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Rocket Pharma Q&A: efficacy of gene therapy in Fanconi anaemia

Due to the need for chemotherapy and the risks caused by allogeneic stem cell transplants in Fanconi anaemia patients, Rocket Pharma has developed gene therapy candidate RP-L102, which doesn’t need conditioning and corrects the gene mutation underlying Fanconi. Rocket CEO Guarav Shah explains RP-L102’s mechanism of action, as well as how the company is working to improve its cell culturing methods and overcome continuing risks of head and neck cancer.


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