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.

https://seekingalpha.com/news/3586846-fda-signs-off-on-rocket-pharmas-gene-therapy-for-infantile-malignant-osteopetrosis