A new type of Gene Therapy demonstrates the potential to treat Blindness

(c) Veronique Juvin, Sciartwork

Doctors have been able to use a form of gene therapy described as optogenetics for the first time to restore partial vision in a blind person. The research team genetically altered retinal ganglion cells to become light-sensitive in a man whose vision was destroyed by retinitis pigmentosa, a genetic disorder that breaks down cells that absorb and convert light into brain signals.

Immune system may slow degenerative eye disease

A new study shows that the complement system, part of the innate immune system, plays a protective role to slow retinal degeneration in a mouse model of retinitis pigmentosa, an inherited eye disease. This surprising discovery contradicts previous studies of other eye diseases suggesting that the complement system worsens retinal degeneration. 

The research was performed by scientists at the National Eye Institute (NEI), part of the National Institutes of Health, and appears in the Journal of Experimental Medicine.

Significant Visual Improvement Reported in Patients with Retinitis Pigmentosa in a cell therapy clinical trial

(c) ReNeuron

ReNeuron Group plc, a UK-based company focused on development of cell-based therapeutics, has announced encouraging results from an early stage trial of its cell therapy for retinitis pigmentosa (RP), which, if approved, could be a competitor to Spark/Novartis’s Luxturna (voretigene neparvovec). Unlike the already-approved gene therapy Luxturna, ReNeuron’s cell-based therapy would not be limited to patients with a disease caused by a certain mutation.

GenSight received MHRA approval for testing Gene Therapy + Device in Retinitis Pigmentosa. May test patients with dry AMD

(c) GenSight Biologics

GenSight Biologics, a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced UK Medicines and Healthcare Regulatory Agency (MHRA) acceptance of the Company’s Clinical Trial Application (CTA) to initiate the PIONEER Phase I/II study of GS030 in patients with Retinitis Pigmentosa (RP).

Gene therapy shows promise for reversing blindness

In a laboratory study in Oxford, researchers have shown how it might be possible to reverse blindness using gene therapy to reprogram cells at the back of the eye to become light sensitive.

Stem-cell derived retina found to improve vision in monkeys with RP

Researchers at the RIKEN Institute in Japan have succeeded in growing human embryonic stem cell-derived retina (hESC-retina) and transplanting that tissue into the retinas of rhesus monkeys with Retinitis Pigmentosa (RP). Three weeks after the treatment, visual tests on two of the monkeys found their vision had improved.

RetroSense Therapeutics Gets Approval for Clinical Trials in Retinitis Pigmentosa

(c) nature.com

RetroSense Therapeutics’ Investigational New Drug (IND) application for gene therapy based clinical trial for their product RST-001 has received clearance from the US Food and Drug Administration. RetroSense is developing RST-001 for the treatment of retinitis pigmentosa, a genetic condition that leads to the progressive degeneration of rod and cone photoreceptors (cells found in the retina that sense light), resulting in severe vision loss and blindness. With its IND now in effect, RetroSense expects to initiate a Phase I/II clinical trial by year-end in order to evaluate the safety and, potentially, efficacy of RST-001.

UCI-led team begins first clinical trial of stem cell-based retinitis pigmentosa treatment

Participants are being enrolled in the first clinical trial that tests the use of retinal progenitor cells to treat retinitis pigmentosa. The product of stem cell research at UCI’s Gavin Herbert Eye Institute and Sue & Bill Gross Stem Cell Research Center, these retinal progenitors are similar to stem cells in terms of potential regenerative properties, but they’re specific to the retina.

NIH research points to microglia as potential therapeutic target in Retinitis Pigmentosa

Spider-like cells inside the brain, spinal cord and eye hunt for invaders, capturing and then devouring them. These cells, called microglia, often play a beneficial role by helping to clear trash and protect the central nervous system against infection. But a new study by researchers at the National Eye Institute (NEI) shows that they also accelerate damage wrought by blinding eye disorders, such as retinitis pigmentosa.

Patients using Argus II report improvement in visual function and quality of life

The three-year clinical trial results of the retinal implant, popularly known as the "bionic eye," have proven the long-term efficacy, safety and reliability of the device that restores vision in those blinded by Retinitis Pigmentosa (RP), a rare, degenerative eye disease. The findings show that the Argus II significantly improves visual function and quality of life for people blinded by RP.

Neuronal-Glial Interactions in Retinal Disease

Matthew Zabel, of the National Eye Institute, talks about Neuronal-Glial Interactions in Retinal Disease.

Nanotechnology breakthrough will lead to a better artificial retina for visually impaired

(c) American Chemical Society

 
Scientists from Hebrew University of Jerusalem researchers in collaboration with colleagues from Tel Aviv University and Newcastle University, have developed a new light-sensitive film that could one day form the basis of a prosthetic retina to help people suffering from retinal damage or degeneration.

Clinical trial for autologous bone marrow stem cells as treatment for AMD & RP shows promise

A phase 1 clinical trial utilizing human bone marrow CD34+ stem cells for patients with retinal vascular occlusion, hereditary or non-exudative age-related macular degeneration (AMD), or retinitis pigmentosa (RP) has shown safety and feasibility.

Rosemary may have a beneficial effect for patients with Age-related macular degeneration and Retinitis Pigmentosa

Scientists at the Del E Web Center for Neuroscience, Aging, and Stem Cell Research, Sanford-Burnham Medical Research Institute, La Jolla, California, have found that carnosic acid (CA), present in rosemary extract, may potentially have clinical application to diseases affecting the outer retina, including age-related macular degeneration (AMD) and retinitis pigmentosa (RP).

Amarantus Applies for Orphan Drug Designation for MANF in treatment of Retinitis Pigmentosa

Amarantus Bioscience Holdings, Inc., a biotechnology company focused on the development of diagnostics and therapeutics for Alzheimer's disease, Parkinson's disease and ophthalmological disorders, announced that it has applied to the Food & Drug Administration in the US for Orphan Drug Designation for MANF (mesencephalic-astrocyte-derived neurotrophic factor) in retinitis pigmentosa (RP).

Harvard and Astellas Pharma collboarate on gene therapy for Retinitis Pigmentosa

Astellas Pharma Inc. has announced a research collaboration with Constance L Cepko, Ph.D., an investigator at Harvard Medical School, Boston, Massachusetts, focused on discovering the pathologic mechanism for retinitis pigmentosa and identification of  new therapeutic targets. Astellas and Harvard will work with the aim to provide a new treatment option with a view of a gene therapy, to retinitis pigmentosa patients who have difficulty maintaining good vision.