Rocket Pharma says first three patients dosed with RP-A501 in modified Phase 2 Danon trial show favorable safety profile

• Rocket Pharmaceuticals reported a positive early safety update from the first three patients treated in its pivotal Phase 2 RP-A501 trial for Danon disease.
• Results have already been observed, showing treatment was well tolerated with no major safety signals reported in these initial patients.
• The company is engaging with the FDA on a path to resume broader dosing and complete the 12-patient, single-arm pivotal study.
• A regulatory pathway update is expected in the second half of 2026, with a broader program update also slated for the second half of 2026.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Rocket Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 202608030700BIZWIRE_USPR_____20260803_BW745342) on August 03, 2026, and is solely responsible for the information contained therein.