Apertura Gene Therapy Licences Blood-Brain Barrier-Penetrant AAV Capsid for CNS Treatments
Thursday, August 14, 2025
Apertura Gene Therapy has signed multiple licensing agreements to advance the use of its blood-brain barrier-penetrant adeno-associated virus (AAV) capsid technology in treatments for central nervous system (CNS) disorders.
The company’s human transferrin receptor 1 capsid (TfR1 CapX) will be used by Galibra Neuroscience and Emugen Therapeutics for their CNS-focused gene therapy programmes. A third biotechnology company, backed by venture capital, has entered into an option agreement for the technology to explore multiple CNS indications.
TfR1 CapX, developed from research by Ben Deverman at The Broad Institute of Harvard and MIT, is an intravenously delivered AAV capsid that binds to the human transferrin receptor (TfR1) to cross the blood-brain barrier. Preclinical studies by independent groups have shown high selectivity for CNS tissues, with over 50% neuron transduction and 90% astrocyte transduction across various brain regions, while limiting activity in other tissues.
Galibra Neuroscience will apply TfR1 CapX to address GABA-related disorders, a group of conditions linked to imbalances in the brain’s primary inhibitory neurotransmitter. Variants in genes regulating GABA signalling are known to cause several rare paediatric disorders. The company aims to deliver a systemic and brain-wide gene replacement approach using its proprietary therapeutic payload.
Emugen Therapeutics will utilise TfR1 CapX in the development of treatments for neurodevelopmental and neurodegenerative diseases. The technology is expected to address a major barrier in gene therapy by enabling non-invasive delivery across the blood-brain barrier, supporting the restoration of neural function.
The undisclosed biotechnology company will explore the use of TfR1 CapX with its own therapeutic payloads for CNS disorders under the option and licensing agreement.
Apertura stated that these collaborations reflect a growing industry demand for capsids capable of safe, systemic CNS delivery without triggering adverse immune responses.
The partnerships aim to accelerate the development of effective therapies for patients with high unmet medical needs.
Source: aperturagtx.com
