Atsena Therapeutics Announces FDA RMAT Designation for ATSN-101 Gene Therapy in LCA1

▎WuXi

Edited by Kant Content Team

Atsena Therapeutics today announced that the U.S. FDA has granted its investigational gene therapy ATSN-101 the Regenerative Medicine Advanced Therapy (RMAT) designation.This RMAT designation is primarily based on the positive efficacy data from the six-month Phase 1/2 clinical trial of the therapy.

Data released at the end of April this year showed that subretinal administration of ATSN-101 was well-tolerated, with no serious adverse events related to the drug.Patients receiving the highest dose treatment showed clinically meaningful improvements in visual functions such as retinal sensitivity and BCVA after 6 months of treatment, with BCVA improvement greater than 0.3 log MAR, while the BCVA of untreated eyes declined.

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Efficacy Results of ATSN-101 Clinical Trial (Image Source: Reference [3])

In terms of safety, no serious adverse events related to the drug were reported, and most adverse events that occurred during treatment were mild and transient.

ATSN-101 (SAR439483) is a drug developed by Atsena Therapeutics for

Gene Therapy in Development for Leber Congenital Amaurosis (LCA1) Caused by GUCY2D Biallelic Mutations

LCA1 is a type of

Monogenic eye diseases caused by GUCY2D gene mutations can disrupt retinal function, leading to early and severe visual impairment or blindness. There are currently no approved treatments for LCA1.

References:

[1] Atsena Therapeutics Receives FDA Regenerative MedicineAdvanced Therapy (RMAT) Designation for ATSN-101 Gene Therapy forGUCY2D-associated Leber Congenital Amaurosis (LCA1). Retrieved November 14,2023 from https://atsenatx.com/press-release/atsena-therapeutics-receives-fda-regenerative-medicine-advanced-therapyrmat-designation-for-atsn-101-gene-therapy-for-gucy2d-associated-leber-congenital-amaurosislca1/

[2] Atsena Therapeutics Announces Positive 6-month Data fromOngoing Phase I/II Clinical Trial of ATSN-101 in Patients with Leber CongenitalAmaurosis Caused by Biallelic Mutations in GUCY2D (LCA1). Retrieved November14, 2023 from https://atsenatx.com/press-release/atsena-therapeutics-announces-positive-6-month-data-from-ongoing-phase-i-ii-clinical-trial-of-atsn-101-in-patients-with-leber-congenital-amaurosis-caused-by-biallelic-mutations-in-gucy2d-lca1/

[3] Safety and Efficacy of ATSN-101 in Patients with Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D (LCA1). Retrieved November 14, 2023 from https://atsenatx.com/wp-content/uploads/2022/10/Atsena-AAO-Final.pdf

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