GenSight Biologics will participate to the 4th Annual Gene Therapy for Rare Disorders which will focus exclusively on overcoming the late-stage commercial challenges drug developers face when delivering gene therapies to market. The event will be virtual from February 22 to 25, 2021.
Magali Taiel, Chief Medical Officer, will give an oral presentation about our most advanced product called LUMEVOQ® (GS010; lenadogene nolparvovec), a gene therapy targeting Leber Hereditary Optic Neuropathy (LHON), a rare mitochondiral disease that lead to blindness in young adults.
Title: Lumevoq® Gene Therapy in Leber Hereditary Optic Neuropathy (LHON)
- LHON is a maternally inherited mitochondrial disease. It is a rare disease, with an incidence of 1500 new patients per year in Europe and in the US. The 11778 mutation on the ND4 mitochondrial gene is the mutation causing the most severe clinical form of LHON. Lumevoq® specifically targets this mutation.
- Lumevoq® is a recombinant adenovirus vector type 2, containing the wild type ND4 DNA. Lumevoq® is injected via Intravitreal Injection.
- GenSight Biologics have filed for EMA registration in September 2020, and then be moving to BLA for US registration in 2021.
Date & Time: Wednesday, February 24, 8.30 9.00am EST
Session: The View from The Cutting Edge: Gaining the Perspective of the Industry Leaders