Latest news with #RetinitisPigmentosa
Yahoo
5 days ago
- Health
- Yahoo
Non-profit fears for future of blindness research if Donald Trump's budget passes
LAKE COUNTY, Ill. (WGN) — Proposed federal funding cuts are hitting home in Lake County, especially when it comes to the blind. The Foundation Fighting Blindness, a Maryland-based non-profit organization who organizes the Chicago Vision Walk, fears clinical trials and promising new treatments for blindness and retinal disease will be frozen or canceled if President Donald Trump's 'Big, Beautiful Bill' passes into law. 'Six years old was when I got the diagnosis,' Rich Idstein said. 'It's tough.' Idstein is a legally blind man who works at Ace Hardware in Libertyville. He suffers from Retinitis Pigmentosa, a blinding retinal disease with no cure. Idstein and his two brothers all suffer from the same disease, which gets progressively worse with age. At the age of 52, Idstein figures he has five-to-ten years left before he's completely blind, but he's hoping clinical trials through the National Institutes of Health will lead to new treatments. 'We are driving research for therapies. They're called gene therapies that can replace or fix that code,' said Ben Shaberman, Vice President of Communications for the Foundation Fighting Blindness (FFB). 'So, the cells in the retina work correctly, and vision can be saved or restored.' The problem is, under Trump's proposed budget, the NIH is facing a potential 40% funding cut to medical research, which would directly impact the dollars going toward the clinical trials that Idstein hopes can help cure the disease that's plagued he and his brothers. The FFB has raised nearly $1 billion over the past five decades for eye and vision research, but its mission could be severely impacted if Congress doesn't act. For more info on the FFB, visit their website: Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Newsweek
02-06-2025
- Newsweek
Internet Unprepared for TSA Agent's Response to Dog at Airport
Based on facts, either observed and verified firsthand by the reporter, or reported and verified from knowledgeable sources. Newsweek AI is in beta. Translations may contain inaccuracies—please refer to the original content. A video of a dog getting a "full pat down" by a TSA (Transportation Security Administration) official at the airport has gone viral on TikTok. The clip, captured on May 18, was shared by @matthewandpaul and has had three million views since it was posted on May 21. Text overlaid on the clip says: "Guide dog getting full pat down by airport security." The video shows Paul Castle, who is from Seattle, Washington, at the airport security check area with his guide dog Mr. Maple. Castle, who is legally blind and has less than five percent of his vision remaining, was born with a rare eye disease known as Retinitis Pigmentosa. "Mr. Maple always gets a pat down at the airport due to the metal in his harness," Castle told Newsweek. The viral clip shows Mr. Maple walking through a metal detection device, with Castle waiting at the other side of it, standing beside a TSA agent. The agent is seen gently patting his hands around the pup's face and body. A caption shared with the post says: "Should we call it a 'pet down'?" Castle, who got Mr. Maple from Guide Dogs for the Blind in Oregon, has had his guide dog for four years. According to the Americans with Disabilities Act (ADA) website of the U.S. Department of Justice, service animals are defined as dogs of any breed and size that are "trained to perform a task directly related to a person's disability." Service animals are not "required to be certified or go through a professional training program" or "to wear a vest or other ID that indicates they're a service dog," the website notes. The U.S. Department of Transportation (DOT) notes that "airlines are required to recognize dogs as service animals and accept them for transport on flights to, within and from the United States." Viewers on TikTok were delighted by the guide dog in the viral clip, with some wondering why the dog needed to get a full pat down at the airport. Søren B Madsen 🇩🇰 asked "What can a dog hide?" and @wordsofnonsense said "where would he hide something." DamselWithoutDistress wrote: "TSA looking for a secret pocket in his fur?" Eris_christina noted' "definitely suspicious, Mr Maple was bribing the TSA with those kisses." Jaimie Moore wrote: "I feel like the security man just wanted an excuse to pet maple." 1KCA1 said: "Any excuse to pet the pups!!!" A stock image of a Labrador held on a leash in an airport setting. A stock image of a Labrador held on a leash in an airport setting. Getty Do you have funny and adorable videos or pictures of your pet you want to share? Send them to life@ with some details about your best friend and they could appear in our Pet of the Week lineup.
