Search This Blog

Monday, June 29, 2020

FDA nod on Rocket Pharma gene therapy for infantile malignant osteopetrosis

Rocket Pharmaceuticals (NASDAQ:RCKT) has received FDA clearance for its IND application for RP-L401, a lentiviral vector-based gene therapy for the treatment of Infantile Malignant Osteopetrosis, a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure.
The non-randomized, open-label Phase 1 clinical trial will enroll two pediatric patients, one month of age or older.
The trial is designed to assess safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status and endocrine abnormalities.

No comments:

Post a Comment

Note: Only a member of this blog may post a comment.