Press Releases
Press Releases

Atsena Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)

RMAT designation recognizes the potential of ATSN-101 to address unmet medical needs for patients with LCA1

ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post-treatment in ongoing Phase I/II clinical trial

DURHAM, NC, November 14, 2023 – 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 granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.

“Receiving RMAT designation from the FDA for ATSN-101 marks a significant regulatory milestone for Atsena, validating the potential of our subretinal gene therapy to improve vision and make a meaningful difference in the lives of patients with LCA1,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics. “As we continue to explore options to advance ATSN-101 into a pivotal clinical trial, we look forward to reporting 12-month data from our ongoing Phase I/II trial by the end of this year.”

“There are no approved treatments for LCA1, an inherited retinal disease that results in early and profound vision impairment or blindness,” said Jason Menzo, Chief Executive Officer of Foundation Fighting Blindness. “RMAT designation is encouraging recognition of the potential of ATSN-101 to be an important treatment and provides hope to children and adults affected by LCA1.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for that disease or condition. RMAT designation provides sponsors with intensive FDA guidance on efficient drug development, including the ability to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review.

Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.

About GUCY2D-associated Leber congenital amaurosis (LCA1)
LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.

About Atsena Therapeutics
Atsena Therapeutics is a clinical-stage gene therapy company developing novel treatments for inherited forms of blindness. The company has two clinical-stage programs, ATSN-201 for X-linked retinoschisis (XLRS) and ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which leverages the company’s novel spreading capsid AAV.SPR, is being evaluated in XLRS patients in a Phase I/II clinical trial known as the LIGHTHOUSE study. The company’s additional proprietary asset is ATSN-301, a dual AAV vector-based gene therapy to prevent blindness from MYO7A-associated Usher syndrome (USH1B). Interim safety and efficacy data from the company’s ongoing Phase I/II clinical trial in patients with LCA1 have demonstrated ATSN-101 is well tolerated and clinically meaningful improvements in vision were observed 6 months post-treatment. Founded by ocular gene therapy pioneers Dr. Shannon Boye and Sanford Boye of the University of Florida, Atsena is based in North Carolina’s Research Triangle, an environment rich in gene therapy expertise. For more information, please visit

Media Contact:
Tony Plohoros
6 Degrees
(908) 591-2839
[email protected]

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