Gene Therapy: Page 4
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Courtesy of Sarepta
Sarepta sells FDA fast pass earned from Duchenne gene therapy approval
The deal enables Sarepta to quickly cash in on the clearance of Elevidys, with $102 million in new funds to help bankroll its drug research.
By Jonathan Gardner • July 5, 2023 -
Courtesy of BioMarin Pharmaceutical
With FDA approval in hand, BioMarin lays out plan to sell $2.9M gene therapy
The agency granted a long-awaited clearance last Thursday, but unexpected aspects of the hemophilia treatment’s label had some investors worried about its commercial prospects.
By Jacob Bell • June 30, 2023 -
Explore the Trendline➔
iStock via Getty Images
TrendlineGene 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 -
Brillianata via Getty Images
BioMarin finally secures FDA approval of hemophilia gene therapy
After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.
By Jacob Bell • June 29, 2023 -
luismmolina via Getty Images
With new data, Nkarta battles doubts facing ‘off-the-shelf’ cell therapies
Though a newer regimen showed promise in patients with acute myeloid leukemia, earlier remissions haven’t held up as well as the company hoped.
By Ben Fidler • June 27, 2023 -
Grigorii Yalukov via Getty Images
Pfizer’s hemophilia B gene therapy inches closer to regulatory approval
The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.
By Delilah Alvarado • June 27, 2023 -
Courtesy of Sarepta
Sarepta prices Duchenne gene therapy at $3.2M
The cost makes Sarepta’s treatment among the most expensive medicines in the world. But company executives said they don’t expect significant insurer pushback.
By Ben Fidler • June 22, 2023 -
Courtesy of Sarepta
First Duchenne gene therapy approved by FDA for young children
The approval of Elevidys was a milestone for research into the progressive and deadly disease. But the subsequent failure of a confirmatory trial has raised questions for the FDA.
By Ben Fidler • Updated June 22, 2023 -
Courtesy of UniQure
UniQure stock tumbles on ‘confusing’ results for Huntington’s gene therapy
While patients appear to be doing better than historical data suggests, mixed results on other measures perplexed analysts and sent shares falling.
By Jacob Bell • June 21, 2023 -
Permission granted by Bluebird bio
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 -
Permission granted by Landmark Bio
State of PlayCell 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 Gwendolyn Wu • June 20, 2023 -
metamorworks via Getty Images
Sponsored by Center for Breakthrough MedicinesA 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 -
Sarah Silbiger via Getty Images
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 Delilah Alvarado • June 13, 2023 -
Christopher Furlong via Getty Images
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 Ben Fidler • June 9, 2023 -
Dr_Microbe via Getty Images
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 Ben Fidler • June 6, 2023 -
fpm via Getty Images
Sponsored by Bio-RadOvercoming 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 -
Alvarez via Getty Images
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 -
Courtesy of Sarepta
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 Ben Fidler • Updated May 24, 2023 -
Sarah Silbiger/Getty Images via Getty Images
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 Delilah Alvarado • May 22, 2023 -
Novartis
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 Ned Pagliarulo • May 22, 2023 -
Maximusnd via Getty Images
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 Jacob Bell • May 18, 2023 -
Dr_Microbe via Getty Images
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 Ned Pagliarulo • May 17, 2023 -
Lydia Polimeni, National Institutes of Health
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 Christopher Newman • Updated May 16, 2023 -
Courtesy of UniQure
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 Christopher Newman • May 15, 2023 -
Courtesy of Sarepta
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 Ned Pagliarulo , Ben Fidler • Updated May 12, 2023 -
Courtesy of Sarepta
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 Ben Fidler • Updated May 10, 2023
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