Gene Therapy
-
Regeneron gene therapy improves hearing in two children
The results add to an early but growing body of evidence suggesting gene therapy may improve outcomes in young kids with a genetic form of deafness.
By Kristin Jensen • Updated May 8, 2024 -
Patient dies in Pfizer study of Duchenne gene therapy
Pfizer said the patient, a young boy who was treated earlier last year, died suddenly from cardiac arrest. The company is working with trial researchers to investigate further.
By Ben Fidler • Updated May 8, 2024 -
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 -
Bluebird, Vertex prep for first commercial use of sickle cell gene therapies
One patient has started the treatment process for Bluebird's Lyfgenia, while five others have done the same for Vertex's Casgevy.
By Ned Pagliarulo • Updated May 7, 2024 -
AstraZeneca ups stake in Cellectis in latest cell therapy bet
The British drugmaker, which has made genetic medicine a larger priority of late, added $140 million to a deal to develop up to 10 gene and cell therapies.
By Jonathan Gardner • May 6, 2024 -
Moderna ends gene editing alliance with Metagenomi
Metagenomi said the companies’ decision to part ways followed a “strategic prioritization” undertaken by Moderna, which has made several investments in gene editing research in recent years.
By Kristin Jensen • May 2, 2024 -
Astellas adds to ‘off-the-shelf’ cell therapy capabilities with Poseida deal
The partnership follows Astellas’ investment in the biotech last year and adds to its portfolio of donor-derived cellular medicines.
By Ben Fidler • May 1, 2024 -
Prime gets FDA green light to begin first trial test of ‘prime editing’
The company will soon start a Phase 1/2 study of its treatment for chronic granulomatous disease, a milestone for the search-and-replace gene editing technology.
By Ned Pagliarulo • April 29, 2024 -
Pfizer hemophilia gene therapy arrives in US to uncertain future
The Food and Drug Administration approval of Beqvez comes as other gene therapies for the bleeding condition struggle to gain traction.
By Ben Fidler • April 26, 2024 -
BioMarin drops drug programs in pipeline cull
The cuts of four pipeline prospects come as BioMarin resets its priorities following the slow launch of its hemophilia gene therapy Roctavian.
By Kristin Jensen • April 25, 2024 -
Gene editing
Regeneron expands in gene editing with Mammoth deal
“With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.
By Gwendolyn Wu • April 25, 2024 -
FDA rejects Abeona cell therapy, asks for more manufacturing data
The complete response letter for Abeona’s treatment is one of several manufacturing setbacks for cell and gene therapy developers in recent years.
By Ben Fidler • April 23, 2024 -
Bristol Myers taps startup to boost cell therapy production
A partnership with Cellares, worth up to $380 million, is meant to help Bristol Myers speed and scale manufacture of CAR-T treatments for cancer.
By Ned Pagliarulo • April 22, 2024 -
Ultragenyx says Angelman therapy is working, but safety questions remain
Treatment appeared to result in functional and cognitive gains in people with the neurological disorder. Three participants experienced lower extremity weakness, however.
By Ned Pagliarulo • April 15, 2024 -
Roche, Adaptimmune part ways on cell therapy research
The two companies have ended a collaboration that was struck in 2021 and focused on “off-the-shelf” cell therapies for cancer.
By Jacob Bell • April 12, 2024 -
Verve pauses base editing trial, shifts strategy after treatment side effect
Lab abnormalities in one study participant led the company to halt enrollment and prioritize a different version of its genetic medicine for heart disease.
By Ned Pagliarulo • April 2, 2024 -
Orchard sets out to sell world’s priciest gene therapy
Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25 million list price, the highest of any genetic medicine to come to market.
By Kristin Jensen • March 20, 2024 -
Capstan heats up ‘in vivo’ cell therapy chase with $175M fundraise
The high-profile startup, co-founded by CAR-T innovator Carl June, will use the funds to bring an autoimmune disease treatment into proof-of-concept studies.
By Ben Fidler • March 20, 2024 -
Orchard follows buyout with FDA approval of rare disease gene therapy
The U.S. clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.
By Kristin Jensen • March 19, 2024 -
Bristol Myers cell therapy wins first-of-its-kind approval
Sold as Breyanzi, the therapy is now cleared for use in certain adults with hard-to-treat forms of relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma.
By Jacob Bell • March 15, 2024 -
J&J, Novo support Asgard’s push to make ‘in vivo’ cell therapies
The Swedish biotech will use the investment to advance a personalized cancer therapy that reprograms tumor cells inside the body.
By Ben Fidler • March 14, 2024 -
FDA to focus on ‘early deaths’ in meeting on broader CAR-T use in myeloma
Briefing documents posted ahead of Friday’s advisory panel zeroed in on increased rates of early death in tests of cell therapies from Bristol Myers Squibb and Johnson & Johnson.
By Jonathan Gardner • March 13, 2024 -
Allogene taps Arbor in pursuit of ‘off-the-shelf’ CAR-T therapies for autoimmune disease
The partnership is designed to help the company stand out in what’s quickly become one of the industry’s hottest areas of drug research.
By Ben Fidler • March 12, 2024 -
Regenxbio data suggest ‘niche’ in Duchenne gene therapy
Study results showed early signs of promise in Duchenne patients who are currently ineligible to receive Sarepta’s Elevidys, triggering a stock surge.
By Kristin Jensen • March 6, 2024 -
Cure Ventures backs a cell therapy startup targeting Parkinson’s
The startup, Kenai Therapeutics, has raised $82 million and will be chaired by Jeff Jonas, a new Cure partner and former CEO of Sage Therapeutics.
By Jacob Bell • Feb. 29, 2024 -
BioMarin preaches patience amid slow sales for hemophilia gene therapy
The company earned only $3.5 million last year from its Roctavian treatment, far below the $50 million to $150 million range it had forecast eight months ago.
By Ben Fidler • Feb. 23, 2024