AviadoBio Licenses Apertura's TfR1 CapX for CNS Gene Therapies

AviadoBio Ltd. and Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, 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, including AVB-406, an investigational, preclinical stage gene therapy for Alzheimer's disease and other tauopathies.

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. AviadoBio's vMiX platform is designed to enable targeted, durable gene silencing using AAV-delivered RNA interference, with 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.

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

Apertura, along with scientists from the Broad Institute of MIT and Harvard, will also present advancements related to TfR1 CapX at the 29th Annual Meeting of ASGCT. Data presented by Apertura show that TfR1 CapX can be manufactured at clinically relevant scale, helping reduce one of the biggest bottlenecks in bringing therapies to patients. Apertura will also present on the utility of different animal models for preclinical evaluation of novel AAV capsids, emphasizing that more human-relevant models are critical for more accurately predicting clinical translation. The company will share how it is expanding access to TfR1 CapX through its Open Aperture program, which provides academic researchers free access and a practical development playbook.

Presentations from the labs of scientists at the Broad Institute will highlight the versatility of TfR1 CapX across therapeutic approaches, including epigenetic silencing and base-editing strategies designed to reduce prion protein levels in the brain and advances in capsid engineering that help evade neutralizing antibodies, potentially enabling gene therapies to reach a broader patient population.

Apertura Gene Therapy has also announced its participation in a consortium selected to receive funding from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE program to develop gene therapies for rare childhood diseases. The company will license a novel AAV capsid to Finding Hope for Frizzle to support the organization's work to advance a gene therapy for FRRS1L disease.