"The therapy can be administered in a medical office setting without the need for goggles or other interventions. ...Based on preliminary evidence from the company's Phase 1/2a study, MCO-010 is potentially applicable not only for RP but other degenerative diseases of the eye irrespective of gene mutations."
"Our data demonstrate that pluripotent stem cell derived cone precursors can engraft into the host retina in close apposition to the host bipolar cells. Half of the transplanted mice exhibited visual behaviour and of these, 33% showed binocular light sensitivity. The majority of retinal ganglion cells (RGCs) exhibited contrast‐sensitive light responses and motion sensitivity, however quite a few exhibited unusual responses reflecting remodelling of the neural retina. Together these data suggest that pluripotent stem cell‐derived cone precursors can elicit conventional and unusual light responsiveness at advanced degeneration stages."
"Although there currently are no options available to treat retinitis pigmentosa (RP), a novel approach has been evaluated in a phase 2 clinical trial that determined the clinical effect of the injection of human retinal progenitor cells to improve vision and visual fields in this patient population by activating and preserving retinal cells to treat RP.
"The trial’s findings indicated that this treatment may be able to enhance vision and visual fields in specific patients with RP whose living cells can be rejuvenated, according to David S. Boyer, MD, an adjunct clinical professor of ophthalmology at the Keck School of Medicine of USC in Los Angeles, California.
"The phase 3 trial will begin in the third quarter of 2022."
The Phase 2b trial which starts in June, will be a randomized, double-blind, sham-controlled, multi-center trial in the USA enrolling patients with advanced RP. The treatment will involve a single intravitreal injection of MCO-010 to confirm its ability to improve the patients' vision. MCO has received orphan drug designations for RP and Stargardt disease from the FDA.
Mark Pennesi, PhD, the Kenneth C Swan Associate Professor of Ophthalmology at the Casey Eye Institute in Portland, 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.”
"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)."