Atsena Therapeutics Secures $150 Million Oversubscribed Series C Financing to Advance Ocular Gene Therapy Programs

Apr 08, 2025 07:20 CST Updated 07:20

Atsena Therapeutics

Gene Therapy Developer for Blindness Treatment

April 8, 2025

eMedClub News

Atsena Therapeutics（「Atsena」）Atsena Therapeutics, Inc., a clinical-stage gene therapy company focused on developing transformative gene therapies to reverse or prevent blindness, announced on April 2 local time the successful completion of...$150 millionOversubscribed Series C Financing. This round of financing was led by Bain Capital（Bain Capita）The life sciences team led, and another new investor, Wellington Management Company（Wellington Management）Also participated. All of the company's existing investors took part in this round of financing.

Atsena Therapeutics Granted U.S. FDA Fast Track Designation

This financing will be mainly used to advance the development of ATSN-201, one of Atsena Therapeutics' leading projects, which is aimed at treating X-linked retinoschisis.（XLRS）XLRS is a hereditary disease that primarily affects males and is usually diagnosed during childhood. It is characterized by abnormal splitting between retinal layers, progressive vision loss with age, and eventual blindness. The proceeds from this financing will also be used to support Atsena's first-in-class preclinical pipeline and expand the application of Atsena's novel diffusive capsid AAV.SPR.

Patrick RitschelAtsena

Therapeutics CEO

This Series C financing marks a pivotal moment for Atsena Therapeutics. Over the past 12 months, we have achieved significant milestones, including advancing ATSN-101 into Leber Congenital Amaurosis type 1.（LCA1）Global pivotal trial, and the initiation of the ATSN-201 LIGHTHOUSE study（XLRS）PART-B.

Amir Zamani

Bain Capital Partner

New technology and impressive clinical data make us believe that Atsena has the potential to bring meaningful impact to patients with inherited retinal diseases.

Atsena's product pipeline consists of novel adeno-associated viruses（AAV）Technology-driven, this technology aims to overcome the barriers brought by hereditary retinal diseases. To date, Atsena Therapeutics' clinical portfolio has been granted by the U.S. Food and Drug Administration.（FDA）Multiple certifications.

One of the leading projects, ATSN-101, is the first to target Leber Congenital Amaurosis Type 1.（LCA1）The research-based gene therapy has completed the Phase 1/2 trial with positive results. As part of an exclusive strategic partnership with Japan's Shinyaku Co., Ltd., Atsena Therapeutics is advancing the global pivotal trial for ATSN-101. ATSN-101 has received designations for Rare Pediatric Disease, Orphan Drug, and Regenerative Medicine Advanced Therapy.

The protagonist of this financing, ATSN-201, has received Fast Track, Rare Pediatric Disease, and Orphan Drug designations. The latest data from the LIGHTHOUSE I/II study evaluating the clinical efficacy of ATSN-201 is expected to be announced in the second half of the year.

ATSENA's Third Leading Product, Dual AAV Vector Gene Therapy ATSN-301, for the Prevention of MYO7A-Related Usher Syndrome（USH1B）Blindness, USH1B is a hereditary disease that affects the retina and inner ear.

Screenshot 2025-04-03 at 2.41.33 PM.png Atsena's Main Pipeline

Recently, the gene therapy field has been hindered by commercialization struggles, pipeline cuts, and funding reductions. Since 2022, only a few ocular gene therapy companies have managed to secure nine-figure financing, including Beacon, Ray, and Frontera. Atsena Therapeutics last disclosed a $24.5 million Series B financing round in October 2023. This latest financing round for Atsena Therapeutics will undoubtedly serve as a significant boost for the CGT industry.

Ref：https://atsenatx.com

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