ABCA4

SAN DIEGO, April 29, 2025 (GLOBE NEWSWIRE) -- Belite Bio, Inc. (NASDAQ: BLTE), a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the Company will give two poster presentations at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting being held May 4–8, 2025, in Salt Lake City, Utah. The presentations will highlight baseline patient characteristics from the ongoing DRAGON trial and pharmacokinetic and pharmacodynamic properties from the ongoing DRAGON II trial, both in Stargardt disease. Poster Presentations: Title: Baseline Characteristics of Adolescent Stargardt Disease Subjects Participating in a Phase 3 Study of Tinlarebant (DRAGON Trial)Presenting Author: Dr. Ruifang SuiPresentation Number: 1463Session Number: 215Session Title: Retinitis Pigmentosa and IRD ISession Date/Time: Monday, May 5, 8:30-10:15 a.m. MDT Title: Pharmacokinetic and Pharmacodynamic Properties of an Oral Investigational Treatment for Stargardt Disease in Adolescent Japanese PatientsPresenting Author: Dr. Kaoru FujinamiPresentation Number: 1431Session Number: 215Session Title: Retinitis Pigmentosa and IRD ISession Date/Time: Monday, May 5, 8:30-10:15 a.m. MDT About Tinlarebant (a/k/a LBS-008)Tinlarebant is a novel oral therapy that is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in Stargardt Disease type 1 (STGD1) and also contribute to disease progression in geographic atrophy, or advanced dry age-related macular degeneration (AMD). Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids. Tinlarebant has been granted Fast Track Designation and Rare Pediatric Disease designation in the U.S., Orphan Drug Designation in the U.S., Europe, and Japan, and Sakigake Designation in Japan for the treatment of STGD1. Stargardt Disease (STGD1)STGD1 is the most common inherited macular dystrophy (causing blurring or loss of central vision) in both adults and children. The disease is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision. The fluorescent properties of bisretinoids and the development of retinal imaging systems have helped ophthalmologists identify and monitor disease progression. Currently, there are no FDA approved treatments for STGD1. Importantly, the appearance of bisretinoids, followed by retinal cell death, and progressive loss of vision is also observed in geographic atrophy (GA) patients. Therefore, Belite Bio is evaluating safety and efficacy of Tinlarebant in GA patients in a 2-year Phase 3 study (PHOENIX). About Belite BioBelite Bio is a clinical-stage biopharmaceutical drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, such as Stargardt disease type 1 (STGD1) and Geographic Atrophy (GA) in advanced dry age-related macular degeneration (AMD), in addition to specific metabolic diseases. Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of toxins in the eye, is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects and a Phase 3 study (PHOENIX) in subjects with GA. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. Media and Investor Relations Contact:Jennifer Wu ir@belitebio.com Julie Fallonbelite@argotpartners.com

Groundbreaking research demonstrates how Nanoscope's synthetic opsin restores vision in patients with severe retinal degeneration DALLAS, March 24, 2025 /PRNewswire/ -- Researchers with Nanoscope Therapeutics Inc., a biotechnology company committed to restoring vision in blind patients in real-world settings by developing and commercializing novel gene therapies for retinal degenerative diseases, have announced the publication of a paper, "A synthetic opsin restores vision in patients with severe retinal degeneration," in Molecular Therapy, the flagship journal of the American Society of Gene & Cell Therapy. The publication of the paper marks a major breakthrough in mutation-independent optogenetic monotherapy for patients suffering from inherited retinal diseases. "We are excited by the therapeutic benefits seen in retinitis pigmentosa patients with progressive and permanent loss of photoreceptors and without any treatment option," said Dr. Santosh Mahapatra, the principal investigator for the study, and Chief Medical Officer of JPM Rotary Eye Hospital (Odisha) in India. "We look forward to further advancing this technology to help those with severe vision loss due to retinal degenerative diseases." Inherited retinal degenerative diseases (IRDs) are the leading cause of blindness in working-age adults worldwide, and in advanced stages, cell loss makes gene editing or replacement ineffective. Optogenetics offers a therapeutic opportunity to restore vision by photosensitizing remaining retinal neurons. However, current opsins are kinetically slow, partially activated in ambient light, unresponsive to different light colors, and target low-resolution retinal cell circuits. To overcome these limitations, the Nanoscope team engineered a synthopsin made of three selectively mutated non-mammalian proteins to achieve a broadband Multi-Characteristic Opsin (MCO). "This paper highlights the outstanding work of the Nanoscope team in developing effective optogenetic therapies for patients with some of the highest unmet needs," said Dr. Samarendra Mohanty, the lead author of the paper and President of Nanoscope. "The positive results of our Phase 1/2a trial outlined in this journal article, along with the randomized controlled trial data in RP, represent a major step forward in treating inherited retinal diseases." The engineered synthopsin was packaged into an optimized AAV2 gene therapy vector that targets human retinal bipolar cells. In an investigator-initiated, open-label study, four blind retinitis pigmentosa patients with ABCA4 variants received a single intravitreal gene therapy injection. Noninvasive imaging confirmed retinal gene expression via a fluorescent reporter protein. Patients showed improvement in visual acuity, shape discrimination, and mobility through the 52-week study period. There were no significant safety issues despite what is likely one of the most synthetic, non-mammalian proteins ever expressed in a human. This is the first report of a gene monotherapy that can restore vision in blind patients in a mutation-independent manner utilizing an optogenetics technology platform. "Our study represents a pivotal milestone in the treatment of inherited retinal degenerations, demonstrating that optogenetics can successfully restore vision in blind patients, regardless of their genetic mutation," said Dr. Vinit B. Mahajan, co-corresponding author and Professor and Vice Chair for Research, Department of Ophthalmology at Stanford University. "The ability to restore vision with a single intravitreal injection marks a significant step toward developing a universal treatment for retinal degenerative diseases." Click here to access the full paper and graphical representation of the clinical study. About Nanoscope Therapeutics Inc. Nanoscope Therapeutics is developing mutation-agnostic, sight-restoring optogenetic therapies for the millions of patients blinded by retinal degenerative diseases for which no treatment currently exists. Following positive end-of-study results from the RESTORE Phase 2b multicenter, randomized, double-masked, sham-controlled clinical trial for RP (NCT04945772), the company announced its plan to initiate a BLA submission for MCO-010 to treat RP in H1 2025. The company has completed the Phase 2 STARLIGHT trial of MCO-010 therapy in Stargardt patients (NCT05417126) and plans to initiate a Phase 3 registrational trial, also in the first half of 2025. MCO-010 has received FDA Fast Track designations and FDA orphan drug designations for both RP and Stargardt. Preclinical programs include an IND-ready non-viral laser-delivered MCO-020 asset for GA, as well as an AAV asset for Leber Congenital Amaurosis, in IND-enabling studies. Investor Contact: Argot Partners 212-600-1902 [email protected] SOURCE Nanoscope Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

