Apertura Enters Multiple Agreements to Advance CNS AAV Gene Therapy Programs

NEW YORK, Aug. 13, 2025 /PRNewswire/ — Apertura Gene Therapy has announced a series of new licensing deals, granting Galibra Neuroscience, Emugen Therapeutics, and a third undisclosed company access to its proprietary human transferrin receptor 1 capsid (TfR1 CapX). The technology is designed to deliver gene therapies across the blood-brain barrier (BBB) for treating central nervous system (CNS) disorders.

Key Technology: TfR1 CapX
Apertura’s TfR1 CapX is a specialized adeno-associated virus (AAV) capsid. It is engineered to be administered intravenously and can cross the blood-brain barrier by binding to the human transferrin receptor 1 (TfR1). This mechanism allows for broad distribution of therapeutic payloads throughout the brain, while preclinical data shows high selectivity for the CNS over other tissues, which is a crucial safety advantage.

Partner Applications
Galibra Neuroscience will use the TfR1 CapX for gene therapy targeting GABA-related disorders, including the rare pediatric condition succinic semialdehyde dehydrogenase deficiency (SSADHD). The goal is to correct the genetic root cause of these disorders.

Emugen Therapeutics plans to leverage the technology to advance its therapeutic strategies for neurodevelopmental and neurodegenerative diseases. The company believes TfR1 CapX will help overcome the significant challenge of delivering gene therapy payloads to the brain.

A third, venture-backed company has also entered into an option agreement for multiple CNS indications, reflecting the industry-wide demand for effective, non-invasive delivery methods for CNS gene therapies.