GUCY2D

Financing led by new investor Bain Capital with participation from new investor Wellington Management and all existing investors Proceeds to support advancement of ATSN-201 through potential approval and launch as well as preclinical programs to treat inherited retinal diseases Norbert Riedel, PhD, will join Atsena’s Board of Directors DURHAM, N.C., April 02, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics (“Atsena” or “the Company”), a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the successful close of an oversubscribed $150 million Series C financing. The financing was led by Bain Capital’s Life Sciences team, with participation from an additional new investor, Wellington Management. All the Company’s existing investors also participated in the round, including Lightstone Ventures, Sofinnova Investments, Abingworth, Foundation Fighting Blindness, Hatteras Venture Partners, Osage University Partners, and the Manning Family Foundation. Proceeds from the financing will be used to advance Atsena’s lead program, ATSN-201, for the treatment of X-linked retinoschisis (XLRS), a genetic condition that is typically diagnosed in childhood and leads to blindness later in life. The proceeds will also support Atsena’s preclinical pipeline of first-in-class therapies and expand the use of Atsena’s novel spreading AAV.SPR capsid. “Closing our Series C marks a pivotal moment for Atsena as we advance our transformative ocular gene therapies and fuel our next phase of growth, innovation, and clinical progress,” said Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics. “It follows a productive 12 months of key achievements including securing a partner to advance ATSN-101 to a global pivotal trial for Leber Congenital Amaurosis type 1 (LCA1) and initiating Part B of the ATSN-201 LIGHTHOUSE study for XLRS. We’re grateful for the support of our investors and partners who share our vision for the future of leveraging genetic medicine to reverse or prevent blindness.” To date, Atsena’s clinical portfolio has received multiple designations by the U.S. Food and Drug Administration (FDA). ATSN-101, for the treatment of LCA1, has received Rare Pediatric Disease designation, Orphan Drug Designation, and Regenerative Medicine Advanced Therapy designation. ATSN-201 has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug Designations. Updated data from the ongoing LIGHTHOUSE Phase I/II clinical trial evaluating ATSN-201 is anticipated later this year. “We believe Atsena has a unique opportunity to deliver meaningful impact for patients with inherited retinal diseases on the basis of novel science and impressive clinical data generated to date,” said Amir Zamani, a Partner at Bain Capital. “We look forward to supporting Patrick and his strong team as they look to unlock the next phase of Atsena’s growth and innovation while thoughtfully advancing potentially groundbreaking therapies toward patients in need.” In conjunction with the financing, Norbert Riedel, PhD, a seasoned scientist and biopharmaceutical executive, will join Atsena’s Board of Directors. Wedbush & Co. served as exclusive placement agent to Atsena for the Series C financing, Cooley LLP acted as its legal advisor. 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.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. For more information, please visit https://atsenatx.com/. Media Contact:Gina Mangiaracina6 Degrees(917) 797-7904gmangiaracina@6degreespr.com Business Contact:info@atsenatx.com

Nov. 13, 2024 -- Nippon Shinyaku Co., Ltd. (Nippon Shinyaku; Headquarters: Kyoto; President, Toru Nakai) and Atsena Therapeutics, Inc. (Atsena; Headquarters: Durham, North Carolina, USA, Chief Executive Officer (CEO): Patrick Ritschel) have entered into an exclusive license agreement for the commercialization of ATSN-101 in the territory of the U.S. and for the development and commercialization of ATSN-101 in the territory of Japan for advancing Atsena’s first-in-class, investigational gene therapy ATSN-101 for Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). Under the terms of the licensing agreement, Nippon Shinyaku will receive exclusive commercial rights in the U.S. and Japan, and Atsena will retain commercial rights in the rest of the world. ATSN-101 will be marketed by NS Pharma, Inc. (New Jersey, USA, President: Yukiteru Sugiyama), a wholly owned subsidiary of Nippon Shinyaku in the U.S. Atsena will receive an upfront payment, additional milestone payments, downstream royalties based on sales and will be reimbursed as it continues development work on ATSN-101, including an anticipated global pivotal trial. ATSN-101 is a first-in-class, investigational gene therapy for the treatment of LCA1. Atsena has received Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy Designation and Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for ATSN-101. In the event Atsena receives a Rare Pediatric Disease Priority Review Voucher (PRV) in connection with the approval of the Biologic License Application for ATSN-101, Atsena shall own and retain all rights, title and interest in such PRV. “ATSN-101 provides a potential, innovative treatment in an area where no approved solutions currently exist,” said Nippon Shinyaku President Toru Nakai. “We are excited by the opportunity of this novel ocular gene therapy and our collaboration with Atsena and its groundbreaking science.” “This collaboration creates a path to accelerate the development of ATSN-101 and validates Atsena’s pioneering technology and development capabilities. We anticipate this will be the first of many ocular gene therapy treatments from our clinical portfolio to come,” said Patrick Ritschel, CEO of Atsena Therapeutics. “We look forward to working with Nippon Shinyaku as we advance ATSN-101 into a pivotal trial and potential approval to provide an innovative solution to patients and families affected by LCA1 around the world.” LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1 is one of the most common forms of LCA and there are no approved treatments for it. ATSN-101 is an investigational, subretinal AAV5 gene therapy being evaluated in an ongoing Phase I/II clinical trial for LCA1. At 12 months post-treatment, ATSN-101 has demonstrated durable, clinically meaningful improvements in vision at the high dose and is well-tolerated. Results from this trial were recently published in The Lancet. The Rare Pediatric Disease Designation is granted by the FDA for promoting the development of new drugs for rare diseases that occur in the U.S. before the age of 18 and affect fewer than 200,000 people. With this designation, the product will be eligible to receive a Priority Review Voucher upon approval that could be used to advance another program. The Regenerative Medicine Advanced Therapy (RMAT) Designation is an FDA-designated system for advanced regenerative medicine therapies targeted at developing serious diseases that have shown certain effect in clinical trials based on the 21st Century Cures Act in the U.S. Companies with RMAT designations are given the opportunity for priority review and accelerated approval for the product. The Orphan Drug Designation is provided for orphan drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. With orphan designation, the FDA grants a seven-year market exclusivity for the product and provides certain incentives such as tax credits towards the cost of development, upon approval. 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. In an ongoing Phase I/II clinical trial, the company is evaluating ATSN-101 for LCA1, one of the most common causes of blindness in children. In another ongoing clinical trial, the company is evaluating ATSN-201 for X-linked retinoschisis (XLRS), a genetic condition affecting boys and men that is typically diagnosed in childhood. 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/. Based on Nippon Shinyaku’s business philosophy, “Helping people lead healthier, happier lives,” we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. The content above comes from the network. if any infringement, please contact us to modify.

