Developing Gene Therapies for Rare Diseases

Video

The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s lead programs and future plans.

“All 7 patients have shown reconstitution of a protein called CD18. This protein is important in LAD-1 because the absence of it prevents neutrophils from fighting infection... therefore these patients have recurrent pneumonia, fungal infections, and hospitalizations and they unfortunately pass away early.”

Rocket Pharma is developing gene therapies for rare, monogenic diseases with the use of both an ex-vivo lentiviral platform and in-vivo adenoviral platform.

Rocket’s lead programs include the lentiviral therapies RP-L102 (NCT04248439) for the treatment of Fanconi anemia and RP-L201 (NCT03812263) for the treatment of leukocyte adhesion deficiency-I (LAD-1), which are both being evaluated in phase 2 studies. They are also developing the adenoviral therapy RP-A501 for the treatment of Danon disease, which is being evaluated in a phase 1 study (NCT03882437).

Rocket recently presented positive data at the 28th Annual Congress of the European Society of Gene & Cell Therapy that demonstrated RP-L201's preliminary efficacy in patients with LAD-1. All 7 patients demonstrated durable CD18 expression which exceeded the threshold for survival into adulthood and reversal of the severe LAD-1 phenotype. Since treatment, no patients have had LAD-I-related infections requiring hospitalization.

GeneTherapyLive spoke with Gaurav Shah, MD, cofounder and chief executive officer, Rocket Pharmaceuticals, to learn more about the company’s programs in Fanconi anemia, LAD-1, and Danon disease. He also discussed the company’s future research and plans.

REFERENCE
Rocket Pharmaceuticals announces positive clinical data from RP-L201 trial for the treatment of LAD-I at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). News release. October 20, 2021. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-positive-clinical-data-rp-l201
Recent Videos
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
Jamie Jacobs, PhD, the program director of the center for psychiatric oncology & behavioral sciences at Mass General Cancer Center
Laura Aguilar MD, PhD, the chief medical officer of Diakonos Oncology
Sarah Hein, PhD, the chief executive officer and cofounder of March Biosciences
Brian Kim, MBA, the chief executive officer of Mission Bio
Related Content
© 2025 MJH Life Sciences

All rights reserved.