Rocket Pharmaceuticals Soars 14.78% on FDA Decision Hopes

On August 20, 2025, Rocket Pharmaceuticals, Inc. (RCKT) surged 14.78% in pre-market trading, reflecting significant investor interest and optimism surrounding the company's recent developments.

Jefferies recently raised its price target for Rocket Pharmaceuticals, Inc. (RCKT) to $3.00, maintaining a 'Hold' rating. This adjustment comes on the heels of multiple potential catalysts expected through 2026, including the anticipated FDA decision on RP-A501, an AAV9-based gene therapy for Danon heart disease. This approval could unlock a market opportunity exceeding $500 million for RCKT.

Additionally, Jefferies highlighted the planned resubmission of a Biologics License Application for Kresladi, a lentiviral therapy for LAD-I, by the end of 2025. This potential opportunity is valued at over $100 million if monetized. The company's ongoing progress in gene therapies for inherited cardiomyopathies, including PKP2 and BAG3 programs, also holds the potential to drive further growth.

Despite these positive developments, Jefferies expressed concern over the company's rapid cash burn, which contributes to the 'Hold' rating. Rocket Pharmaceuticals, Inc. is based in the U.S. and focuses on developing gene therapies for rare and life-threatening diseases.