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Enrollment has been completed in a phase 1/2 clinical study of a gene therapy product candidate for the treatment of X-linked retinoschisis, Applied Genetic Technologies Corporation announced in a press release.
The open-label, dose escalation trial will assess the safety and efficacy of the adeno-associated virus-based gene therapy through intravitreal administration in 27 patients with X-linked retinoschisis (XLRS) caused by mutations in the RS1 gene, the release said.
“There are currently no FDA-approved treatment options for XLRS, a leading cause of macular degeneration in young men,”
Biological therapy involves the use of living organisms, substances derived from living organisms, or laboratory-produced versions of such substances to treat disease. Some biological therapies for cancer use vaccines or bacteria to stimulate the body&rs...
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene th...
Bioinformatics is the application of computer software and hardware to the management of biological data to create useful information. Computers are used to gather, store, analyze and integrate biological and genetic information which can then be applied...