Amaurosis Congenita of Leber, Type 1

March 12, 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 Fast Track 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 are pleased that the FDA has granted Fast Track designation to ATSN-201, reinforcing its potential to address the significant unmet need in XLRS, a rare inherited retinal disease with no approved treatments,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “This designation, along with the previously granted Orphan Drug and Rare Pediatric Disease designations, marks an important milestone in advancing the development of ATSN-201. The Atsena team remains dedicated to developing transformative gene therapies and improving the quality of life of individuals suffering from XLRS and other inherited retinal diseases.” Fast Track designation is granted to treatments intended to address serious or life-threatening diseases that have demonstrated the potential to meet an unmet medical need. Treatments that receive Fast Track designation may benefit from more frequent interactions with the FDA throughout drug development. In addition, the Fast Track program allows for Priority Review, if relevant criteria are met. 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. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). 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. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/. 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), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi-org.libproxy1.nus.edu.sg/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.

Dec. 16, 2024 -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced dosing has been completed in Part A of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of 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. “Nine adults have been treated in Part A of the study in which three dose levels of ATSN-201 are being evaluated for safety and efficacy. We have seen both structural and functional benefits in patients treated at all dose levels. Additionally, no serious adverse events related to treatment have been reported,” said Kenji Fujita, MD, Atsena’s Chief Medical Officer. “Collecting data from Part A of the trial is an important breakthrough for Atsena because it validates the use of our novel capsid to effectively treat inherited retinal disease and it informs the safest and most effective path forward for Part B. We look forward to providing further updates from those patients early next year.” Currently, there are no approved treatments for XLRS which is typically diagnosed in early childhood and primarily affects males. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and ultimately leads to blindness. Approximately 30,000 males in the U.S. and EU have this inherited retinal disease. “We are thrilled that dosing is complete in Part A of the LIGHTHOUSE study for ATSN-201 and that we’ve begun hearing positive anecdotes from those involved. This marks a significant milestone for patients with XLRS as we push to bring a therapeutic option to individuals that otherwise have no approved treatment,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “It also represents potential for individuals living with other inherited retinal diseases that could benefit from our novel capsid. We look forward to the insights gained from this trial as we pioneer new approaches to ocular gene therapy.” The LIGHTHOUSE study is a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial evaluating the safety and tolerability of ATSN-201 in male patients ages six and older with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. 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, 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 stark 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 does not cause inflammation. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/. Atsena Therapeutics 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 program for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children, has completed a Phase 1 / 2 trial with positive results (https://doi-org.libproxy1.nus.edu.sg/10.1016/s0140-6736(24)01447-8) and is moving towards initiation of a pivotal trial. 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/. The content above comes from the network. if any infringement, please contact us to modify.

DURHAM, N.C., Oct. 10, 2024 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced that safety and efficacy data on ATSN-201 for the treatment of X-linked retinoschisis (XLRS) will be presented during a panel discussion at the American Academy of Ophthalmology (AAO) 2024 Annual Meeting taking place October 18-21, 2024, in Chicago. 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. Presentation details are as follows: Session: First-time Results of Clinical TrialsDate and Time: Friday, October 18, 2024, 4:54 - 5:33 p.m. CDTPresentation Title: Interim Safety and Efficacy of ATSN-201 Dose Escalation Study in Patients With X-linked Retinoschisis (XLRS)Presentation Time: 5:08 p.m. CDTPresenter: Christine Nichols Kay, MD, Clinical Ophthalmology Advisor, Atsena Therapeutics The safety and tolerability of ATSN-201 is being evaluated in the LIGHTHOUSE study, a Phase I/II, open-label, 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. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). ATSN-201 has received Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration. 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 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 bleb margins 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 bleb margins. This is in stark contrast to benchmark AAV vectors, which remain confined to those original margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and does not cause inflammation. 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 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 affecting boys and men that is typically diagnosed in childhood and leads to blindness later in life. Another ongoing Phase I/II clinical trial is evaluating ATSN-101 for Leber congenital amaurosis type 1 (LCA1), one of the most common causes of blindness in children. 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:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com Business Contact:info@atsenatx.com