Rocket rises as it receives RMAT status for LAD-I gene therapy

Shares of US gene therapy company Rocket Pharmaceuticals (Nasdaq: RCKT) were up more than 9% at $53.14 in early afternoon trading on Tuesday, after the company revealed that the Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L201, its investigational gene therapy for the treatment of leukocyte adhesion deficiency-I (LAD-I).

The RMAT designation was granted based on encouraging preliminary safety and efficacy data from the ongoing Phase I/II clinical trial of RP-L201. Rocket gained the same status in 2018 for its RP-L102, the company’s lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi anemia (FA).

Additionally, patient enrollment has been fully completed for the Phase I/II trial. The study is being conducted at the University of California Los Angeles, University College London (UCL)/Great Ormond Street Children’s Hospital, and Hospital Infantil Universitario Niño Jesús.