- Rocket Pharmaceuticals Inc (NASDAQ:RCKT) has announced topline safety and efficacy data from its Phase 2 trial of RP-L201 gene therapy in severe Leukocyte Adhesion Deficiency-I (LAD-I).
- Data were shared at the American Society of Gene and Cell Therapy.
- LAD-I is a disorder that causes the immune system to malfunction, resulting in immunodeficiency.
- Immunodeficiencies are conditions in which the immune system cannot protect the body effectively from foreign invaders such as viruses, bacteria, and fungi.
- Also Read: Rocket Pharma To Test Lower Dose Of Gene Therapy In Genetic Disorder Trial.
- All patients, aged five months to nine years, demonstrated sustained CD18 restoration and expression on more than 10% of neutrophils (20%-87%, median: 56%).
- At one year, the overall survival without allogeneic hematopoietic stem cell transplantation across the cohort is 100%.
- All patients demonstrated a statistically significant reduction in the rate of all-cause hospitalizations and severe infections relative to pre-treatment.
- The safety profile of RP-L201 has been highly favorable in all patients with no RP-L201-related serious adverse events to date.
- Price Action: RCKT shares are down 1.11% at $9.83 during the market session on the last check Thursday.
