To identify the gene or genes responsible for developmental causes of blindness in the world, and to establish a Pediatric Ophthalmic Genetics and Research Program at the University of Wisconsin–Madison.
Clinical Trials
XOLARIS
To gain a better understanding of disease progression over time in male subjects with X-linked retinitis pigmentosa (XLRP).
CHM SURVEY TRIAL
Choroideremia (CHM) Health Outcomes
CATALINA
A Phase II Multicenter, Randomized, Double-Masked, Sham-Controlled Study of the Safety and Efficacy of Intravitreal Injections of NGM621 in Subjects with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD) (CATALINA)
Pro-EYS
Rate of Progression in EYS Related Retinal Degeneration (Pro-EYS).
AGTC A3
A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of AGTC 402, a Recombinant Adeno-associated Virus Vector Expressing CNGA3, in Patients with Congenital Achromatopsia Caused by Mutations in the CNGA3 Gene
AGTC B3
A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing CNGB3 (rAAV2tYF-PR1.7-hCNGB3) in Patients with Congenital Achromatopsia Caused by Mutations in the CNGB3 Gene
NIGHT
Natural History of the Progression of Choroideremia Study (NIGHT)
STAR
A Randomized, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy For Choroideremia Using An Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1) (STAR)