SRPT - This Gene Therapy Player Duchenne Data Is Impressive Analyst Upgrades REGENXBIO | Benzinga
RBC Capital Markets upgraded REGENXBIO Inc (NASDAQ:RGNX), a company focused on developing gene therapies.
Recently, REGENXBIO reported additional interim safety and efficacy data in the Phase 1/2 AFFINITY DUCHENNE trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) ages 4 to 11.
In new data from the first patient, aged 12.1 years, who received RGX-202 at dose level 2, RGX-202 microdystrophin expression was measured to be 75.7% compared to control at three months.
A reduction in serum creatinine kinase (CK) levels of 77% was observed at ten weeks from baseline.
Elevated ...