US-based biotechnology company Apertura Gene Therapy has signed several licensing agreements with Galibra Neuroscience and Emugen Therapeutics.
Under the agreements, Galibra and Emugen will use Apertura’s advanced transferrin receptor 1 capsid (TfR1 CapX) for their central nervous system (CNS) treatments.
The TfR1 CapX is a novel AAV capsid that binds to the human transferrin receptor, allowing it to cross the blood-brain barrier and effectively target brain tissues.
In preclinical tests, the AAV capsid has shown high selectivity for the CNS, with over 50% neuron transduction and 90% astrocyte transduction.
The TfR1 CapX enables broad brain distribution through intravenous dosing, addressing the need for a non-invasive gene therapy solution.
Apertura corporate development vice president Diego Garzón said: “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, a gene therapy start-up, aims to use the TfR1 CapX for treating gamma-aminobutyric acid (GABA)-related disorders.
The disorders stem from imbalances in GABA, a key neurotransmitter responsible for regulating neural communication.
Variants in over 20 genes can disrupt GABA signalling, leading to various rare paediatric conditions.
Galibra Neuroscience co-founder Alexander Rotenberg said: “Galibra will use Apertura’s TfR1 CapX with our proprietary payload for a brain-wide and systemic gene replacement approach.
“We are excited to use the Apertura technology to safely deliver critical genetic materials to desired cellular targets, resulting in highly effective and safe therapeutic options for patients in need. We look forward to our productive collaboration.”
Emugen Therapeutics will develop gene therapy solutions for neurodevelopmental and neurodegenerative diseases using the TfR1 CapX.
Emugen Therapeutics chief operating officer Tyler Brown said: “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.”
In addition to Galibra and Emugen, Apertura has signed an option agreement with another biotechnology company, keeping specific terms of the deal undisclosed.
The collaboration aims to explore the use of TfR1 CapX with proprietary payloads targeting CNS disorders.
Apertura director and gene therapy program lead Andrew Steinsapir said: “TfR1 CapX was designed with these uses in mind.
“We’re proud to partner with companies large and small who utilise a gene therapy approach, recognise the potential of TfR1 CapX, and select it as the foundation for their CNS gene therapy strategies.
“These collaborations reflect our shared commitment to overcoming delivery challenges and accelerating meaningful therapies for patients.”
