Gene Therapy: Page 3


  • Forge Biologics' headquarters in Columbus, Ohio, covered in snow.
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    Courtesy of Forge Biologics
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    Ajinomoto spices up its biopharma business with gene therapy deal

    Forge Biologics operates a contract manufacturing business and develops its own gene therapies, which appears to have attracted the Japan-based food and biotechnology company.

    By Nov. 13, 2023
  • A photo of Sekar Kathiresan, CEO of Verve Therapeutics
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    Seth Babin/BioPharma Dive
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    Gene editing

    First look at Verve study data offers base editing ‘proof of principle’

    Early clinical trial results showed Verve’s therapy can substantially lower bad cholesterol. Still, investors sent the biotech’s shares down by 40% Monday.

    By Nov. 12, 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 sickle cell and normal blood cells are seen in red against a blue background in this colorized microscope image.
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    Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia/AP

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    Deep Dive // Gene editing

    What if a CRISPR cure isn’t such an easy choice?

    Casgevy can mute sickle cell disease’s most damaging symptoms. Yet treatment may not be as simple as its dramatic benefit makes it seem.

    By , Nov. 8, 2023
  • Workers gather inside BioMarin’s gene therapy manufacturing plant in Novato, California.
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    Courtesy of BioMarin Pharmaceutical
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    BioMarin to replace CEO Bienaimé with Genentech veteran

    The rare disease drug developer is turning to former Genentech head Alexander Hardy as the launch of its Roctavian gene therapy stumbles.

    By Kristin Jensen • Nov. 2, 2023
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    Sarah Silbiger via Getty Images
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    Vertex, CRISPR therapy for sickle cell passes FDA panel test

    The high-profile meeting focused on the theoretical risks of CRISPR gene editing, as both the FDA and its advisory committee appeared convinced by the efficacy of the companies’ exa-cel treatment.

    By Oct. 31, 2023
  • A play-by-play of the FDA’s meeting on Vertex, CRISPR’s sickle cell therapy

    Expert advisers dug deep on the risks of off-target gene editing and debated how best to set guidelines for the fast-growing field. Catch up on their full discussion here. 

    By , , Updated Oct. 31, 2023
  • A photograph of a Sarepta building in Boston, MA
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    Courtesy of Sarepta
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    Sarepta gene therapy for Duchenne misses main goal of key study

    Results from the EMBARK study, which were meant to confirm the approval of Sarepta's Elevidys, sent the company's shares down more than 40% Tuesday.

    By Updated Oct. 31, 2023
  • The headquarters of the FDA, which has recently issued a recall for Teleflex air filters used in hospital respirators.
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    Sarah Silbiger/Getty Images via Getty Images
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    FDA staff focus on ‘off-target’ risk in review of Vertex, CRISPR sickle cell therapy

    Documents released ahead of a Tuesday advisory meeting show agency scientists to be generally convinced by the treatment’s benefit, a sign analysts read as positive.

    By Oct. 27, 2023
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    Joey Sirmons / BioPharma Dive/BioPharma Dive
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    Vertex gearing up for launch as sickle cell therapy review advances

    Executives at the biotech say they’re trying to get ahead of the payer and production challenges that will face their gene editing treatment exa-cel, which is now under FDA review. 

    By Oct. 26, 2023
  • Intellia Therapeutics
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    Permission granted by Intellia Therapeutics
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    Intellia cleared by FDA to start Phase 3 test of gene editing treatment

    Developed with Regeneron, Intellia’s treatment is designed to inactivate a gene to treat an inherited disease called transthyretin amyloidosis.

    By Oct. 18, 2023
  • A close up a cell with a large screen in the background displaying a injection needle.
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    Gregor Fischer/DPA/Newscom

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    Deep Dive // Gene editing

    A decade later, biotech’s CRISPR revolution is still going strong

    Once the specialty of a few select drugmakers, CRISPR gene editing is now an essential technology for a growing group of biotechs, many led by former students of the field's pioneering scientists.

