GenSight Biologics, a biopharma company listed on Euronext (SIGHT) and dedicated to developing gene therapies for retinal neurodegenerative and central nervous system disorders, has announced that the French health authority ANSM has approved compassionate use of its GS010/LUMEVOQ gene therapy. This program allows patients with serious, rare, or disabling conditions to access treatments that have not yet received full marketing authorization when no suitable alternatives exist and there is an unmet medical need. Access under this scheme is limited to patients who meet specific eligibility criteria and applications must be submitted by healthcare professionals for individual patients. The therapy is evaluated for its potential benefit-risk profile before approval.
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Leber Hereditary Optic Neuropathy, or LHON, is a rare maternally inherited mitochondrial disorder that leads to rapid degeneration of retinal ganglion cells, resulting in severe, often irreversible vision loss that can progress to legal blindness. The ND4 mitochondrial mutation is the most common variant and is linked to the poorest visual outcomes among patients with LHON. The compassionate use authorization provides a pathway for patients with this mutation to potentially benefit from GS010/LUMEVOQ, addressing a critical unmet need in a population with very limited treatment options.
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This approval highlights GenSight Biologics’ commitment to advancing innovative therapies for patients facing devastating neuro-ophthalmic conditions and represents a key step toward broader access to gene therapy for rare eye diseases in France.
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