Gene Therapy: Page 9
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ASH/Todd Buchanan
At ASH, doctors acclaim new sickle cell gene therapies, but are cautious on details
Uptake of Casgevy and Lyfgenia may be slow despite their dramatic benefit, physicians said, citing complexities in treatment, manufacturing and reimbursement.
By Gwendolyn Wu , Ned Pagliarulo • Dec. 13, 2023 -
Courtesy of Nick Agro/American Society of Hematology
ASH23: Pharma branding, Editas’ high bar and clinical trial diversity
Editas had the tall task Monday of convincing ASH attendees its gene therapy for sickle cell disease could improve on Casgevy and Lyfgenia.
By Gwendolyn Wu , Ned Pagliarulo • Dec. 11, 2023 -
Explore the Trendline➔
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 -
Photo illustration: Shaun Lucas/Industry Dive; CRISPR Therapeutics; Gregor Fischer/DPA/Newscom
Deep Dive // Gene editing‘No tolerance for failure’: An oral history of the first CRISPR medicine
A new sickle cell disease therapy developed by CRISPR Therapeutics and Vertex Pharmaceuticals is now approved in the U.S. and U.K. This is the story of how it came to be.
By Ned Pagliarulo , Shaun Lucas • Dec. 10, 2023 -
ASH/Todd Buchanan 2023
Bluebird gene therapy, now approved for sickle cell, shows durable benefit in study update
Follow-up data continue to show Lyfgenia can address the pain crises people with sickle cell experience, although Bluebird’s therapy will be compared to Vertex and CRISPR Therapeutics’ Casgevy.
By Gwendolyn Wu • Dec. 9, 2023 -
Permission granted by CRISPR Therapeutics
Gene editingCRISPR therapy for sickle cell approved by FDA in gene editing milestone
Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy is the first drug based on the Nobel Prize-winning gene editing technology to reach market.
By Ned Pagliarulo • Updated Dec. 8, 2023 -
Permission granted by CRISPR Therapeutics
CRISPR eyes autoimmune disease in revamp of cell therapy plans
The gene editing biotech is shelving two of its most advanced cancer drugs, and joining a growing group of companies exploring cell-based medicines for inflammatory diseases like lupus.
By Ben Fidler • Dec. 5, 2023 -
Courtesy of BioMarin Pharmaceutical
BioMarin secures hemophilia gene therapy coverage in Germany
Drawn-out negotiations led to a lower price than initially expected, but analysts called the agreement a step forward for the biotech company.
By Kristin Jensen • Nov. 29, 2023 -
Michael M. Santiago via Getty Images
Freeline, after cuts, agrees to take-private deal with Syncona
The planned acquisition adds to a string of bargain buyouts and reverse mergers in the gene therapy field.
By Ned Pagliarulo • Nov. 22, 2023 -
Getty Images
World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia
Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.
By Ned Pagliarulo • Nov. 16, 2023 -
Courtesy of Forge Biologics
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 Gwendolyn Wu • Nov. 13, 2023 -
Seth Babin/BioPharma Dive
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 Ned Pagliarulo • Nov. 12, 2023 -
Janice Haney Carr/CDC/Sickle Cell Foundation of Georgia/AP
Deep Dive // Gene editingWhat 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 Ned Pagliarulo , Shaun Lucas • Nov. 8, 2023 -
Courtesy of BioMarin Pharmaceutical
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 -
Sarah Silbiger via Getty Images
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 Ned Pagliarulo • Oct. 31, 2023 -
Yujin Kim / MedTech Dive, original photo courtesy of U.S. Food and Drug Administration
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 Ned Pagliarulo , Jonathan Gardner , Gwendolyn Wu • Updated Oct. 31, 2023 -
Courtesy of Sarepta
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 Ned Pagliarulo • Updated Oct. 31, 2023 -
Sarah Silbiger/Getty Images via Getty Images
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 Ned Pagliarulo • Oct. 27, 2023 -
Joey Sirmons / BioPharma Dive/BioPharma Dive
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 Jacob Bell • Oct. 26, 2023 -
Permission granted by Intellia Therapeutics
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 Gwendolyn Wu • Oct. 18, 2023 -
Gregor Fischer/DPA/Newscom
Deep Dive // Gene editingA 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 Gwendolyn Wu , Shaun Lucas , Julia Himmel • Oct. 11, 2023 -
Courtesy of UniQure
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 Gwendolyn Wu • Oct. 5, 2023 -
Jonathan Gardner/BioPharma Dive
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 Ned Pagliarulo • Oct. 5, 2023 -
Permission granted by Intellia Therapeutics
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 Jacob Bell • Oct. 3, 2023 -
Sarah Silbiger via Getty Images
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 -
Jonathan Gardner/BioPharma Dive
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 Jonathan Gardner • Sept. 18, 2023
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