Gene Therapy: Page 2
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Love Employee via Getty Images
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 -
Courtesy of Iovance Biotherapeutics
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 -
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 -
Courtesy of Sarepta
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 -
Mohammed Haneefa Nizamudeen via Getty Images
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 -
Dr_Microbe via Getty Images
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 -
jetcityimage via Getty Images
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 -
Yabusaka Design via Getty Images
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 -
Courtesy of Vertex Pharmaceuticals
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 -
Courtesy of Intellia Therapeutics
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 -
Grigorii Yalukov via Getty Images
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 -
Permission granted by Novartis
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 -
Mario Tama via Getty Images
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 -
Courtesy of UniQure
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 -
Meletios Verras via Getty Images
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 -
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 -
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 -
Meletios Verras via 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
Gene editingFirst 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
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