Other Events ·
REGENXBIO Inc. announced that the FDA placed a clinical hold on its Investigational New Drug application for RGX-121…
RGNXREGENXBIO Inc.
REGENXBIO Inc. announced that the FDA placed a clinical hold on its Investigational New Drug application for RGX-121, an investigational gene therapy for Mucopolysaccharidosis type II (MPS II). The hold follows asymptomatic spine MRI findings in five participants of the CAMPSIITE study. REGENXBIO does not expect to resubmit the Biologics License Application for RGX-121 in the near term. The company stated that patients remain clinically stable and attributed potential findings to the disease itself rather than the therapy. This regulatory setback impacts the MPS II program, though the firm maintains its pipeline focus on Duchenne muscular dystrophy and wet AMD candidates. REGENXBIO Inc. is a biotechnology company developing AAV gene therapies for rare and retinal diseases.