FORT COLLINS, Colo. & NEW YORK — Feb. 18, 2026 — Finding Hope for Frizzle, a parent-led nonprofit focused on developing treatments for FRRS1L disorder, has entered a license agreement with Apertura Gene Therapy to support the initiation of a gene therapy clinical trial planned for the second half of 2026, pending regulatory review.
Under the agreement, Finding Hope for Frizzle will license TfR1 CapX, Apertura’s next-generation adeno-associated virus (AAV) capsid engineered for central nervous system (CNS) delivery. The intravenously administered capsid is designed to bind human transferrin receptor 1 (hTfR1), enabling transport across the blood–brain barrier (BBB).
FRRS1L disease—also known as early infantile epileptic encephalopathy type 37 (EIEE-37)—is a rare, recessive neurodevelopmental disorder caused by loss-of-function mutations in the FRRS1L gene. The gene encodes a protein critical for the assembly of AMPA-type glutamate receptors in the brain. Affected children develop early-onset epilepsy, hyperkinetic movement disorders, severe developmental regression, and profound motor impairment. There are currently no disease-modifying treatments.
Preclinical studies in FRRS1L mouse models have demonstrated promising results with AAV-based gene replacement, according to the organization.
TfR1 CapX represents a second-generation capsid platform derived from research led by Ben Deverman, Ph.D., at the Broad Institute. In transgenic mouse models expressing hTfR1, the capsid has demonstrated robust and selective CNS transduction across multiple brain regions.
The planned Phase I/II study aims to evaluate gene replacement therapy delivered using the TfR1 CapX platform, with trial initiation targeted for late 2026.
FRRS1L disorder was first characterized in 2016 and currently affects more than 100 known patients worldwide, though prevalence is expected to rise with expanded genomic testing.
If successful, the program could represent a significant advance not only for FRRS1L patients but also for broader CNS-targeted gene therapy applications.
