Projects
Active clinical trials
News on national clinical trials
Projects
Research on Inherited Retinal Diseases affecting Native Americans in the Southwest
The Retina Research Foundation of America provides funding for research in inherited retinal diseases. The Retina Research Foundation is promoting research in genetic causes for inherited retinal diseases affecting Native Americans in the Southwest such as inherited retinal diseases including retinitis pigmentosa. Research on L-DOPA for Age related macular degeneration
Age related macular degeneration (AMD) is the most common cause of vision loss over age 50. Retina Research Foundations support research on the use of L-DOPA to modify disease progression in age related macular degeneration.
Active Clinical Trials
Acelyrin 22103: A Phase 2b Pivotal Study to Evaluate the Efficacy and Safety of Izokibep in Subjects with Non-infectious, Intermediate-, Posterior- or Pan-uveitis.
Genentech Roche GR43828: A Phase I, Multicenter, Open-Label Study Of The Safety, Tolerability, Pharmocokinetics, And Immunogenicity Of Intravitreal Injections Of RO7446603.
Henlius HLX04-O-wAMD: A Phase 3, Two-part (Open-label Followed by Randomized Double-masked Active Controlled) Study to Compare the Efficacy and Safety of HLX04-O Administered by Intravitreal Injection with Ranibizumab in Subjects with wet Age-related Macular Degeneration (wAMD).
Rezolute RZ402-201: A Randomized, Double-Masked, Placebo-Controlled, Parallel-Arm Study to Evaluate the Efficacy and Safety of RZ402 in Participants with Diabetic Macular Edema (DME).
Genentech Roche BP44175 (PreGonda): A Multi-Center, Non-Randomized, Open-Label, Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Ro7497372 Following Intravitreal Administration in Participants with Diabetic Macular Edema.
FFB Uni-Rare: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated with Rare Disease- Causing Genetic Variants
Ocugen OCU400-101: A Phase 1/2 Study To Assess The Safety And Efficacy Of OCU400 For Retinitis Pigmentosa Associated With NR2E3 And RHO Mutations And Leber Congenital Amaurosis With Mutation(s) In CEP290 Gene.
Ocugen OCU400-104: A prospective and retrospective natural history study of retinitis pigmentosa associated with nr2e3 and rho mutations and leber congenital amaurosis with mutation(s) in cep290 gene.
Clearside CLS1002-202 (Odyssey): a phase 2b study of suprachoroidally administered cls-ax in participants with neovascular age-related macular degeneration.
Opthea OPT302-1005 (COAST): OPT-302-1005 – COAST – A Phase 3, Multicentre, Double-masked, Randomized Study to Evaluate the Efficacy and Safety of Intravitreal OPT-302 in Combination with Aflibercept, Compared with Aflibercept Alone, in Participants with Neovascular Age-related Macular Degeneration (nAMD).
Aviceda AVD-104-C01 A single and multiple dose study to evaluate the safety, pharmacokinetics, and treatment effect for intravitreal AVD-104 in participants with geographic atrophy secondary to age related macular degeneration.
Ocugen OCU410: A phase1/2 study to assess the safety and efficacy of OCU410 for Stargardt Disease.
Ocugen OCU410-101 A phase 1/2 study to assess the safety ad efficacy of OCU410 for geographic atrophy secondary to dry age-related macular degeneration.
News on National Clinical Trials
Update on research and treatments for inherited retinal diseases
The potential impact of current research and clinical trials for many forms of inherited retinal diseases and degenerations have been summarized in a paper in International Journal of Molecular Sciences. The published review examines various approaches in the research and development of treatments for inherited retinal degenerations. This paper explores treatments using the most current and innovative molecular techniques. Numerous clinical trials are ongoing. This review examines the role of neuroprotection in the potential treatment of diseases and discusses several gene therapy types (gene augmentation, editing, potential RNA modifications, and optogenetics). The role of stem cell or precursor-based treatments are also discussed. Recent successes in gene therapy have created a drive for similar treatments for numerous diseases with a genetic origin. The potential impact of current research and trials has the potential to treat retinal disease as restore visual function.
