The company’s pipeline leverages adeno-associated virus technology designed to address the challenges of inherited retinal conditions. Credit: 4 PM production/Shutterstock.

Atsena Therapeutics has completed an oversubscribed Series C financing round, raising $150m to support the advancement of its lead gene therapy programme, ATSN-201, to treat X-linked retinoschisis (XLRS).

The funds are to be used to further develop its preclinical pipeline of therapies and broaden the usage of the company’s adeno-associated virus with laterally spreading capsid.

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Bain Capital’s Life Sciences team headed the funding round, with contributions from new investor Wellington Management.

Current investors such as Lightstone Ventures, Abingworth, Foundation Fighting Blindness and Hatteras Venture Partners participated in this latest round.

XLRS is a condition that leads to blindness and is typically diagnosed in childhood.

Atsena’s clinical portfolio has already gained US Food and Drug Administration (FDA) rare paediatric disease and orphan drug designations for both ATSN-101 and ATSN-201.

ATSN-101, which targets Leber Congenital Amaurosis type 1 (LCA1), has also been granted the regenerative medicine advanced therapy designation.

Updated outcomes from the ongoing Phase I/II LIGHTHOUSE trial of ATSN-201 are expected in late 2025.

The company also welcomed biopharmaceutical executive and scientist Dr Norbert Riedel to its board of directors.

For the Series C financing, Wedbush & Co acted as exclusive placement agent to the company, while Cooley provided legal counsel.

Atsena’s investigational gene therapy, ATSN-101, has shown positive outcomes in a Phase I/II trial and is moving towards a pivotal trial in partnership with Nippon Shinyaku.

The company’s pipeline leverages adeno-associated virus technology designed to address the challenges of inherited retinal conditions.

In March 2025, the company received fast track designation from the FDA for ATSN-201 to treat XLRS.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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