Jun. 16 at 3:31 AM
$RGNX 2 patient death on SRPT ELEVIDYS should not negatively correlate to RGX-202. Rather, it strengthens RGX-202's superior safety profile for several reasons. 1) RGX-202 uses vector AAV8 vs SRPT's rh74 and PFE's AAV9. 2) Novartis has been globally selling >
$1 billion blockbuster drug ZOLGENSMA for SMA developed using RGNX's AAV8 vector. 3) RGX-202 has C-terminal domain and depleted CpG deletion, which benefit expression and immunogenicity. 4) RGNX's superior cGMP manufacturing consistency/purity > 80% allows less total vector load. #3 & #4 are from BoA's research report. I believe RGX-202 will become the best-in-class DMD gene therapy!