Gene Therapy
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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 -
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 -
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 -
NEJM paper fills in details on ‘remarkable’ CAR-T result in autoimmune disease
The full results spotlight cell therapy’s potential to treat lupus and other inflammatory conditions, but also the problems drugmakers must still solve.
By Ben Fidler • Feb. 21, 2024 -
Iovance, with approval of ‘TIL’ cell therapy, readies for complex launch
The biotech set a price of roughly $515,000 per patient for its therapy Amtagvi, the first to be approved based on a decades-old technique of using tumor-infiltrating lymphocytes.
By Ben Fidler • Feb. 20, 2024 -
FDA to review expanded use of Sarepta Duchenne gene therapy
The agency will decide by June 21 whether to broaden eligibility for Elevidys, and won’t convene a group of outside experts beforehand.
By Jonathan Gardner • Feb. 16, 2024 -
Intellia, ReCode partner on genetic medicines for cystic fibrosis
The partnership will use Intellia's "DNA writing” technology, and focus on people with the lung disease who have limited or no available treatments
By Jacob Bell • Feb. 15, 2024 -
New CMS pilot to test payment scheme for pricey sickle cell gene therapies
The agency is planning a pilot program across states to help sickle cell patients access treatments like the newly approved Casgevy and Lyfgenia.
By Ned Pagliarulo • Jan. 31, 2024 -
Lilly gene therapy finding puts focus on hearing loss treatment pipeline
An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.
By Ned Pagliarulo • Updated Jan. 25, 2024 -
Gene therapy biotech Jaguar spins out manufacturing company
Called Advanced Medicine Partners, the new company will offer specialized cell and gene therapy manufacturing services to biotech and pharma clients.
By Ned Pagliarulo • Updated Jan. 18, 2024 -
FDA widens approval of Vertex’s CRISPR medicine to treat beta thalassemia
The agency’s decision to expand use of Casgevy, which won a landmark OK for sickle cell disease in December, comes two months ahead of schedule.
By Ned Pagliarulo • Jan. 16, 2024 -
Intellia to lay off staff, cut some early research
The CRISPR specialist joins some of its genetic medicine peers in trimming staff, announcing an “organizational streamlining” to focus resources on prioritiy drug programs.
By Gwendolyn Wu • Jan. 4, 2024 -
Pfizer wins Canadian OK for hemophilia treatment, its first gene therapy
The hemophilia B therapy, which Pfizer will sell in Canada as Beqvez, is also under review in the U.S., where the FDA expects to make a decision by the second quarter.
By Jonathan Gardner • Jan. 3, 2024 -
Novartis deepens ties with Voyager via $100M gene therapy deal
The deal builds on an existing alliance and includes tools Novartis will use in gene therapies for Huntington’s disease and spinal muscular atrophy.
By Delilah Alvarado • Jan. 2, 2024 -
J&J bets bigger on MeiraGTx’s eye gene therapy
Ahead of a Phase 3 readout that could come next year, the pharma is paying $130 million in upfront and near-term cash for rights to the retinitis pigmentosa treatment that it didn’t already own.
By Jonathan Gardner • Dec. 21, 2023 -
Investors still aren’t sold on UniQure’s gene therapy for Huntington’s
The biotechnology company’s share price fell more than 10% Tuesday after the disclosure of more data from a small study.
By Jacob Bell • Dec. 19, 2023 -
Editas cashes in on CRISPR patent with Vertex deal
The deal, struck after Editas was awarded ownership of a key patent for the landmark gene editing technology, could be the first of many like it.
By Kristin Jensen • Dec. 14, 2023 -
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 -
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 -
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 -
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