Martinez Velazquez LA, Ballios BG. The Next Generation of Molecular and Cellular Therapeutics for Inherited Retinal Disease. International Journal of Molecular Sciences. 2021; 22(21):11542. https://doi.org/10.3390/ijms222111542
Latest News:
New treatment for retinitis pigmentosa by Ocugen
The latest news out from Ocugen’s OCU400 Gene Therapy Study is encouraging. Ocugen, a biotechnology company is conducting a study with a treatment called OCU400 which could help people with Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA).
The study is showing that vision is improving in some of the studies participants and they can see more clearly. Interestingly this treatment is helpful regardless of what gene is causing the issue.
Dr. Shankar Musunuri, who’s in charge at Ocugen, said, “We want to help people with eye problems, and we believe OCU400 can make a big difference.”
The study was conducted with 18 people who had eye problems. Some got a little bit of OCU400, and some got more. They came from different places, were different ages, and had different eye problems. Ocugen also studied kids with eye problems.
What are these eye problems, you ask?
Well, Retinitis Pigmentosa and Leber Congenital Amaurosis are problems that make it hard for people to see. Over time, it can get worse and make them go blind.
Dr. Arun Upadhyay explained, “We’re working hard to help people with eye problems. We won’t stop until we make things better for them.”
The study looked at how well OCU400 works. They discovered that 83% of people got better or stayed the same in their vision. A few lucky individuals could see even better than before!
According to Dr. Lejla Vajzovic,”This news is exciting for people with eye problems. OCU400 can help them see better.”
The study showed that most people responded well to the treatment however a few participants had some problems, but they got better quickly.
New treatment for retinitis pigmentosa by Nanoscope
Nanoscope Therapeutics has been studying a treatment called MCO-010 which could help people with a condition called retinitis pigmentosa (RP). This condition makes it difficult for people to see and sometimes even causes blindness. Currently there are not any effective treatments for it, but MCO-010 might change that.
In a special test called the Phase 2b RESTORE trial, it showed that 88.9% of people who got MCO-010 could see better. They could recognize things and move around more safely. MCO-010 might be a game-changer for folks with retinitis pigmentosa.
MCO-010 also does not seem to be having any major side effects. This- as even the U.S. Food and Drug Administration (FDA) thinks MCO-010 is unique, and they gave it some special designations.
The company is planning to present these encouraging results at upcoming medical conferences, and are ready to work with regulatory agencies to make MCO-010 available to those who need it. While this is an ongoing study it does show promise for people who might be facing vision problems.
New treatment for Stragardt disease
Stargardt disease is a condition that can make people lose their vision, and it’s one of the most common reasons why even younger people go blind due to genetic eye issues. In the United States alone, more than 30,000 people have this disease. It happens because there’s a problem with a gene called ABCA4. This happens because of the build-up of harmful substances in the eye. Over time, this accumulation damages the retina, leading to gradual vision loss.
A new treatment called gildeuretinol (sometimes called ALK-001), is showing promising results for people with Stargardt disease. This medication is engineered to slow down the progression of the disease by reducing the production of toxic substances in the eye, which are related to vitamin A. The results from clinical trials have been positive, with a noticeable decrease in retinal damage among patients who received this treatment. The U.S. Food and Drug Administration has recognized its potential and granted it special status to make it available to patients sooner. This is a significant development that brings hope to those facing the challenges of Stargardt disease.
Gildeuretinol isn’t just limited to Stargardt disease; it’s also being investigated for its effectiveness in treating Geographic Atrophy (GA). GA is a type of age-related macular degeneration that can cause loss of central vision. It’s a widespread problem, affecting millions of people worldwide, especially those over the age of 50. Researchers are currently conducting studies to determine if it can slow down the progression of GA and help individuals maintain their vision.