Apertura Gene Therapy, a biotechnology company focused on advanced gene therapy solutions, has announced multiple licensing deals for its innovative human transferrin receptor 1 capsid (TfR1 CapX). The agreements are with Galibra Neuroscience, Emugen Therapeutics, and a third, unnamed biotechnology company, all of which are focused on developing treatments for central nervous system (CNS) disorders.

This groundbreaking technology addresses a major hurdle in gene therapy: the delivery of genetic material to the brain. Most gene therapies struggle to cross the blood-brain barrier (BBB), necessitating invasive procedures like direct brain injections. Apertura's TfR1 CapX overcomes this by enabling a safe, non-invasive, and well-tolerated approach.

Developed from the research of Apertura’s academic founder, Dr. Ben Deverman, TfR1 CapX is an adeno-associated virus (AAV) capsid engineered for systemic, intravenous (IV) dosing. It works by binding to the human transferrin receptor 1 (TfR1) which is a natural mechanism for iron transport into the brain. This "receptor-mediated transcytosis" allows the capsid and its therapeutic payload to efficiently cross the BBB and broadly distribute throughout brain tissues. Extensive preclinical testing has demonstrated the capsid's impressive capabilities and safety profile.

Apertura's licensing of its novel TfR1 CapX delivery platform will significantly empower its partners to advance their gene therapy programs. Specifically, Galibra Neuroscience plans to utilize the capsid for a "brain-wide and systemic" gene replacement therapy to treat rare GABA-related disorders like succinic semialdehyde dehydrogenase deficiency (SSADHD), which has been shown to be reversible through gene therapy. Similarly, Emugen Therapeutics will leverage the technology to overcome the critical delivery challenge for their proprietary payloads aimed at restoring neural function and addressing the root causes of various neurodevelopmental and neurodegenerative diseases. Additionally, a third, undisclosed biotechnology company has entered into an option agreement to explore the use of TfR1 CapX for multiple central nervous system indications. These collaborations demonstrate a shared commitment to accelerating the development of meaningful therapies for patients facing significant unmet medical needs by overcoming a major obstacle in gene therapy delivery.