Leber Congenital Amaurosis

DURHAM, N.C., April 29, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced it will present at the following scientific conferences in May: Retinal Therapeutics Innovation Summit 2025 – Salt Lake City, UTSession: Retinal Genetic AugmentationDate and Time: Friday, May 2, 2025, 3:30-3:45 p.m. MTPresentation Title: Advancements in IRD Gene Therapy: From the Bench to the ClinicPresenter: Kenji Fujita, MD, Chief Medical Officer, Atsena Therapeutics ARVO 2025 Symposium – Salt Lake City, UTSession: Innovating Breakthroughs in Ocular Gene TherapyPresentation Date and Time: Thursday, May 8, 2025, 8:51-9:09 a.m. MTPresentation Title: Interim safety and efficacy data in XLRS patients validates the use of a novel, laterally spreading capsid for the treatment of retinal diseasePresenter: Lesley Everett, MD, PhD, Assistant Professor, Ophthalmology, Oregon Health & Science University Poster Session: Retinal Function Testing and Advanced TherapiesPresentation Date and Time: Thursday, May 8, 2025, 2:00-3:45 p.m. MTPosterboard Number: A0499Poster Title: Quantitative Comparison of Two Physical Mobility Tests as Functions of FST and BCVA to Evaluate Therapeutic Benefit in LCA1 Patients Treated with Unilateral Gene TherapyPresenter: Alexandra Viviann Garafalo, Research Project Manager, Perelman School of Medicine, University of Pennsylvania American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting – New Orleans and VirtualSession: Gene Therapy Trials - In-Vivo Gene Therapy ModificationDate and Time: Friday, May 16, 2025, 5:15 p.m. ET Presentation Title: Safety and Efficacy of ATSN-201 Dose Escalation in Patients with X-linked Retinoschisis (XLRS)Presenter: Shannon Boye, PhD, Founder and Chief Scientific Officer, Atsena Therapeutics 3rd Annual Retinal Imaging Biomarkers & Endpoints Summit – Boston, MASession: Delving into Commercial Trends, Trial Insights, and Therapeutic Applications to Spearhead DevelopmentDate and Time: Thursday, May 29, 2025, 2:30 p.m. ETPresentation Title: Delving Into Emerging Insights for Trial Endpoints: Analyzing Correlations Between FST & MLMTPresenter: Kenji Fujita, MD, Chief Medical Officer, Atsena Therapeutics About X-linked Retinoschisis (XLRS)XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments. About AAV.SPRAAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/. About Atsena TherapeuticsAtsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/. Media Contact:Gina Mangiaracina6 Degrees(917) 797-7904gmangiaracina@6degreespr.com Business Contact:info@atsenatx.com

April 15, 2025 -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment. “We’re honored that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-201, further underscoring its potential to address the urgent, unmet need in XLRS—a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “This regulatory momentum, coupled with the recent close of our oversubscribed $150 million Series C financing, reinforces our commitment to advancing meaningful gene therapies that have the potential to improve vision and quality of life for individuals living with XLRS and other inherited retinal diseases.” Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for that disease or condition. RMAT designation provides sponsors with intensive FDA guidance on efficient drug development, including the ability to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review. Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and the EU. The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones, and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in the U.S. and EU have XLRS, for which there are currently no approved treatments. AAV.SPR, one of Atsena’s novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina (where schisis cavities predominate in XLRS patient retinas) when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins. This is in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. Atsena Therapeutics (“Atsena”) is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company’s lead program is evaluating ATSN-201 in an ongoing Phase I/II clinical trial for X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. ATSN-101, Atsena’s first-in-class, investigational gene therapy for Leber congenital amaurosis type 1 (LCA1) has completed a Phase 1 / 2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8). Atsena is advancing ATSN-101 toward the initiation of a global pivotal trial as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd. Atsena’s pipeline is powered by novel adeno-associated virus (AAV) technology tailored to overcome the hurdles presented by inherited retinal diseases. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. The content above comes from the network. if any infringement, please contact us to modify.

近日， Atsena Therapeutics宣布其在研基因治疗产品 ATSN-201 获得美国FDA授予的“再生医学先进疗法（RMAT）”认定，用于治疗X连锁性视网膜劈裂症（XLRS）。这标志着ATSN-201已获得FDA四项关键认定包括：RMAT（再生医学先进疗法）  Fast Track（快速通道）  Rare Pediatric Disease（罕见儿科疾病）  Orphan Drug（孤儿药认定） 关于X连锁性视网膜劈裂症（XLRS）XLRS是一种由RS1基因突变引起的单基因X连锁遗传病。RS1编码的视网膜分泌蛋白（Retinoschisin）主要由视杆细胞、视锥细胞及双极细胞分泌。该病表现为视网膜结构的异常分层（即“劈裂”），导致不可通过眼镜矫正的视力下降，病情逐渐加重，最终可能导致失明。XLRS几乎仅影响男性，通常在儿童时期确诊，目前尚无获批疗法。关于AAV.SPRAAV.SPR是Atsena公司开发的一种新型腺相关病毒（AAV）衣壳，其特点是在视网膜下注射后可横向扩散，超出原始注射区域，有效转导中央视网膜（XLRS患者视网膜劈裂病灶主要集中区域）。非人灵长类动物的临床前研究显示，AAV.SPR可显著扩大转基因表达范围，相较于传统AAV载体，其不需要进行中央凹剥离手术便可有效转导中心凹视锥细胞，且具有良好的安全性。临床进展：正在进行I/II期“LIGHTHOUSE”临床试验（NCT05878860），招募6岁以上确诊XLRS的男性患者。RMAT认定意义重大RMAT认定可加速产品审批进程，获得FDA更密切的指导和监管支持，为后续BLA申请、优先审评等铺平道路。Atsena公司同时也在推进治疗LCA1的基因疗法ATSN-101，未来还将启动全球关键性临床试验。来源：Atsena Therapeutics关于派真生物