Apertura Gene Therapy, a biotechnology company focused on innovative gene therapy solutions announced several licensing deals with Galibra Neuroscience and Emugen Therapeutics to utilize Apertura’s human transferrin receptor 1 capsid (TfR1 CapX) for their central nervous system (CNS) 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. 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 recognize the need for more potent and less invasive capsids targeting the CNS.” said Dr. Diego Garzón, Apertura’s Vice President of Corporate Development.
Apertura’s TfR1 CapX builds on the work from the company’s academic founder, Ben Deverman1, Senior Director of Vector Engineering at The Broad Institute of Harvard and MIT. TfR1 CapX is an intravenously (IV) dosed AAV capsid that binds to the human transferrin receptor, TfR1, to cross the blood-brain barrier and broadly transduce tissues in the brain. In extensive preclinical testing conducted independently by several groups, TfR1 CapX has demonstrated a selectivity to the CNS compared to other tissues with more than 50% neuron transduction and 90% astrocyte transduction across different brain regions.
Galibra Neuroscience will utilize TfR1 CapX for GABA-related disorders, which is the major brain inhibitory neurotransmitter keeping neural communication in check and preventing over-excitation. GABA levels in healthy individuals are highly regulated with over 20 genes responsible for regulating GABA signaling. Variants of these genes lead to GABA imbalance, the root cause of several rare pediatric disorders. “Our work at Boston Children’s Hospital demonstrated that the symptoms of a rare GABA disorder, succinic semialdehyde dehydrogenase deficiency (SSADHD) are reversible by gene replacement therapy.” said Dr. Henry Lee, MPhil, PhD, Co-Founder of Galibra Neuroscience, Inc. “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.’ said Alexander Rotenberg, MD PhD, Professor of Neurology at Boston Children’s Hospital and Harvard Medical School and Co-Founder of Galibra Neuroscience, Inc.
Emugen Therapeutics is developing novel gene therapy solutions to address neurodevelopmental and neurodegenerative diseases. “We are advancing therapeutic strategies that target the root causes of complex neurological disorders,” said Dr. Tyler Brown, Emugen’s Chief Operating Officer. “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.”
Apertura has also entered into an option and licensing agreement with an undisclosed biotechnology company to explore the use of TfR1 CapX with their proprietary payloads targeting CNS disorders. Specific terms of the agreement are not disclosed at this time. “Across the industry, partners are looking for capsids that not only reach the CNS effectively but also support safe, systemic delivery without triggering adverse immune responses,” said Andrew Steinsapir, Director, Gene Therapy Program Lead at Deerfield Management and Acting CTO of Apertura. “TfR1 CapX was designed with these uses in mind. We’re proud to partner with companies large and small who utilize a gene therapy approach, recognize the potential ofTfR1 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.”
