"Treatment with subretinal transplantation of allogeneic human retinal progenitor cells (hRPC) showed promising early signs of efficacy as a treatment for individuals with retinitis pigmentosa, according to findings from a phase 1/2a study presented at the 2019 American Academy of Ophthalmology (AAO) Annual Meeting."
"The company today reported results from its flagship drug program, in which its proprietary drug delivery technology was used on a 3D model of a retina created from human stem cells. According to Phylogica, the study achieved greater than 90% effectiveness after a single dose."
"A phase I/II trial run by the Dutch company ProQR has found that its RNA therapy could significantly improve the vision of people with Leber’s congenital amaurosis, a rare genetic disease for which there is no treatment."
While this is not for Usher Syndrome, a new treatment that is successful for one type of RP opens the possibility for all types of RP.
"Neuronal transplantation therapies show great promise to restore vision, but have been severely imped- ed by poor synaptic positioning within retinal host. This perspec- tives article highlights our group’s abilities to remarkably direct the migration of transplantable cells by applying electro-chemical fields within retina-customized microtechnology. Integration of our in vi- tro results with current ex vivo and in vivo studies has the potential to transformatively impact outcomes of cell replacement therapy."
"With total of 22 patients now treated and the study still ongoing, ReNeuron said the efficacy in subsequent patients was seen but at a lower rate and magnitude, with improvement in visual acuity ranging from +5 to +11 letters in the treated eye three months after treatment."
"In February data from three patients treated with Reneuron’s retinitis pigmentosa stem cell candidate raised hopes that the group might finally have a decent asset. Now, findings in five more patients have given the company a reality check. As well as showing less impressive efficacy, two subjects suffered procedure-related vision loss."
"In experiments with rats, pigs and monkeys, Johns Hopkins Medicine researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and effective in humans, the technique could provide a new, more permanent therapeutic option for patients with common diseases such as wet age-related macular degeneration (AMD), and it could potentially replace defective genes in patients with inherited retinal disease.
"The new approach, described in the August 13, 2019, issue of The Journal of Clinical Investigation, uses a small needle to inject harmless, genetically engineered viruses into the space between the white of the eye and the eye’s vascular layer, called the suprachoroidal space. From there, the virus can spread throughout the eye to deliver therapeutic genes to cells in the retina."
"The brain implant was put there by UCLA neurosurgeon Nader Pouratian. Pouratian developed the device with Second Sight Medical Products. It starts with a tiny camera on Esterhuizen's glasses, which sends an image to a video processing unit. A wireless signal is then sent to the implant, which stimulates his visual cortex."
“Photoreceptor replacement therapy is steadily moving closer to the clinic,” Dr. Stone concluded. “With the use of patient-derived cells, we can reduce the need for expensive long-term immunosuppression. Robots will help reduce manufacturing costs, which will increase access to these treatments for our patients.”
"Abstract / Synopsis:
The visual cortical prosthesis system is designed to bypass diseased or injured eye anatomy and to transmit these electrical pulses wirelessly to an array of electrodes implanted on the surface of the brain’s visual cortex, where it is intended to provide the perception of patterns of light."