Advantages of Developing Platform-Agnostic Gene Therapies


The chief executive officer of Rocket Pharmaceuticals, Gaurav Shah, MD, discussed the company’s ex-vivo and in-vivo gene therapy platforms.

“We are platform agnostic in the sense that we develop our therapies based on high unmet need in the clinic, so we start with the end in mind. First, there are patients with rare monogenic diseases, many of whom have no treatment alternatives... There are thousands of these patients out there. Because of this philosophy, Rocket has... both an ex-vivo lentiviral platform and an in-vivo AAV platform... We want clear, clean mechanisms of action that will predict good responses with gene therapy and we want sizable enough indications to really benefit a large number of patients.”

Rocket Pharma uses both an ex-vivo lentiviral platform and in-vivo adenoviral platform to develop gene therapies for rare, monogenic diseases.

The company’s lead programs include RP-L102 (NCT04248439) for the treatment of Fanconi anemia and RP-L201 (NCT03812263) for the treatment of leukocyte adhesion deficiency-I (LAD-1), both lentiviral therapies being evaluated in phase 2 studies. 

Rocket also recently announced positive data from the phase 1 study (NCT03882437) of the adenoviral therapy RP-A501 for the treatment of Danon disease, which showed that the therapy is well-tolerated with sustained clinical benefit. The trial will continue evaluating the low-dose but not the high-dose therapy after a dose-limiting toxicity of thrombotic microangiopathy was observed.

GeneTherapyLive spoke with Gaurav Shah, MD, cofounder and chief executive officer, Rocket Pharmaceuticals, to learn more about the company’s platform-agnostic approach to creating gene therapies tailor-fit to different monogenic diseases. He discussed manufacturing and advantages of both ex-vivo and in-vivo approaches.

Rocket Pharmaceuticals announces positive updates from phase 1 clinical trial of RP-A501 in Danon disease. News release. Rocket Pharmaceuticals. November 15, 2021.
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