Gene Therapy for Rare Diseases: Latest Updates


Kinnari Patel, PharmD, president and chief operating officer of Rocket Pharmaceuticals, discussed Rocket Pharma’s programs.

“We're really excited that rocket is focused on rare diseases, whether it's a bone marrow derived disease, or it's a heart derived disease, we're able to work with patient communities, collaborators, and scientists around Europe and US to bring these amazing, innovative therapies to patients."

While international Rare Disease Day has come and gone, groundbreaking work in diseases such as Danon disease, leukocyte adhesion deficiency type 1 (LAD-1), pyruvate kinase deficiency (PKD), and Fanconi anemia continue to push forward.

Rocket Pharma is developing gene therapies to help meet the unmet needs in these diseases. The company has programs in phase 1 trials for Danon disease (NCT03882437) and PKD (NCT04105166), a phase 1/2 program in LAD-1 (NCT03812263), and a phase 2 program in Fanconi anemia subtype A (NCT04069533). Rocket reported positive data in Danon disease, LAD-1, and Fanconi anemia in 2022.

CGTLive spoke with Kinnari Patel, PharmD, president and chief operating officer, Rocket Pharma, to learn more about the company’s programs in these rare diseases as well as upcoming pipeline milestones.

Rocket Pharmaceuticals reports fourth quarter and full year 2021 financial and operational results. News release. Rocket Pharmaceuticals. February 24, 2022.
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