Autosomal dominant retinitis pigmentosa is known to be caused by...

Autosomal dominant retinitis pigmentosa is known to be caused by mutations in four pre-mRNA splicing factors.  One of these factors is PRPF31, which is believed to cause approximately 25% of all retinitis pigmentosa cases. PRPF31 is the splicing factor associated with RP11.

RP11 is PYC’s lead target indication.

So, it’s of interest that Biogen has just entered an exclusive licensing agreement with Harvard’s Massachusetts Eye and Ear to develop a gene therapy to help retinitis pigmentosa patients with mutations in PRPF31.

Mass Eye and Ear has published preclinical research demonstrating that their AAV-mediated gene therapy partially restored the structure and function of retinal pigment epithelium cells in mutated PRPF31 genes. Following its agreement with Mass Eye and Ear, Biogen will now take over clinical development.

Biogen has already demonstrated interest in both gene therapies and ocular diseases. In March last year, Biogen acquired Nightstar Therapeutics for US $877 million. Nightstar Therapeutics was is in early development of a treatment for X-linked retinitis pigmentosa.

https://masseyeandear.org/news/pres...nd-ear-enters-licensing-agreement-with-biogen

https://www.fiercebiotech.com/resea...gy-harvard-pact-focused-inherited-eye-disease

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6909184/

https://investors.biogen.com/news-r...nces-agreement-acquire-nightstar-therapeutics