Rocket Pharmaceuticals
Rocket Pharma Starts Rolling BLA Submission for Fanconi Anemia Gene Therapy
RP-L102, which the firm designed to treat a form of disease caused by FANCA mutations, is already under review with the European Medicines Agency.
Rocket Pharma Completes Enrollment of Pivotal Trial Assessing Danon Disease Gene Therapy
The company said the single-arm multicenter Phase II trial may support accelerated approval for the RP-A501 gene therapy in the US.
FDA Delays Decision on Rocket Pharma's Immunodeficiency Gene Therapy for Second Time
In a complete response letter, the FDA said it is seeking additional CMC information on the leukocyte adhesion deficiency-I gene therapy Kresladi.
In Brief This Week: Sophia Genetics, Eisai, Biogen, Lifespin, Acibadem, Rocket Pharmaceuticals
News items for the week of May 27, 2024.
ASGCT24: Regenxbio, Rocket Pharmaceuticals, Orchard Therapeutics, and More
At the meeting, companies and academic centers presented early and follow-up data on investigational gene therapies for nMPS II, PKD, MLD, and more.