Several years ago, in 2010, when we discovered that our family members have retinal degeneration, we were informed that there was no cure for the condition and that blindness would eventually occur. Over the past few years, extensive studies have been conducted on the PRPF31 gene, leading to new discoveries concerning its connection to retinal degeneration.
Today, there are at least two patents for drugs designed to treat retinal degeneration resulting from mutations in the PRPF31 gene. One of these drugs is undergoing clinical trials in the United States. Additionally, there have been significant advancements in research on retinal degeneration in general, with ongoing studies and clinical trials for drugs aimed at restoring a degenerative retina. A mutation in the PRPF31 gene is one of the more common causes of retinitis pigmentosa. Therefore, we welcome studies that will allow testing of the effect of various methods on rehabilitating a degenerated retina caused by this gene. With this relative abundance of research, we assume that more research laboratories and groups are developing drugs for both PRPF31-specific retinal degeneration and retinal degeneration in general. Armed with this information, we have embarked on a quest to find a cure for retinal degeneration resulting from a mutation in PRPF31.
Follow the links below for more information.
Specific Patents and Trials for Retinal Degeneration due to PRPF31 Gene Mutation
- A medication designed to insert a normal PRPF31 gene into retinal cells and the Retinal Pigmented Epithelium (RPE) layer.
– Patent.
– Announcement of drug manufacturing by Biogen.
– Biogen’s drug development and production through Vigeneron - A medication designed to deliver normal RNA genetic material into retinal cells and the RPE layer.
– Information about the disease and the drug on PYC Therapeutics website.
– Link to an active drug trial on the U.S. government website.
Research and Experiments for General Retinal Degeneration
- Endogena’s development of a drug utilizing the patient’s retinal stem and progenitor cells to preserve and restore vision.
– About Endogena’s Phase 1-2a trial of EA-2353 for retinitis pigmentosa.
– Link to an active drug trial on the U.S. government website.