Competitive LandscapeDespite both assets demonstrating a reliably safer side effect profile, RGX-202, but not SGT-003, requires the use of prophylactic eculizumab, which carries black box warnings and requires patient observation, further complicating gene therapy administration.
Regulatory ConcernsRecent leadership changes at the FDA could lead to a near-term overhang on the stock until it becomes clear if the FDA still intends to use regulatory flexibility and accelerated approval pathways for rare diseases.