May 01, 2023

Atsena Therapeutics Receives FDA Clearance of IND Application for ATSN-201, an Investigational Gene Therapy for the Treatment of X-linked Retinoschisis

ATSN-201 leverages AAV capsid that spreads laterally beyond subretinal injection site to facilitate safe delivery of RS1 to photoreceptors in the central retina/fovea

Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for a Phase I/II clinical trial of ATSN-201 in patients with X-linked retinoschisis (XLRS). ATSN-201 leverages one of the company’s novel spreading capsids, AAV.SPR, to overcome the challenges associated with intravitreally delivered AAVs in the treatment of XLRS.

“Intravitreally delivered AAVs have limitations, as they do not drive sufficient gene expression in photoreceptors to confer therapy and can lead to vision-compromising inflammation,” said Shannon Boye, PhD, Founder and Director of Atsena Therapeutics. “AAV.SPR is well-suited for use in XLRS as it can drive therapeutic levels of gene expression in photoreceptors while avoiding the surgical risks of foveal detachment, which is important because XLRS patients have fragile retinas due to the presence of schisis lesions. Building on decades of research, we’re excited to progress our novel gene therapy for patients with XLRS who currently lack an approved treatment option.”

“With the FDA’s clearance of the IND application for ATSN-201, we’re preparing to advance our first program utilizing AAV.SPR into the clinic for the treatment of XLRS in mid-2023,” said Kenji Fujita, Chief Medical Officer of Atsena Therapeutics. “We look forward to evaluating ATSN-201 and addressing the unmet need for a treatment to improve or restore vision in patients with XLRS.”

The Lighthouse Study, a Phase I/II, open-label, dose-escalation clinical trial, will evaluate subretinal injection of ATSN-201 in male patients ages 6-65 with a clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1.

Read the full press release here.

About the Scholar

Ophthalmology, Rare/Orphan

Shannon Boye, PhD

University of Florida
Gund Harrington

More about Shannon Boye