COLgenen

COLgenen is a Dutch biotechnology company that specializes in the development and commercialization of gene therapy products. Founded in 2011, the company is headquartered in Leiden, the Netherlands. COLgenen's primary focus is on the treatment of rare genetic diseases, particularly those affecting the eye and the central nervous system. The company's approach involves the use of adeno-associated viral vectors (AAVs) to deliver therapeutic genes to target tissues, aiming to correct genetic defects and restore normal function.

One of COLgenen's notable products is Luxturna, a gene therapy for the treatment of Leber congenital amaurosis type 2 (LCA2), a rare and severe form of vision loss. Luxturna was approved by the U.S. Food and Drug Administration (FDA) in 2017 and by the European Medicines Agency (EMA) in 2018. The therapy involves the injection of a single dose of AAV2 vector into the retina, which delivers a therapeutic gene to correct the genetic defect and restore vision.

COLgenen's research and development efforts are supported by a strong pipeline of investigational gene therapies, including treatments for other rare genetic eye diseases and conditions affecting the central nervous system. The company collaborates with academic institutions, research organizations, and other industry partners to advance its therapeutic programs and accelerate the development of new gene therapies.

COLgenen is committed to improving the lives of patients with rare genetic diseases through innovative gene therapy approaches. The company's mission is to deliver life-changing therapies that address unmet medical needs and enhance the quality of life for patients and their families.