AviadoBio Ltd. Licenses Novel AAV Capsid from Apertura Gene Therapy to Advance CNS Gene Therapy Pipeline

29 April 2026 | Wednesday | News

TfR1 CapX™ agreement enhances delivery across the blood-brain barrier, supporting development of AVB-406 for Alzheimer’s disease and other tauopathies using AviadoBio’s RNAi-based vMiX™ platform

AviadoBio Ltd. (“AviadoBio”) and Apertura Gene Therapy (“Apertura”), a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, today announced a licensing agreement for Apertura’s TfR1 CapX™, a novel, intravenously-delivered AAV capsid designed to target human transferrin receptor 1 (hTfR1), cross the blood-brain barrier, and enable broad distribution to the brain and spinal cord.

Under the agreement, AviadoBio will leverage TfR1 CapX to drive the development of its pipeline of genetic medicines, built on AviadoBio’s proprietary vMiX™ RNAi platform. This includes AVB-406, an investigational, preclinical stage gene therapy for Alzheimer’s disease and other tauopathies.

“This agreement with Apertura strengthens a key component of our strategy to develop transformative genetic medicines for neurodegenerative diseases,” said Lisa Deschamps, Chief Executive Officer, AviadoBio. “We believe this agreement enhances our ability to advance AVB-406 and broadens the potential of our vMiX platform in Alzheimer’s disease, other tauopathies, and additional high-need indications.”

TfR1 CapX has demonstrated widespread transduction of neurons and astrocytes throughout the brain and spinal cord, as shown in preclinical testing conducted by AviadoBio and independently by several groups.

“TfR1 CapX has been designed to specifically target human TfR1, the most extensively studied, clinically validated brain shuttle receptor,” said Alex Bloom, Ph.D., Chief Technology Officer, AviadoBio. “Our agreement to license TfR1 CapX enhances our ability to advance our vMiX gene-silencing platform to enable one-time, IV-delivered gene silencing in rare and common disorders with high unmet medical need.”

AviadoBio’s proprietary vMiX platform is designed to enable targeted, durable gene silencing using AAV-delivered RNA interference. By harnessing the natural cellular mechanism of RNA interference, vMiX has the potential for lifelong reduction of disease-causing gene expression following a single administration. The platform is intended to support flexible therapeutic approaches, including the ability to target one or multiple genes, and is being applied across AviadoBio’s pipeline of genetic medicines for neurodegenerative diseases.

“We are thrilled that AviadoBio has selected TfR1 CapX to pair with its vMiX platform, which has shown compelling potential to address the root causes of neurodegenerative diseases,” said Dave Greenwald, Ph.D., Managing Director at Deerfield Management and Executive Chairman of the Board at Apertura Gene Therapy. “We believe this combination has the opportunity to provide transformational benefit to patients in a safe and scalable approach.”

In addition, AviadoBio will present three oral presentations on AVB-406 at the upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting taking place May 11-15, 2026 in Boston. These presentations will highlight preclinical, translational, and manufacturing data from the program, which will leverage TfR1 CapX as part of its ongoing development.

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