"The company's modifier gene therapy platform aims to target nuclear hormone receptors (NHRs) that regulate multiple functions within the retina, giving it the potential to address many different gene mutations – and, in turn, multiple retinal diseases – with a single product.

"The planned Phase 1/2 study will evaluate the safety and proof-of-concept of OCU400, utilizing unilateral sub-retinal injection (one eye per study subject) in 18 patients, with an extension for contralateral eye and long-term safety follow up."

"Long-term survival of the co-graft in the rat subretinal space and improvement in visual function were observed. Immunohistochemistry showed that co-grafts grew, generated new photoreceptors and developed neuronal processes that were integrated into the host retina. This novel approach can be considered as a new therapy for complete replacement of a degenerated retina."

"Conclusions: Gene therapy is an extremely promising therapeutic tool for retinal diseases. The experimental data reported in this review offer a strong hope that gene therapy will be effectively available in clinical practice in the next years."

"AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, today announced the publication of the peer-reviewed manuscript, "Co-grafts of Human Stem Cell Derived Retina Organoids and Retinal Pigment Epithelium for Retinal Reconstruction in Immunodeficient Retinal Degenerate Royal College of Surgeons Rats," in the journal Frontiers in Neuroscience. The study, led by researchers at AIVITA Biomedical and the Stem Cell Research Center at the University of California, Irvine, used retina organoids and polarized retinal pigment epithelium sheets generated from human stem cells developed by AIVITA to test their viability as a "total retina patch" for vision loss."

Professor Gregg Suaning is pushing vision capture and nerve stimulation technologies beyond their limits to help clear a path. Read about his work to develop a bionic eye implant.

"The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership (AMP) program and project-managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases."

SCIENTISTS have developed a 'pellet' implant that's injected into the eye to prevent cataracts forming - and might even reverse the growth of existing cataracts without surgery.

"Gene therapy, given via a single intravitreal injection, introduces a gene that codes for a light-sensitive protein (ChrimsonR-tdT) into the ganglion cells of the retina, making them responsive to light and bypassing photoreceptors caused by diseases such as retinitis pigmentosa (RP)."

One of our funded researchers, Leah Byrne, PhD, University of Pittsburgh

"A new gene therapy company attempting to commercialize the research of Penn Medicine's Dr. Jean Bennett launched Wednesday in North Carolina with $19 million in seed capital.

"The company, Opus Genetics, is the first spinout company internally conceived and launched by the Retinal Degeneration Fund (RD Fund), the venture arm of the Foundation Fighting Blindness. The fund was created to further the foundation's mission of backing research to prevent, treat and cure retinal degenerative diseases."