    By , , Oct. 11, 2023
  • Employees of biotechnology company UniQure work in a laboratory.
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    Courtesy of UniQure
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    UniQure lays off 20% of staff, cuts research to ‘significantly’ lower costs

    All told, the biotech said it will discontinue more than half of its research and technology projects, including a Parkinson’s disease treatment.

    By Oct. 5, 2023
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    Jonathan Gardner / BioPharma Dive
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    Orchard sells to Kyowa Kirin in gene therapy buyout

    Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.

    By Oct. 5, 2023
  • Intellia Therapeutics
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    Permission granted by Intellia Therapeutics
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    Regeneron, Intellia target neurological diseases in expanded gene editing deal

    The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.

    By Oct. 3, 2023
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    Sarah Silbiger via Getty Images
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    Taysha drops lead gene therapy following FDA feedback

    Additional studies proposed by the FDA would be too challenging, according to Taysha, which simultaneously announced that its partner Astellas has declined to pick up an option for the therapy, called TSHA-120.

    By Kristin Jensen • Sept. 20, 2023
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    Jonathan Gardner / BioPharma Dive
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    Orchard nears FDA decision on rare disease gene therapy

    Three years after gaining European approval, Libmeldy is now under U.S. review with a deadline set for March.

    By Sept. 18, 2023
  • An illustration of two lungs on a scientific background colored orange.
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    Mohammed Haneefa Nizamudeen via Getty Images
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    UK biotech AlveoGene launches with plans for inhaled gene therapy

    The company is working on a treatment of alpha-1 antitrypsin deficiency, which it claims it can deliver direct to lung cells via a nebulizer.

    By Kristin Jensen • Sept. 14, 2023
  • A  3D illustration of ribonucleic acid, or RNA, strands
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    Christoph Burgstedt via Getty Images
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    Wave readies clinical testing for first RNA editing therapy

    The biotech, one of several advancing RNA editing medicines, expects to obtain “proof-of-mechanism” data in patients with the inherited disorder alpha-1 antitrypsin deficiency by next year.

    By Kristin Jensen • Updated Sept. 5, 2023
  • Futuristic 3D cubes showing DNA base pairs and a double helix.
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    JuSun via Getty Images
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    Beam begins US testing of first-of-its-kind ‘base editing’ cancer drug

    The announcement marks progress for Beam after trial delays, and is the first time in the U.S. that a patient has received a base editing treatment.

    By Sept. 5, 2023
  • An illustration of immune system T cells attacking cancer cells.
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    Meletios Verras via Getty Images
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    Startup Cellares adds $255M as investors pour cash into cell therapy production

    Bristol Myers Squibb is among those backing the company, which claims the manufacturing capacity at its New Jersey plant can surpass that of conventional CDMO facilities. 

    By Kristin Jensen • Aug. 24, 2023
  • Scientist observing vial
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    Permission granted by MilliporeSigma
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    Sponsored by MilliporeSigma

    5 ways experience matters in gene therapy manufacturing

    By partnering with an accomplished CTDMO, gene therapy innovators can benefit from guidance and support.

    Aug. 21, 2023
  • Mergers and acquisitions
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    PeopleImages via Getty Images
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    Bruker to buy PhenomeX and its cell research tools for $108M

    The company said PhenomeX’s technology will help its customers speed development and manufacturing of cell and gene therapies.

    By Susan Kelly • Aug. 18, 2023
  • A 3D illustration of sickled cells in blood
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    Artur Plawgo via Getty Images
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    FDA won’t hold advisory meeting for Bluebird’s sickle cell gene therapy

    The decision stands in contrast to the agency’s plans for a would-be rival gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that is also under a regulatory review. 

    By Kristin Jensen • Aug. 16, 2023
  • An illustration of T cells attacking a cancer cell
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    Peddalanka Ramesh Babu via Getty Images
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    Precision sells lymphoma drug to Imugene in retreat from cell therapy research

    The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.

    By Aug. 16, 2023
  • Computer rendering of a virus
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    nopparit via Getty Images
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    HIV cell therapy startup Addimmune heads to Wall Street via blank-check merger

    The deal will help fund a cell-based treatment that completed Phase 1 testing last year and is designed to provide a “functional cure” for patients with HIV infections.

    By Aug. 10, 2023