Gene therapy
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.
Separate challenges exist for companies developing gene therapies for rare and common cardiovascular conditions, experts told BioSpace.
Sarepta Therapeutics on Wednesday called the launch of the gene therapy for Duchenne muscular dystrophy “exceptional” but the company does not expect to see significant growth in the first half of 2024 due to its currently limited patient pool.
The regulator has granted a priority review of the efficacy supplement for Sarepta Therapeutics’ gene therapy Elevidys with a target decision date of June 21, 2024.
Kelonia Therapeutics’ in vivo gene placement system is being tapped to help Astellas Pharma expand its portfolio of in vivo CAR-T cell therapies for cancer.
Citing the need for more time to review additional Chemistry, Manufacturing and Controls information, the FDA has extended its target action date for Rocket Pharmaceuticals’ investigational gene therapy by three months.
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