Rocket Pharma gene therapy shines in pediatric rare disease trial

May 20, 2022

Rocket Pharma has announced encouraging top-line safety and efficacy data from its phase 2 gene therapy trial in severe Leukocyte Adhesion Deficiency-I (LAD-I) — a rare, and typically fatal, genetic pediatric disorder with no cure.

Rocket's investigational gene therapy, RP-L201, consists of hematopoietic stem cells from the patient that have been genetically modified with a lentiviral vector to contain a functional copy of the ITGB2 gene.

During the 25th Annual Meeting of the American Society of Gene and Cell Therapy, Rocket revealed that 3-24 months after RP-L201 infusion, all nine trial patients sustained stable CD18 expression with no therapy-related serious adverse events. Furthermore, among all participants, the overall survival at one year is 100% — and patients are reporting a statistically significant reduction in all hospitalizations.

The NJ-based biotech is hopeful that RP-L201 has the potential to change the treatment paradigm for patients living with severe LAD-I — one of the most aggressive and highly fatal immunodeficiencies ever characterized. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon, according to Rocket.

Rocket's CEO, Gaurav Shah, says the results "point to the great possibilities lentiviral-based gene therapies can offer for patients with devastating diseases.”

Based on the trial results, the company is initiating discussions with health authorities on filing plans and anticipates regulatory filings in the first half of 2023.