Rocket Pharma's Gene Therapy Shows 100% Overall Survival In Immune Deficiency Disorder

Rocket Pharmaceuticals Inc 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.