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 (8x10¹³ GC/kg) showed enhanced PKP2 protein expression, incl. 110% & 398% increase in 2 pts with low…
