An unmet need in advanced disease<\/p>\n
Current therapies for inherited retinal diseases (IRDs) and macular degeneration aim to slow progression and prevent further vision loss. According to Mon\u00e9s, none can treat advanced cases by restoring vision, and some IRD therapies also depend on the specific gene involved.<\/p>\n
The ideal therapy, Mon\u00e9s said, would restore vision in patients with advanced disease. It would also be gene-agnostic, meaning it could treat any condition regardless of which gene caused the outer retinal atrophy. He described these as the fundamentals of optogenetic therapy.<\/p>\n
Mechanism of action<\/p>\n
Sonpiretigene isteparvovec is a gene therapy that uses an adeno-associated virus serotype 2 (AAV2) vector to transfect retinal bipolar cells with an opsin. The opsin makes bipolar cells able to detect light and convert it into electrical signals, which bypasses the missing photoreceptors.<\/p>\n
Clinical and preclinical results<\/p>\n
In legally blind patients with retinitis pigmentosa (RP), Mon\u00e9s reported a clinically meaningful and significant gain of 0.3 logMAR, or approximately 3 lines, with a very safe profile. These results led to testing the therapy in patients with Stargardt disease. Vision in the macular phenotype improved by about 12 letters, and by 20 to 30 letters with a magnifier. Mon\u00e9s said the safety profile was again very good.<\/p>\n
The therapy is now also directed at geographic atrophy (GA). Investigators developed a new non-human primate model in which OCT-based laser injuries selectively targeted the outer retina, and tested the drug in it. The opsin was expressed in more than 70% of those cells. Mon\u00e9s described this as anatomical success, with additional physiological improvement on multifocal electroretinography (ERG).<\/p>\n
Next steps<\/p>\n
Based on these data, the plan is to start a large phase 2 trial in Stargardt disease and in GA. Mon\u00e9s said treatment would begin in legally blind patients. Once confidence in safety is established in that group, treatment would extend to patients with less severe vision loss.<\/p>\n
From slowing loss to restoring vision<\/p>\n
\u201cThe biggest key takeaway is that we may have a therapy that can restore vision,\u201d Mon\u00e9s said. He contrasted this with existing therapies, which aim only to limit vision loss. He also noted that patients with advanced disease have had no clinical trials or valid therapy for decades.<\/p>\n
\u201cImproving vision is something that we could not imagine a few years ago,\u201d he said, adding that \u201cit\u2019s not science fiction anymore.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"An unmet need in advanced disease Current therapies for inherited retinal diseases (IRDs) and macular degeneration aim to…\n","protected":false},"author":2,"featured_media":639166,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[10],"tags":[163,85,46],"class_list":["post-639165","post","type-post","status-publish","format-standard","has-post-thumbnail","category-health","tag-health","tag-il","tag-israel"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/639165","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/comments?post=639165"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/posts\/639165\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media\/639166"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/media?parent=639165"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/categories?post=639165"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/il\/wp-json\/wp\/v2\/tags?post=639165"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}