Gene Therapy
-
Meletios Verras via Getty Images
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 -
Image courtesy of Ultragenyx.
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 -
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 -
Nemes Laszlo via Getty Images
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 -
Seth Babin/BioPharma Dive
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 -
iStock via Getty Images
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 -
Nemes Laszlo via Getty Images
Emerging biotech
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 -
libre de droit via Getty Images
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 -
Permission granted by Bristol-Myers Squibb
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 -
Design Cells via Getty Images
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 -
Meletios Verras via Getty Images
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 -
Nemes Laszlo via Getty Images
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 -
metamorworks via Getty Images
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 -
Courtesy of Cure Ventures
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 -
ismagilov via Getty Images
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 -
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 -
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
