In December, Rocket Pharma presented results from a Phase I trial on RP-L102, a gene therapy for Fanconi anemia. Unlike earlier trials, these new results were from patients treated with a commercial-grade version of the product.

According to Gaurav Shah, MD, CEO of Rocket, the company quickly moved from a research to a commercial version of RP-L102 thanks to his experience working at Novartis.

“I’d say Novartis paved the way for the whole industry with CAR-T,” he said, in an exclusive GEN interview. “They did it first and we’re now building on the shoulders of giants.”

His experience developing CAR-T treatments for leukemia at Novartis made him aware, for gene therapies, that “it’s not easy to bridge from an academic process to a commercial-grade process.” He explains that many companies and drug developers believe that, provided the proof-of-concept is robust, clinical development will be easy. Moreover, regulatory agencies will immediately buy into the process.

According to Shah, just because the Novartis leukemia treatments had a high response rate, that didn’t guarantee they received an automatic approval from the regulators. Learning that robustness and reproducibility for a commercial process was different from an academic one, prevented Rocket from making missteps early on, he says.

Instead, their commercial product for Fanconi anemia uses a commercial vector and a commercial-grade cell processing site. “We’re fortunate to have done this early in the lifecycle of the product so we don’t have to make major changes late in the game,” he adds.

The commercial product uses a proprietary transduction enhancer to improve performance. Fanconi anemia is an autosomal recessive disease where patients inherit two mutated genes that cause bone marrow failure. The transduction enhancer ensures every bone marrow stem cell receives at least one copy of a corrected gene.

Gene therapy approach for Fanconi Anemia. [Rocket Pharma Rocket]

The company has also worked to maximize the cell dose and run its clinical trials with multiple operators. “We do the same thing multiple times, in the same way, with multiple people, so regulatory agencies can rest assured the product being delivered to the patient is not guesswork,” he says.

Rocket is currently enrolling patients for final-stage, registration-enabling trials of RP-L102.

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