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News & Events

See what’s new in the field of hereditary eye diseases

Blog managed by the DBGen team, with updated therapies, news and events involving hereditary eye diseases. We invite you to take part in this forum.

Novel dual-vector delivery system for Stargardt disease gene therapy

The American biotechnology company Applied Genetic Technologies Corporation (AGTC), specialized in the development of gene therapies for rare diseases, has just announced Stargardt disease as its new preclinical gene therapy program in ophthalmology. Stargardt disease characterized by a progressive loss of central vision affects one in 8,000-10,000 individuals and is the most common form of inherited macular degeneration. The first...

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We celebrate our first anniversary with a summary of the year

20/12/18 - DBGen, Genetic Diagnosis,

In January 2018, Dr. Roser Gonzàlez-Duarte and Dr. Gemma Marfany founded DBGen Ocular Genomics, a spin-off of the University of Barcelona. Their goal was to approach the genetic diagnosis of ocular hereditary diseases with the highest scientific standards. DBGen Ocular Genomics was born from the experience of more than 25 years of research on the genetic and molecular bases of...

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First CRISPR gene editing therapy for inherited visual disorders

On the 30th November EDIT-101, developed by Editas Medicine, received the approval from the Food and Drug Administration (FDA) to be the first in vivo CRISPR medicine administered to people anywhere in the world. EDIT-101. EDIT-101 is an experimental CRISPR genome editing medicine for the treatment of Leber Congenital Amaurosis (LCA, an inherited early-onset visual disorder that leads to infantile and juvenile...

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LUXTURNA- The first one-time gene therapy treatment for an inherited retinal blindness is approved for use in the US

The FDA (Food and Drug Administration de EEUU) has just approved the first gene therapy treatment for inherited retinal dystrophies that targets a rare disease caused by mutations in a specific gene (precision medicine). After successful clinical trials, FDA has granted LUXTURNA to Spark Therapeutics. With a cost around 850.000 $ (two eyes treatment), LUXTURNA is addressed to patients that...

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