BIIB112

Unassigned

New Medicines

X-linked retinitis pigmentosa (XLRP)

Information

Advanced therapy medicinal product (ATMP)
Biogen
Biogen

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase II Clinical Trials

Category

XLRP, caused by RPGR gene mutation, leads to poor rod and cone function, retinal degeneration and blindness. A single injection of AAV-RPGR gene therapy delivers correct gene into the retina to slow retinal degeneration and preserve visual function.[1,2]
The prevalence of RP is estimated at ~1 in 5,000 individuals. XLRP accounts for 6-10% of cases [3,4]. This means ~ 1000 people in England are likely to affected at on time but not all will be eligible for this.
X-linked retinitis pigmentosa (XLRP)
Subretinal injection
Parenteral