Apertura Gene Therapy has entered several licensing agreements to advance central nervous system (CNS) treatments using its transferrin receptor 1 capsid (TfR1 CapX) technology.

The deals were signed with Galibra Neuroscience and Emugen Therapeutics, alongside an option agreement with a third, undisclosed biotech company.

TfR1 CapX is an intravenously administered adeno-associated virus (AAV) capsid that targets the human transferrin receptor (TfR1). This enables it to cross the blood-brain barrier and facilitate widespread transduction of brain tissues.

Apertura corporate development vice-president Dr Diego Garzón stated: “Genetic therapy medicines are often limited by delivery challenges, particularly to crucial areas like the CNS, creating an urgent need for advanced capsids that can effectively access these tissues.

“Not only is it important to target the CNS, but equally important to avoid other tissues to avoid triggering adverse events. We believe Apertura’s TfR1 CapX is the best option of a capsid with a promising efficacy and safety profile. Apertura is excited about TfR1 CapX because we recognise the need for more potent and less invasive capsids targeting the CNS.”

Galibra Neuroscience will use this technology for disorders related to gamma-aminobutyric acid (GABA), a crucial neurotransmitter in the brain that prevents neural over-excitation.

In healthy individuals, GABA levels are regulated by more than 20 genes that govern GABA signalling. Variations in these genes can result in GABA imbalances, which are the underlying cause of several rare paediatric disorders.

Emugen Therapeutics aims to develop gene therapies targeting neurodevelopmental and neurodegenerative diseases.

Emugen chief operating officer Dr Tyler Brown stated: “We are advancing therapeutic strategies that target the root causes of complex neurological disorders.

“TfR1 CapX enables us to overcome one of gene therapy’s key challenges — delivering payloads across the blood-brain barrier — by providing a non-invasive capsid platform. We are excited to leverage this technology alongside our transformative gene therapy payloads aimed at restoring neural function and improving outcomes for patients with high unmet medical need.”

The undisclosed biotech company's option agreement with Apertura will investigate the potential of TfR1 CapX in conjunction with its payloads aimed at treating CNS disorders.

The specific terms of the agreement remain confidential.