Atsena Therapeutics raises $150M for ocular gene therapy programs

By Barry Teater | This article originally appeared on NCBiotech's blog

Durham-based Atsena Therapeutics has raised $150 million to advance gene therapies for preventing or reversing types of inherited blindness.

Proceeds from the Series C financing will be used to advance the company’s lead program, ATSN-201, for the treatment of X-linked retinoschisis (XLRS), a genetic retinal disorder typically diagnosed in childhood that 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 its novel gene therapy platform.

“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. “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.”

The oversubscribed financing was led by Bain Capital’s Life Sciences team, with participation from an additional new investor, Wellington Management. All of the company’s existing investors also participated in the round, including Lightstone Ventures, Sofinnova Investments, Abingworth, Foundation Fighting Blindness, Durham-based Hatteras Venture Partners, Osage University Partners and the Manning Family Foundation.

The financing follows several achievements by Atsena over the last 12 months, including:

• Reporting positive preliminary data from the first cohort of a Phase I/II clinical trial evaluating ATSN-201 for XLRS and initiating a new component of the study
• Receiving Rare Pediatric Disease, Orphan Drug and Fast Track designations for ATSN-201 from the U.S. Food and Drug Administration
• Securing a partner, Nippon Shinyaku Co. of Kyoto, Japan, to advance another gene therapy, ATSN-101, to a global pivotal trial for Leber Congenital Amaurosis type 1 (LCA1), an inherited retinal disease that is one of the more common causes of blindness in children

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.

In addition to ATSN-101 and ATSN-201, Atsena is developing a third gene therapy, ATSN-301, to prevent blindness caused by a type of Usher syndrome, an inherited disease that affects 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.”

Atsena’s product pipeline is based on novel adeno-associated virus (AAV) technology that uses altered viruses to deliver corrective genes into cells. The technology is designed to overcome the limitations of current gene therapies and address the significant unmet need in inherited retinal diseases.

“Our technologies are uniquely suited for the prevention or reversal of blindness,” the company’s website says. “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.”

Atsena has about 20 full-time employees. With the new financing, the company will hire more staff “in the near future,” mostly in clinical roles and jobs in chemistry, manufacturing and controls, Ritschel said.