ABCA4

SAN DIEGO, Oct. 14, 2024 (GLOBE NEWSWIRE) -- Belite Bio, Inc (NASDAQ: BLTE) (“Belite” or the “Company”), 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 present data from its completed Phase II adolescent Stargardt disease trial at the American Academy of Ophthalmology Annual Meeting (AAO 2024) being held October 18-21, 2024, in Chicago, IL. Podium Presentation: Title: Tinlarebant (LBS-008) for Adolescents with Stargardt DiseasePresentation Type: Late breaking developments (live broadcast)Presenting Author: Quan Dong Nguyen, MD, MScSession: RET16Date/Time: Saturday, October 19, 2024; 10:33 AM – 10:40 AM CDTLocation: Arie Crown Theater (Lakeside Center building, 1F), Chicago, IL E-poster Presentation Title: Investigation of an Oral Retinol Binding Protein 4 Antagonist in the Treatment of Childhood-onset Stargardt DiseasePresenting Author: Nathan L Mata, PHDDate/Time: On-demand 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 Bio Belite 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. Safety and efficacy of Belite’s lead candidate, Tinlarebant, an oral therapy intended to reduce the accumulation of vitamin A-based toxins in the eye, is currently being evaluated in three late-stage clinical trials. In STGD1, a 2-year, Phase 3 study (DRAGON) and a 2-year, Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects are currently ongoing. In GA, a 2-year, Phase 3 study (PHOENIX) is ongoing. For more information, follow us on Twitter, Instagram, LinkedIn, Facebook or visit us at www.belitebio.com. Forward Looking Statements This press release contains forward-looking statements about future expectations and plans, as well as other statements regarding matters that are not historical facts. These statements include but are not limited to statements regarding the potential implications of clinical data for patients, and Belite Bio’s advancement of, and anticipated preclinical activities, clinical development, regulatory milestones, and commercialization of its product candidates, and any other statements containing the words “expect”, “hope” and similar expressions. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including but not limited to Belite Bio’s ability to demonstrate the safety and efficacy of its drug candidates; the clinical results for its drug candidates, which may not support further development or regulatory approval; the timing to complete relevant clinical trials and/or to receive the interim/final data of such clinical trials; the content and timing of decisions made by the relevant regulatory authorities regarding regulatory approval of Belite Bio’s drug candidates; the potential efficacy of Tinlarebant, as well as those risks more fully discussed in the “Risk Factors” section in Belite Bio’s filings with the U.S. Securities and Exchange Commission. All forward-looking statements are based on information currently available to Belite Bio, and Belite Bio undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law. Media and Investor Relations Contact: Jennifer Wu / ir@belitebio.com Julie Fallon / belite@argotpartners.com

MILAN, Oct. 08, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces three oral presentations at the European Society of Gene & Cell Therapy 31st Annual Meeting (ESGCT), to be held October 22-25 in Rome. AAVantgarde is validating its platform technology in two inherited retinal diseases with clear unmet need, with Usher syndrome type 1B already in the clinic and Stargardt advancing towards clinical development. At this conference, we will be presenting NHP preclinical data from our Stargardt’s program and the process development, analytics and manufacture of the dual vectors to enable the clinical supply for our programs. In addition, our Scientific Founder, Professor Alberto Auricchio, TIGEM Scientific Director, will give a presentation on expediting AAV-based gene therapies. Oral Presentations details: Presentation Date/Time: Tuesday 22 October 2024, 14.00-16.30 h CESTSession title: ExpEditing AAV gene TherapySession Room: Plenary HallFinal abstract number: INV01 Presentation Date/Time: Tuesday 22 October 2024, 17.00-19.30 h CESTSession title: Safety and expression of intein-based Dual AAV8.ABCA4 in the non-human-primate retinaSession Room: Meeting room 2Final abstract number: OR017 Presentation Date/Time: Friday 25 October 2024, 11.00 – 13.00 h CESTSession title: Production of dual AAV vectors for the delivery of large genesSession Room: Plenary HallFinal abstract number: INV73 About AAVantgardeAAVantgarde Bio is a clinical stage, international biotechnology company that has developed two proprietary Adeno-Associated Viral (AAV) vector platforms to address the gene therapy cargo capacity limitations of AAV vectors. The AAVantgarde platforms could be used to deliver large genes to ocular and non-ocular tissues. AAVantgarde is initially validating its platforms in two inherited retinal diseases with clear unmet need, with its lead program in Usher syndrome type 1B already in the clinic. For more information, please visit: www.aavantgarde.com Contact:Magda Blanco – Head of Corporate Development AAVantgardeEmail: info@avvantgarde.com