BARCELONA, Spain I March 13, 2025 I SpliceBio, a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes, today announced that it has dosed the first patient in the Phase 1/2 ASTRA study of SB-007, a dual adeno-associated viral (AAV) vector, for the treatment of Stargardt disease. Stargardt disease is an inherited retinal disorder caused by mutations in the ABCA4 gene. It is the most common form of juvenile macular degeneration, leading to progressive central vision loss and ultimately resulting in blindness, with no approved treatments available. “Stargardt disease has remained elusive to gene therapies due to the large size of the ABCA4 gene. SB-007 is the first gene therapy in clinical development designed to restore expression of the full-length ABCA4 protein across all Stargardt disease patients, regardless of their mutations,” said Miquel Vila-Perelló, Ph.D., Chief Executive Officer and Co-Founder of SpliceBio . “Treating the first patient in the ASTRA study with SB-007 is an important milestone for SpliceBio and the Stargardt disease patient community, who are in desperate need for a therapy. I am immensely proud of our outstanding team at SpliceBio whose depth of experience in leading numerous ophthalmology gene therapy trials in the last 15 years is unparalleled.” Paul Yang, M.D., Ph.D., Chief of the Paul H. Casey Ophthalmic Genetics Division at Oregon Health & Science University Casey Eye Institute and Principal Investigator of Phase 1/2 ASTRA Study, commented: “Stargardt disease is a devastating inherited retinal disorder with no approved treatments available. The initiation of this study with the dual AAV vector gene therapy, SB-007, represents a critical advancement in finding a potential treatment option for patients with this disease. It is a privilege to be able to collaborate with SpliceBio on this groundbreaking clinical study, and I look forward to advancing research that could bring hope to patients affected by Stargardt disease.” Aniz Girach, MD., Chief Medical Officer of SpliceBio, remarked : “We are excited to have treated the first patient in the US with our lead gene therapy. A single dose of SB-007 has the potential to address the root cause of Stargardt disease across all ABCA4 mutations. The Phase 1/2 ASTRA study will assess the safety and efficacy of SB-007 in patients with Stargardt disease. We look forward to continuing enrolment in our Phase 1/2 ASTRA study, alongside our foundational POLARIS trial, with the ultimate purpose of providing an effective and safe therapy for patients. As the study progresses, we aim to present data at upcoming scientific conferences.” Alongside the ASTRA study, SpliceBio continues to actively enrol patients through its POLARIS trial, a natural history study monitoring disease progression and endpoints in Stargardt disease patients. For more information or to enquire about participation in the studies, please visit https://splice.bio/clinical/ . About SpliceBio SpliceBio is a clinical-stage genetic medicines company pioneering Protein Splicing to address diseases caused by mutations in large genes. The Company’s lead program, SB-007, targets the root cause of Stargardt disease, a genetic eye disease that causes blindness in children and adults. SpliceBio’s pipeline comprises additional gene therapy programs across therapeutic areas, including ophthalmology and neurology. SpliceBio’s platform is based on technology developed in the Muir Lab at Princeton University after more than 20 years of pioneering intein, Protein Splicing, and protein engineering research. For additional information, please visit www.splice.bio . About SB-007 SB-007 is an investigational Protein Splicing dual AAV gene therapy in development for the treatment of Stargardt disease. It is designed to restore expression of the native full-length ABCA4 protein in the retina. SB-007 has been granted Orphan Drug Designation from both the FDA in the US and the European Commission in Europe. In December 2024, SB-007 received FDA IND clearance, marking the first-ever clearance for a dual AAV gene therapy in Stargardt disease. Alongside initiation of the Phase 1/2 ASTRA study, SpliceBio continues to advance POLARIS, a natural history study of the disease. Both studies are actively recruiting. For more information or to enquire about participation in the studies, please visit https://splice.bio/clinical/ . About Stargardt disease Stargardt disease is the most common form of inherited juvenile macular degeneration, affecting approximately 1 in 8,000 to 10,000 individuals. Caused by mutations in the ABCA4 gene, Stargardt disease leads to the progressive loss of central vision due to damage to the central region of the retina known as the macula. The disease is variable in the age of onset, including early onset in children and adolescents and late-onset forms in adulthood. There are no approved treatments, and patients face significant challenges in daily life as the disease progresses. Stargardt disease has remained elusive to genetic medicines due to the large size of the ABCA4 gene. SOURCE: SpliceBio