Durham-based Atsena Therapeutics has successfully closed an oversubscribed $150 million Series C financing round to advance its pipeline of gene therapies aimed at preventing or reversing inherited forms of blindness. The funding was led by Bain Capital's Life Sciences team, with participation from new investor Wellington Management and all existing investors including Lightstone Ventures, Sofinnova Investments, and Hatteras Venture Partners.
The significant capital infusion comes after a year of notable achievements for the clinical-stage gene therapy company, positioning it to accelerate development of multiple programs targeting serious ocular diseases.
Advancing Treatment for X-linked Retinoschisis
A substantial portion of the proceeds will support advancement of Atsena's lead program, ATSN-201, which targets X-linked retinoschisis (XLRS). This genetic retinal disorder is typically diagnosed in childhood and progressively leads to vision loss and eventual blindness later in life.
The company recently reported positive preliminary data from the first cohort of its Phase I/II LIGHTHOUSE clinical trial evaluating ATSN-201 and has initiated a new component of the study. The U.S. Food and Drug Administration (FDA) has granted ATSN-201 multiple designations, including Rare Pediatric Disease, Orphan Drug, and Fast Track status, potentially expediting its development pathway.
"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. "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."
Strategic Partnership for LCA1 Program
Beyond XLRS, Atsena has secured a strategic partnership with Nippon Shinyaku Co. of Kyoto, Japan, to advance ATSN-101, a gene therapy for Leber Congenital Amaurosis type 1 (LCA1). LCA1 is an inherited retinal disease that represents one of the more common causes of blindness in children.
Under the agreement, Nippon Shinyaku will be responsible for commercializing ATSN-101 in the United States and Japan, while Atsena retains rights in the rest of the world. The therapy has already completed a Phase I/II trial with positive results and is advancing toward a global pivotal trial.
ATSN-101 has received multiple FDA designations, including Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy designation, highlighting its potential significance for patients with this serious condition.
Novel AAV Technology Platform
Atsena's product pipeline is built on innovative adeno-associated virus (AAV) technology that uses altered viruses to deliver corrective genes into cells. The company's approach is designed to overcome limitations of current gene therapies and address significant unmet needs in inherited retinal diseases.
"Our technologies are uniquely suited for the prevention or reversal of blindness," according to the company's website. "We utilize an AAV capsid, payload and delivery approach that is engineered for each disease, and our AAV vectors are designed for delivery via subretinal or intravitreal injection."
The financing will also support expansion of Atsena's novel spreading AAV.SPR capsid technology, which could potentially enhance the delivery and efficacy of gene therapies for ocular conditions.
Expanding Pipeline and Team
In addition to ATSN-101 and ATSN-201, Atsena is developing ATSN-301, a gene therapy designed to prevent blindness caused by a type of Usher syndrome, an inherited disease affecting both the retina and inner ear.
"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."
With approximately 20 full-time employees currently, Atsena plans to expand its team in the near future, primarily adding clinical roles and positions in chemistry, manufacturing, and controls, according to CEO Ritschel.
The company also announced that Norbert Riedel, PhD, a seasoned scientist and biopharmaceutical executive, will join Atsena's Board of Directors in conjunction with the financing.
Market Context and Unmet Need
Inherited retinal diseases represent a significant unmet medical need, with limited treatment options currently available. These genetic conditions often lead to progressive vision loss and blindness, substantially impacting patients' quality of life from an early age.
Gene therapy approaches like those being developed by Atsena offer the potential to address the underlying genetic causes of these diseases rather than just managing symptoms, potentially preserving or restoring vision for affected individuals.
The substantial investment in Atsena reflects growing confidence in gene therapy approaches for ocular diseases, an area that has seen increasing interest from both investors and pharmaceutical companies in recent years.
