Shots:
- The US FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601 for the treatment of PKP2-arrhythmogenic cardiomyopathy
- Designation was supported by ongoing P-I trial, where all 3 adults treated with a single dose of RP-A601 (8×10¹³ GC/kg) showed enhanced PKP2 protein expression, incl. 110% & 398% increase in 2 pts with low baseline levels, & improved desmosomal integrity with relocalization of key structural proteins
- Trial also showed improvements or stability across key endpoints incl. right ventricular function, ventricular arrhythmias, & QoL, with 34–41 point increases in KCCQ-12 scores & NYHA class improvement from II to I; data was presented at ASGCT 2025
Ref: Rocket Pharmaceuticals | Image: Rocket Pharmaceuticals| Press Release
Related News:- Quince Therapeutics Completes Patient Enrolment in P-III (NEAT) Trial of eDSP for Ataxia-Telangiectasia
PharmaShots! Your go-to media platform for customized news ranging for multiple indications. For more information connect with us at connect@pharmashots.com