Yahoo
10-05-2025
- Health
- Yahoo
Skyline Therapeutics Presents Late-Breaking Abstract on SKG1108, a Novel Optogenetic Gene Therapy for Retinitis Pigmentosa, at ASGCT 2025
SHANGHAI, May 10, 2025 /PRNewswire/ -- Skyline Therapeutics, an innovation-driven clinical-stage gene therapy company focused on developing unique and novel solutions for rare and severe diseases, today announced a late-breaking abstract presentation on SKG1108, its investigational optogenetic gene therapy for Retinitis Pigmentosa (RP), at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting (May 13–17, 2025, New Orleans, Louisiana). ASGCT 2025 Late-Breaking AbstractAbstract Title: SKG1108: Optogenetic Gene Therapy with AAV-BWLP for Visual Restoration in Retinitis Pigmentosa via a Single Intravitreal InjectionAbstract No.：2023Date & Time: May 13, 2025 | 6:00 PM – 7:30 PM CT About SKG1108SKG1108 is an optogenetic adeno-associated virus (AAV) gene therapy vector designed to restore vision in RP patients with photoreceptor degeneration. Its novel design combines a proprietary intravitreal capsid and a uniquely engineered Broad Wavelength Light-sensing Protein (BWLP) comprising opsins of multiple sources, which demonstrated strong and broad wave-length range light-responsive activity in in vitro electrophysiological studies. In in vivo studies, a single intravitreal injection of SKG1108 showed superior efficacy and safety in rd1 mice, with striking functional improvements in visual pathway electrophysiology, light perception and visual acuity behavioral tests. The therapy delivers a single-stranded DNA payload encoding light-activatable proteins to the retina, bypassing the need to target specific genetic mutations. By generating new photo-sensing cells, SKG1108 aims to restore visual function in late-stage RP patients, regardless of their underlying genetic defect. SKG1108 received U.S. FDA Orphan Drug Designation (ODD) in September 2024. About Retinitis Pigmentosa (RP)Retinitis Pigmentosa (RP) is a type of inherited retinal diseases (IRDs) linked to mutations in over 100 different genes, leading to progressive vision loss that typically begins with night blindness (rod degeneration), followed by tunnel vision and eventual legal blindness due to near-total photoreceptor death (rod and cone degeneration). Currently, no effective treatments worldwide can effectively slow, halt, or reverse photoreceptor degeneration, and existing gene-specific therapies only apply to a small subset of RP patients. SKG1108's mutation-agnostic optogenetic approach—designed to restore vision independent of genetic defects—has the potential to become a transformative treatment for the vast majority of RP patients, including those with advanced disease. About Skyline TherapeuticsSkyline Therapeutics is an innovation-driven, clinical-stage gene therapy company focused on developing unique and novel therapeutic solutions for rare and severe diseases with high unmet needs. Leveraging our cutting-edge adeno-associated virus (AAV) platform, which encompasses multiple proprietary technologies for capsid discovery, vector design and engineering, process development and GMP manufacturing, we are advancing a diverse pipeline of gene therapies targeting ocular, neurological, and cardiovascular disorders. Our lead programs are rapidly progressing through clinical trials with regulatory approvals and special designations from the US FDA and China NMPA, bringing us closer to providing life-changing therapies to patients in need. About ASGCTThe American Society of Gene & Cell Therapy (ASGCT) is a professional non-profit medical and scientific organization dedicated to the understanding, development and application of gene, related cell and nucleic acid therapies, as well as promotion of professional and public education in the field. With more than 5,000 members in the US and worldwide, ASGCT is the largest association of individuals involved in gene and cell therapy research. The ASGCT Annual Meeting is the premier event for gene and cell therapy professionals to learn from the latest scientific research and stay current on new technologies. View original content: SOURCE Skyline Therapeutics
Business Wire
24-04-2025
- Business
- Business Wire
Ray Therapeutics Awarded $8M CIRM Grant to Advance RTx-015 Gene Therapy for Retinitis Pigmentosa
BERKELEY, Calif.--(BUSINESS WIRE)-- Ray Therapeutics, a biotechnology company developing optogenetic gene therapies for vision restoration, today announced it has been awarded an $8 million grant from the California Institute for Regenerative Medicine (CIRM). The grant will support the company's ongoing clinical development of RTx-015 for the treatment of retinitis pigmentosa (RP), a progressive and debilitating inherited retinal disease that leads to blindness. Ray Therapeutics' approach uses an optimized optogenetic gene therapy to deliver light-sensitive proteins to the retina of the eye to restore visual function to patients with RP, regardless of the underlying genetic cause. 'Retinitis pigmentosa remains a devastating condition with no approved treatments for the vast majority of patients,' said Paul Bresge, CEO & Co-Founder, Ray Therapeutics. 'We are deeply grateful to CIRM for their belief in our science and their continued support of our programs. We are honored to partner with CIRM as we advance therapies that have the potential to transform the lives of patients.' 