Cell and Gene Therapy
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To improve its reviewers’ understanding of cell and gene therapy manufacturing, the agency has launched a program that will involve a tour of manufacturing facilities and daily workshops for its staffers.
Vertex has filed a complaint against the Department of Health and Human Services, seeking to make its fertility preservation program available to federally insured patients needing Casgevy treatment.
Lexeo Therapeutics’ investigational gene therapy reduces left ventricular volume and wall thickness in patients with Friedreich’s ataxia, according to a small study.
Interius BioTherapeutics has received approval from Australia’s Human Research Ethics Committee to begin the first-in-human trial of an investigational in vivo CAR-T therapy designed to treat B-cell malignancies.
Following a disappointing readout last year, uniQure on Tuesday posted promising Phase I/II data for its investigational gene therapy AMT-130 and nabbed the first-ever Regenerative Medicine Advanced Therapy designation from the FDA in Huntington’s disease.
The groundwork being done in 2024 is building the foundation for global collaboration in the future.
Johnson & Johnson and Legend Biotech’s Carvykti cell therapy significantly improved survival in patients with multiple myeloma when used in the second-line setting, the companies announced on Tuesday.
Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review.
One patient died of respiratory failure in a Phase I study of Lyell Immunopharma’s investigational CAR-T therapy. The company on Wednesday said it has not definitively linked the fatality to the treatment.
After winning expanded approval for its gene therapy for Duchenne muscular dystrophy, Sarepta’s leadership and analysts see a sizeable commercial opportunity on the horizon.