Ascidian Therapeutics has a secret list of nearly 400 diseases that its gene-altering technology might help cure. But you’ll only find one listed on their website. And so far, that single-mindedness is paying off. Earlier this year, the Boston-based biotech became the first company to receive the FDA’s blessing to begin a clinical trial of an RNA editing therapy in the US. Ascidian is one of several small biotechs designing therapies that change the code of short-lived messenger RNA molecules, rather than permanently altering DNA. And while most RNA editing companies are trying to make single-letter changes to genetic code, Ascidian has devised a way to swap out large swaths of RNA, spanning hundreds to thousands of letters. It’s a search-and-replace tool for RNA that could treat many diverse causes of a genetic condition with a single therapy. “We were highly aware of all the other things going on,” CEO Michael Ehlers told Endpoints News . “We wanted to leapfrog all of that.” Ascidian hasn’t made as big of a splash as other startups in the gene editing field, in part because its $90 million in funding from Apple Tree Partners and single disclosed program feels modest compared with the hundreds of millions of dollars that other gene editing startups have raised for pipelines spanning a dozen diseases or more. The startup currently has about 40 employees, and Ehlers doesn’t anticipate getting much bigger.  “Our philosophy is focus,” he said. Its lead target is treating an inherited form of vision loss called Stargardt disease. The cause of the condition is well-known. Any one of more than 1,000 different mutations in a gene called ABCA4 can cause progressive death of photoreceptors in the eye, often in children and young adults. In theory, replacing the whole gene should treat the condition. But ABCA4 is simply too big to fit into the viral vectors commonly used in gene therapy. And using gene editing to fix each of the many DNA mutations is impractical. Instead, Ascidian designed a single therapy that replaces a large swath of the RNA where roughly two-thirds of mutations arise. The company is currently enrolling 13 patients in its Phase 1 clinical trial. It’s primarily focused on safety, but Ascidian will also monitor the vision-robbing lesions in the back of the eye. Although Ehlers doesn’t expect to restore vision that’s already lost, he hopes to dramatically slow, and maybe halt, the disease. “Coming out of it I think we will be able to say quite a bit on the efficacy signals and safety of the drug,” Ehlers said. Ascidian has yet to publish or present details on exactly how its RNA editing works — another reason it has kept a low profile — but CSO Robert Bell explained that it hijacks the familiar cellular process called RNA splicing. RNA molecules are like photocopies of the DNA master file. But before cells can use them to make proteins, they must remove genomic fillers and stitch together the actual code, regions known as exons. Normally, this splicing occurs within a single molecule. But some organisms — including ascidians, better known as sea squirts — can swap exons between different RNA molecules in a process called trans-splicing. Partly inspired by those squishy marine animals, Ascidian’s technology uses an RNA molecule to trigger trans-splicing at will. Its Stargardt therapy replaces the first 22 of 50 exons in the ABCA4 gene where the majority of mutations arise. If the approach works, the company could make a second therapy to address mutations in the remaining exons, Ehlers said. Ascidian’s origins trace back to a 2018 paper from University of Pennsylvania scientist Jean Bennett, who developed the RNA editing approach for a different rare form of vision loss. Her technology was first licensed to Limelight Bio and then spun out into Ascidian in early 2020 before Limelight shut down. Since then, Ascidian has worked to improve the rates of RNA editing to as much as 80% in some applications, Ehlers said. As for what’s beyond Stargardt, the company isn’t saying much. The startup recently struck a partnership with Roche worth $42 million upfront and as much as $1.8 billion in milestone payments, with a focus on unnamed neurological diseases. The startup won’t say how long its current funding will last, but chief financial and business officer Daniel Rosan said that “a significant amount” of the Roche partnership is in potential near-term payments. “And because the application of the tech is so broad, we are entertaining other partnerships,” he said. Key backer: Apple Tree Partners Find the full list of 2024 Endpoints 11 winners here .