EurekAlert!

Researchers use patients' cells to test gene therapy for rare eye disease

Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood. A form of Leber congenital amaurosis, ...
Business Wire

VARIANT Receives European Orphan Drug Designation for its Gene Therapy Product Candidate, VAR002, for the Treatment of Both Leber Congenital Amaurosis and Cone-Rod dystrophy

PARIS--(BUSINESS WIRE)--Variant, a company dedicated to developing innovative treatments for inherited eye orphan diseases, announces the decision of the European Commission to grant Orphan Drug ...
MarketWatch

VARIANT Receives Positive Feedback from the European Medicines Agency (EMA) for its Innovative Gene Replacement Therapy, VAR002

PARIS, April 3, 2025 /PRNewswire/ -- Variant, a company dedicated to developing innovative treatments for orphan inherited eye diseases, and the Rare Ocular Diseases Center at the University of ...
News Medical

NEI scientists develop and test a promising gene therapy strategy for rare eye disease

Scientists at the National Eye Institute (NEI) have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood. A form of Leber congenital amaurosis, ...
Morningstar

VARIANT Receives Positive Feedback from the European Medicines Agency (EMA) for its Innovative Gene Replacement Therapy, VAR002

This gene replacement therapy provides an unmutated copy of the human CRX gene to replace the defective gene, inducing the expression of a functional CRX protein in the photoreceptors. VAR002 is ...