Cell and Gene Therapy
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With an advisory committee meeting slated for Friday, the regulator has posted briefing documents in which it has raised concerns about early deaths in patients treated with Bristol Myers Squibb’s Abecma and Johnson & Johnson’s Carvykti.
Allogene Therapeutics and Arbor Biotechnologies will use their allogeneic CAR T and next-generation gene-editing platforms to develop novel off-the-shelf CAR-T therapies for autoimmune diseases.
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.
BMS and J&J will meet with the Oncologic Drugs Advisory Committee Friday to discuss their CAR-T therapies Abecma and Carvykti as the companies seek their approval as earlier lines of treatment.
The FDA’s busy week ahead involves three decision dates for potential industry firsts and a highly anticipated advisory committee meeting for two CAR-T therapies.
Separate challenges exist for companies developing gene therapies for rare and common cardiovascular conditions, experts told BioSpace.
Formerly known as Ryne Bio, Kenai Therapeutics emerged on Thursday with backing from several groups and has a cell therapy candidate going after Parkinson’s disease.
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.
Cell and gene therapy professionals gathered in Miami last month to discuss new manufacturing approaches for these up-and-coming treatments.
Johnson & Johnson and Legend Biotech got a positive opinion from a European Medicines Agency panel for earlier lines of treatment, as they ready for a March FDA advisory committee meeting.