News Feature
Pioneering Gene
Therapy for Vision
GenSight Biologics is a clinical-stage biotechnology company advancing novel gene therapies to preserve and restore vision.
We translate scientific and technological advances in gene therapy into innovative therapeutic approaches, supported by clinical expertise, robust data, peer-reviewed publications and long-term investor backing.
2
Proprietary Technology Platforms
1
Phase 3 Product Candidate
1
Phase 1/2 Product Candidate
250+
Patients Treated
5 Years
of Patient-Level Clinical Data
30+
Scientific Publications
€250M+
Raised
Technology Platforms
Disease Focus
GenSight Biologics currently focuses on severe inherited neurodegenerative diseases of the eye that cause blindness and have limited treatment options.
Leber Hereditary Optic Neuropathy (LHON)
LHON is the world’s most prevalent mitochondrial disease and an important cause of inherited blindness in young adults. Resulting from inherited mitochondrial DNA mutations, LHON causes sudden, usually irreversible vision loss, profoundly impacting a patient’s autonomy and quality of life.
Retinitis Pigmentosa (RP)
RP is the world’s most prevalent inherited retinal disease and a leading cause of visual disability and blindness, often beginning in childhood or adolescence. Resulting from diverse inherited genetic mutations, RP causes progressive, irreversible vision loss, profoundly impacting a patient’s autonomy and quality of life.
