"Researchers at the National Eye Institute (NEI) have determined how certain short protein fragments, called peptides, can protect neuronal cells found in the light-sensing retina layer at the back of the eye. The peptides might someday be used to treat degenerative retinal diseases."
“We hope to leverage what we learned about RPE regeneration in zebrafish and use that information to develop new therapies in the human eye,” said Gross.
"Spark’s Luxturna made waves in 2017 when it was approved to treat inherited retinal degradation by replacing the mutated RPE65 gene with a normal copy. But instead of replacing a faulty gene to regain function, what if scientists could reprogram whole cells in vivo?
"On Tuesday, a UK-based startup reeled in $17 million to pursue just that."
Nanoscope Therapeutics Inc., a clinical-stage biotechnology company that is developing gene therapies for the treatment of retinal diseases, today announced that vision improvements for all evaluated advanced retinitis pigmentosa (RP) patients persisted through one year following a single intravitreal injection in a Phase 1/2a clinical study with MCO.
“We expect to begin the first randomized, placebo-controlled, double-masked Phase 2b multi-center optogenetic trial in the US this summer to further validate our gene therapy’s ability to improve clinically meaningful vision in RP patients. If successful, it will be the first-ever restorative drug for millions of RP patients worldwide,” said Nanoscope CEO Sulagna Bhattacharya.
"Recent advances in viral vector engineering, as well as an increased understanding of the cellular and molecular mechanism of retinal diseases, have led to the development of novel gene therapy approaches. Furthermore, ease of accessibility and ocular immune privilege makes the retina an ideal target for gene therapies. In this study, the nuclear hormone receptor gene Nr2e3 was evaluated for efficacy as broad-spectrum therapy to attenuate early to intermediate stages of retinal degeneration in five unique mouse models of retinitis pigmentosa (RP)."
"The case marks the first time sight has been partially restored using optogenetics, a type of biological research aimed at controlling nerve cells via light."
"But there are hundreds of different mutations linked to vision loss, and a universal cure for blindness remains distant. Now, a new start-up plans to disrupt the ocular gene therapy sector with a therapy that would work for many forms of vision loss, whether inherited or acquired during life.The company, Vedere Bio II, launched with $77 million in series A financing to develop what it calls a “mutation agnostic optogenetics technology” to restore vision."
"Endogena Therapeutics Inc., which discovers and develops endogenous regenerative medicines, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for its EA-2353 ophthalmic suspension targeting retinitis pigmentosa, a rare condition that causes slow and progressive loss of vision."
"Mark Pennesi, MD/PhD, the Kenneth C. Swan Associate Professor of Ophthalmology at the Casey Eye Institute in Portland (OR), USA, who will lead the phase I/IIa studies, said: 'I’m eager to begin clinical proof-of-concept studies to establish whether Endogena’s approach could offer a new treatment paradigm in retinitis pigmentosa. This novel method of tissue repair holds great promise for patients with this devastating degenerative eye condition.'"
"This is the very first time that anyone's ever actually tried to do gene-editing from inside the body," said Dr. Lisa Michaels, chief medical officer at the company sponsoring the study, Editas Medicine of Cambridge, Mass. "We're actually delivering the gene-editing apparatus to the part of the body where the disease takes place in order to correct it."
The gene-agnostic approach shows promise for people with late-stage RP and related diseases.