Developing Gene Therapies for Rare Diseases


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.

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.
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