'Restoring vision is one of the most powerful ways we can improve quality of life,' said Jonathan Thomas, PhD, JD, President and CEO, CIRM. 'Ray Therapeutics is advancing a potential breakthrough treatment for a high unmet medical need for people in California and around the world with advanced RP, for whom there are currently no treatment options. We are proud to support this exciting program.' Ray Therapeutics' gene therapy program received a unanimous vote of support from CIRM's scientific and patient advocate reviewers. It was recognized by CIRM's independent Grants Working Group (GWG) as having exceptional scientific merit and a high potential for impact, with all 15 reviewers scoring the application at the highest level. About Retinitis Pigmentosa RP is a genetic disease in which the photoreceptors gradually degenerate resulting in complete, or nearly complete blindness for most patients. The symptoms of RP include night blindness, reduced visual fields, and eventual loss of visual acuity. Patients are typically diagnosed in the first decades of life. It is estimated that more than half a million people are affected by RP worldwide. At present, no effective treatment is available for RP. About Ray Therapeutics Ray Therapeutics is a clinical-stage biopharmaceutical company advancing optogenetic therapies to restore vision in patients with severe retinal degeneration. By delivering a bioengineered, highly light-sensitive protein to targeted retinal cells, the approach is designed to improve visual function regardless of the underlying genetic mutation. The company's lead candidate, RTx-015, targets retinal ganglion cells and is currently being evaluated in a Phase 1 clinical trial for patients with retinitis pigmentosa and choroideremia. A second program, RTx-021, which targets retinal bipolar cells, is in late-stage preclinical development for Stargardt disease and geographic atrophy secondary to age-related macular degeneration. Ray Therapeutics is headquartered in Berkeley, California. For more information, visit About the California Institute for Regenerative Medicine (CIRM) The California Institute for Regenerative Medicine (CIRM) is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with $3 billion from the state of California to support ongoing research, and in 2020, was funded again with another $5.5 billion through Proposition 14 to continue the Agency's important work. CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency's research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state. For more information, go to
Yahoo
17-04-2025
- Health
- Yahoo
'You just walked into the road'
"People who are fully sighted are actually blind to the needs of people with a visual impairment," says a Hampshire mayor, adding "that needs to change". The Mayor and Mayoress of Eastleigh have been shopping in the town's centre wearing goggles designed to impair their vision. They want to improve accessibility for those living with a visual impairment (VI) and raise awareness among other town councils. Dave and Jean Pragnell spent more than an hour shopping to try to understand what people living with VI experience. The goggles they wore were designed to mimic sight loss caused by Retinitis Pigmentosa, a genetic condition also described as pin-hole vision. Rob Hiley, who also accompanied the mayor on his shopping spree, was born with the condition but didn't notice his vision deteriorate until he was 45. Now 72, he says what he sees is similar to the tunnel-vision goggles worn by the mayor: "I think it's fantastic and long overdue that people in authority actually take an interest in people with a visual impairment." With the help of her grandchildren, the mayoress walked around town wearing goggles mimicking diabetic retinopathy; a complication of diabetes which damages the retina. She described the experience as having "very blurry vision with large dark floaters". "Eastleigh was not the easiest place for a person with a sight loss to move about as there are so many things on the pavements," she said. The goggles were supplied by local charity OpenSight which supports people in Hampshire with VI. CEO Chris Dixon, who joined the mayor on his experience, said people living with VI find accessing the community a real challenge: "It's really important for us to be able to demonstrate those challenges and to try and get that empathy and awareness for the community to try and help." During the walk Mr Pragnell highlighted areas of navigation he found difficult, such as the lack of tactile pavements, which indicate crossings, and floral planters: "They [planters] look great for people that can see, but unfortunately they're right in the way of people with sight impairment. We need to rethink that sort of thing." He added that all council officers needed to do put the goggles on and experience what he did. "Without it, they cannot do their jobs properly," he said. You can follow BBC Hampshire & Isle of Wight on Facebook, X, or Instagram. Goggles 'give back' sight to Maisy Goggles help blind girl 'see' animals again Blind opera singer's concern over pothole danger Press Pack: How books are helping blind children experience stories through touch 'Learning guitar helped me cope with going blind' Eastleigh Borough Council Mayor of Eastleigh OpenSight
