Latest news with #pigmentosa
Business Wire
14-07-2025
- Business
- Business Wire
Golden Age Health and Innostellar Biotherapeutics Sign Exclusive 10-Year Collaboration to Accelerate LX-101 Gene Therapy for Inherited Retinal Dystrophies due to RPE 65 gene mutation in Mainland China
SHANGHAI--(BUSINESS WIRE)--Golden Age Health Pte. Ltd. (' GAH ') and Innostellar Biotherapeutics Co., Ltd. (' Innostellar ') today announced an exclusive ten-year Promotion Services Agreement that grants GAH sole rights to commercialise and promote Innostellar's first-in-class gene-therapy candidate LX-101 across Mainland China. Leveraging its full-spectrum patient-focused launch platform covering disease and medical education, market access and patient support to address the unmet needs along the patient journey such as disease awareness, timely diagnosis and treatment, treatment accessibility and affordability. GAH, along with Innostellar will act with urgency to bring this life-changing therapy to patients affected by inherited retinal dystrophies due to RPE 65 gene mutation in Mainland China so they can ' see the future, clearly'. Golden Age Health and Innostellar team up on a 10-year pact to bring sight-saving LX-101 gene therapy to China, aiming to help RPE65 IRD patients 'see the future, clearly.' 'Partnering with Innostellar positions GAH at the forefront of gene therapy in China's rapidly expanding ophthalmology market,' said Francis Wan, Chief Executive Officer of Golden Age Health. 'Our integrated approach aims to deliver sight-saving innovation to patients swiftly and comprehensively.' Dr Wang Fenghua, Founder & CEO of Innostellar, added: 'By joining forces with GAH, we can ensure LX-101 reaches patients across China rapidly and—crucially—at prices families can afford, thanks to efficiencies gained through local development and manufacturing. Improving patient access lies at the heart of our mission. About Inherited Retinal Dystrophies Inherited retinal dystrophies (IRDs)—including retinitis pigmentosa (RP) —are genetic disorders that progressively destroy photoreceptors, leading to severe vision impairment or blindness, often from childhood. RP affects approximately 1 in 3,000–4,000 people worldwide and is officially listed in China's National Rare Disease Catalog (first list, 2018), underscoring the country's commitment to improving outcomes for affected patients. About LX-101 LX-101 is an adeno-associated-virus (AAV) gene therapy delivering a functional RPE65 gene directly to retinal cells, aiming to restore the visual cycle in patients with biallelic RPE65-mutation IRDs. The programme is in Phase III clinical development in China, with top-line results expected in Q4 2025 and initiate NMPA New Drug Application soon after. If approved, LX-101 would provide eligible patients with a single-dose, durable treatment designed to halt—or potentially reverse—vision loss. About Golden Age Health Golden Age Health is a specialty pharmaceutical company dedicated to redefining patient access to medicine across China, Asia-Pacific and beyond. Headquartered in Singapore, GAH pairs data-driven market-access expertise with deep medical-affairs and RWE capabilities to bring high-impact therapies to underserved populations. About Innostellar Biotherapeutics Innostellar Biotherapeutics is a Shanghai-based biotechnology firm advancing innovative gene-therapy medicines for ocular and other genetic diseases and chronic diseases. Its pipeline is led by LX-101, the first gene therapy of its kind to enter late-stage development in China. Forward-Looking Statements This release contains forward-looking statements, including expectations regarding clinical development, regulatory approvals and commercial launches. These statements involve risks and uncertainties that may cause actual outcomes to differ materially.
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
BBC News
25-02-2025
- Sport
- BBC News
Ex-Paralympian Simon Goodall says running is 'both my passion and saviour'
A former Paralympian has said running is both his "passion and saviour" as he prepares to take on two half-marathons for Goodall was 11 when he was told he would one day go blind due to rare, genetic eye disease called retinitis pigmentosa (RP). While at a boarding school for the visually impaired, he found a passion for goalball - something he competed at nationally before representing Great Britain at the 2012 Paralympic Games in London - before experiencing total vision loss when both of his eyes were a volunteer for the Derby branch of the Samaritans charity, the 55-year-old will take on the two runs with sighted guides in a bid to raise £500. At the boarding school, Simon - from Crich in Derbyshire - said he thought he had learned a lot about what it would be like to lose his sight. In reality, he said, it was "very difficult" - but that did not stop him from doing what he wanted. It was during his school life in the 1980s that he found a club that played goalball - a game designed specifically for athletes with visual went on to be accepted into the Great Britain team, competing in national and international championships before taking part in the 2012 Paralympics. Although he eventually moved away from the sport, Simon said he remained determined to keep fit, and joined a local parkrun. "I had a dream to run a marathon and then one day, someone in the gym foolishly told me 'oh you'll never be able to do that - you're blind'," he said. 'Life doesn't just stop' Some years after the Paralympics, in 2017, Simon took part in a medical trial for a RP treatment, which involved having a tiny implant in his eye. "It worked for about six months and then stopped working," he said. The implant - which Simon said was a small chip - would "limit" him going forward. He said it could affect magnetic resonance imaging (MRI) scans, for example, and therefore decided to have his eye removed in 2020. His second eye was removed in 2022, he said, and later had two false eyes fitted."A lot of people don't understand that life doesn't just stop because you're blind," he said. "It is really challenging being diagnosed with anything but you do learn to cope - and that's what I do through my running."Having found a way to face life's challenges, Simon said he wanted to "give something back" to those who might still be struggling, and became a volunteer call handler at the Derby Samaritans branch."There's so many people out there who've got something going on," he said. Speaking about becoming a volunteer, he said: "I didn't truly know what to expect when I started. "But when you sit there and you answer the phone and you're helping people and you're listening to people, it's incredibly rewarding." In March, Simon will compete in the Dorney Lake half-marathon in Buckinghamshire and the Bournemouth Half Marathon in a bid to raise Derby branch has been running for more than 60 years."Every 10 seconds Samaritans responds to a call for help, so it's vital that the important work of the branch continues, and I just want to play my part," Simon added. If you have been affected by any of the issues in this story, support is available on the BBC Action Line.
