up data in its review of Roctavian, pushing back the potential U.S. arrival of BioMarin’s drug once again.

    By Kristin Jensen • March 7, 2023
  • A photo of Alfred Sandrock, former chief medical officer and head of R&D at Biogen
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    Courtesy of Business Wire
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    Voyager licenses gene therapy tools to Novartis

    The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.

    By March 6, 2023
  • An Intellia Therapeutics lab
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    Courtesy of Intellia Therapeutics
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    FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

    The announcement, which follows regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst.

    By March 2, 2023