Gene therapy
Pfizer will go toe-to-toe with CSL Behring following the FDA’s Friday approval of its hemophilia B gene therapy Beqvez and will launch a warranty program based on the durability of response.
From gene-corrected cell therapies to a new CAR-T, the cell and gene therapy space looks to expand its reach into the market.
Nanoscope Therapeutics nabbed a victory on the changed primary endpoint of its Phase IIb vision loss gene therapy trial Tuesday, teeing the biotech up to file for FDA approval of the candidate this year.
With several recent approvals in the space and more on the horizon, BioSpace looks at some of the key decisions and their larger significance both for patients and science.
Brazil’s Ministry of Health and nonprofit Caring Cross announced a collaboration Tuesday aimed at local manufacturing of CAR-T cell and stem cell gene therapies at a much lower cost than Europe and the U.S.
Two days after winning FDA approval, Orchard Therapeutics on Wednesday provided its U.S. launch plans for metachromatic leukodystrophy gene therapy Lenmeldy, which has a wholesale acquisition cost of $4.25 million for the one-time treatment.
To help support the launch of Lyfgenia, bluebird bio on Monday entered into a five-year term loan deal with Hercules Capital that will extend the biotech’s cash runway through the first quarter of 2026.
Orchard Therapeutics on Monday secured the FDA’s first approval for an autologous gene therapy to treat the rare metabolic disease metachromatic leukodystrophy in children.
Asgard Therapeutics, a Swedish gene therapy biotech, has closed a $32 million Series A round with help from prominent pharma players as it prepares for a 2026 IND.
Vertex and CRISPR Therapeutics are setting up treatment centers for patients with beta thalassemia and sickle cell disease to compete with bluebird’s established infrastructure.
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