Shanghai, China, May 7, 2024 – Skyline Therapeutics, an innovation-driven gene therapy company dedicated to developing unique and novel solutions to address unmet needs in rare and severe diseases, presents data of multiple novel AAV gene therapy programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting.
Selection and characterization of AAV capsids with improved tropism for ocular tissues in NHP
Poster #1444
Through a meticulous screening process in non-human primates (NHPs), our research identified AAV capsids with heightened tropism and transduction efficiency for retinal cells. Following thorough characterization and testing in NHP and rodents via intravitreal injection, these novel capsids consistently outperformed the benchmark AAV2 and a reference capsid, displaying increased transduction rates and biodistribution throughout the retina. Our findings suggest that these capsids possess favorable properties for traversing the retinal layers, suggesting their potential application in gene therapy for ocular diseases.
SKG0106, an intravitreal delivered AAV vector, for effective, safe and durable treatment of nAMD
Poster #1626
SKG0106 emerges as a promising gene therapy for neovascular age-related macular degeneration (nAMD), demonstrating potent inhibition of vascular endothelial growth factor (VEGF) through intravitreal delivery. Preclinical studies revealed sustained efficacy in rabbit and monkey models, with comparable outcomes to Eylea® and a favorable safety profile. The first-in-human, dose-escalation study that is being completed confirms SKG0106's safety and tolerability in patients across all dose cohorts, as well as its preliminary efficacy indicated by the trend of stabilization or improvement in BCVA and reductions in CST, marking a significant milestone in advancing this novel AAV gene therapy towards addressing the unmet needs of nAMD treatment. The multi-regional Phase I/II clinical trials are currently ongoing in the US and China.
SKG1201: Liver De-targeting Vector for Spinal Muscular Atrophy Gene Therapy via Intrathecal Injection
Poster #628
SKG1201 represents a pivotal advancement in spinal muscular atrophy (SMA) gene therapy, aiming to mitigate systemic toxicity while enhancing efficacy. Through innovative liver de-targeting capsids and a fully optimized SMN expression cassette, SKG1201 demonstrates exceptional potency in improving survival and motor function in preclinical models. Notably, intrathecal delivery (IT) of SKG1201 exhibits promising results, with significantly higher SMN expression in the spinal cord and minimal liver distribution compared to reference vectors. With ongoing studies to assess safety and biodistribution, SKG1201 emerges as a prospective gene therapy candidate for type II/III SMA patients, offering a potentially safer and more efficacious treatment option.
SKG0702, an AAV Gene Therapy Product for the Treatment of Wilson Disease
Poster #1081
SKG0702 represents a groundbreaking gene therapy approach for Wilson disease, a debilitating liver disorder. Utilizing an AAV vector expressing a codon-optimized mini-ATP7b transgene driven by a liver-selective promoter, SKG0702 demonstrates superior hepatocyte transduction in both in vitro and in vivo studies. In Atp7b-/- mice, SKG0702 significantly reduces hepatic copper accumulation and achieves high levels of mini-ATP7b expression, and in NHP, the vector achieves broader biodistribution and higher transduction in the liver than the vector using AAV5 capsid, offering promising gene therapy solution for treating Wilson disease with potential clinical benefits over conventional therapies.
About Skyline Therapeutics
Skyline Therapeutics is an innovation-driven, clinical-stage gene therapy company dedicated to developing unique and novel solutions to address unmet needs in rare and severe diseases. Our cutting-edge adeno-associated virus (AAV) platform comprises multiple proprietary technologies for capsid discovery, transgene design, and vector engineering. Combining our AAV expertise with robust in-house process and analytical development, as well as GMP manufacturing for clinical-grade plasmid and viral vector production, we are advancing a diverse pipeline of gene therapies for ocular, neurological, cardiovascular and metabolic disorders. Our lead programs are currently in clinical trials with regulatory approvals from the US FDA and China NMPA, bringing us closer to providing life-changing therapies to patients in need. Skyline Therapeutics has established a global presence for research, development, regulatory, and manufacturing in Shanghai and Hangzhou, China, and Boston, MA, USA. www.skytx.com
About ASGCT
The 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. To learn more about ASGCT, visit its official website www.asgct.org
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