Apertura Gene Therapy Enters into Three Licensing Deals for Its Blood-Brain Barrier-Penetrant AAV Capsid

Rare Daily Staff

Apertura Gene Therapy said it has entered into licensing deals with Galibra Neuroscience and Emugen Therapeutics to utilize Apertura’s human transferrin receptor 1 (TfR1) capsid for their central nervous system programs.

A third venture-backed biotechnology company has entered into an option agreement with Apertura for multiple CNS indications.

“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,” said Diego Garzón, vice president of corporate development for Apertura. “Not only is it important to target the CNS, but it is equally important to avoid other tissues to prevent triggering adverse events.”

Apertura’s TfR1 CapX builds on the work of the company’s academic founder, Ben Deverman, senior director of vector engineering at the Broad Institute of Harvard and MIT. TfR1 CapX is an intravenously dosed adeno-associated virus (AAV) capsid that binds to the human transferrin receptor (TfR1) to cross the blood-brain barrier and broadly transduce tissues in the brain.

In preclinical testing conducted independently by several groups, TfR1 CapX demonstrated selectivity for the CNS compared to other tissues, with more than 50 percent neuron transduction and 90 percent astrocyte transduction across different brain regions.

Galibra Neuroscience will utilize TfR1 CapX for GABA-related disorders. Gamma-aminobutyric acid (GABA) is the brain’s major inhibitory neurotransmitter, which keeps neural communication in check and prevents overexcitation. GABA levels in healthy individuals are tightly regulated, with over 20 genes responsible for controlling GABA signaling. Variants of these genes can lead to GABA imbalances, the root cause of several rare pediatric disorders.

Emugen Therapeutics is developing novel gene therapy solutions to address neurodevelopmental and neurodegenerative diseases.
Apertura has also entered into an option and licensing agreement with an undisclosed biotechnology company to explore the use of TfR1 CapX with its proprietary payloads targeting CNS disorders. Specific terms of the agreement have not been disclosed.

Apertura has also entered into an option and licensing agreement with an undisclosed biotechnology company to explore the use of TfR1 CapX with its proprietary payloads targeting CNS disorders. Specific terms of the agreement have not been disclosed.