FRIDAY, Sept. 6, 2024 -- Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report. The illness is caused by mutations in the GUCY2D gene, which is critical to producing proteins that enable vision. People with this form of Leber congenital amaurosis (LCA1) typically lose their vision in early childhood. Just under 100,000 people are thought to be affected worldwide. However, a team of researchers at the University of Pennsylvania say that a new gene therapy injected under the retinas of children and adults with LCA1 improved vision by 100 to 10,000 times. “Even though we previously predicted a large vision improvement potential in LCA1, we did not know how receptive patients’ [eye] photoreceptors would be to treatment after decades of blindness,” noted study co-lead author Artur Cideciyan , a research professor of ophthalmology at the university. “It is very satisfying to see a successful multi-center trial that shows gene therapy can be dramatically efficacious.” The findings were published Sept. 5 in The Lancet journal. A total of 15 LCA1 patients -- 12 adults and three children -- participated in the new trial. All had vision that was equal to or below 20/80, meaning that objects a normally sighted person could see at 80 feet could only be clear at 20 feet away for a person with LCA1. Glasses were of little help, because glasses only address the eye's focusing ability, not any underlying medical cause driving vision loss. The new gene therapy is called ATSN-101 and was developed by Atsena Therapeutics, which funded the new trial. This was a small phase 2 trial, meant to gauge a treatment's safety and explore differences when various dosages are used. The injected therapy was tried at three different dosages. Three adult patients each received low, medium and high doses of ATSN-101. Once the treatments' safety was assessed, six participants (including the three children) received high-dose ATSN-101. According to the research team, vision began to improve quickly in all patients, usually within a month of treatment. Improvements have lasted for at least a year. Patients' vision was tested in various ways, including their ability to move through a sometimes dimly-lit pathway, eye charts and "light flash" tests conducted in a dark environment. Two of nine patients who got the high-dose treatment had a 10,000-fold improvement in their sight, Cideciyan's group reported. “That 10,000-fold improvement is the same as a patient being able to see their surroundings on a moonlit night outdoors as opposed to requiring bright indoor lighting before treatment,” said Cideciyan, who also co-directs the Center for Hereditary Retinal Degenerations at UPenn. “One patient reported for the first time being able to navigate at midnight outdoors only with the light of a bonfire.” Side effects could happen, and were usually related to the surgery used to implant the gene therapy. These included tiny hemorrhages within the eye that later healed, or transient inflammation of the eye, treated with steroids. The patients' vision is being tracked going forward, the researchers said, and a bigger, randomized trial is being planned as a requirement of any U.S. Food and Drug Administration approval of the new therapy. The new work follows on the team's prior success using gene therapy to reverse another form of Leber congenital amaurosis, this time connected to a mutation in the CEP290 gene. “The treatment success in our most recent clinical trials together with our earlier experience brings hope for a viable treatment for about 20 percent of infantile blindness caused by inherited retinal degenerations,” study co-author Dr. Tomas Aleman said in a UPenn news release. He is research professor in ophthalmology and co-director of the Center for Hereditary Retinal Degenerations. “The focus now is on perfecting the treatments and treating earlier manifestations of these conditions once safety is confirmed," he said. "We hope similar approaches will lead to equally positive outcomes in other forms of congenital retinal blindness.” Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.