News

Three programs using the capsid are expected to enter clinical trials in the coming year

Last week’s ASGCT Annual Meeting in Boston brought the gene and cell therapy community together for one of the field’s most important yearly gatherings. The meeting was a great time to connect with colleagues and to learn about the latest advances in the field.

It also illustrated how momentum is building around TfR1 CapX™, our 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.

There were 13 oral and poster presentations shared at the conference relating to TfR1 CapX. Together they show how TfR1 CapX is being explored to carry different genetic payloads to the CNS to treat a variety of diseases, including prion disease, a rare genetic disorder, and Alzheimer’s disease.

Presentations from the labs of Sonia Vallabh, Ph.D., Director, Prion Therapeutic Science at the Broad Institute, and Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute and Scientific Founder of Apertura, described the versatility of TfR1 CapX across therapeutic approaches. These included 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. A program in development to treat prion disease that uses TfR1 CapX is expected to enter clinical trials in the first half of 2027.

Apertura partner AviadoBio recently announced that they will be using TfR1 CapX to drive the development of their pipeline of genetic medicines. This includes AVB-406, an investigational, pre-clinical gene therapy for Alzheimer’s disease and other tauopathies. At ASGCT, AviadoBio shared positive pre-clinical data related to the program and announced that they plan to advance AVB-406 into clinical trials for Alzheimer’s disease by the end of this year.

Our Acting Chief Technology Officer Andrew Steinsapir participated in a panel with Apertura partners Finding Hope for Frizzle (FRRS1L) and Viralgen on the topic of accelerating AAV manufacturing for rare diseases. During the panel Viralgen announced that they have completed a 500L clinical scale manufacturing of the drug product that uses TfR1 CapX. Pending regulatory review, Finding Hope for Frizzle plans to initiate a gene therapy clinical trial later this year to treat FRRS1L disease, a rare, recessive genetic disease resulting from loss of function mutations in the FRRS1L gene. The program uses TfR1 CapX to deliver a genetic payload to the CNS. It recently received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA).

Researchers from the University of Texas Southwestern shared data on how TfR1 CapX targets the human transferrin receptor (hTfR1), enabling enhanced and broad transduction of the CNS. (Details of the presentation can be found in abstract 610 here.)

Our own Christopher Davis, Ph.D., Director, Non-clinical and Translational Research, shared findings that emphasize how human-relevant models are critical for predicting clinical translation of novel AAV capsids like TfR1 CapX.

Von Wiltman, Associate Director, Technical Operations, Apertura, presented a poster on our Open Aperture program, which provides academic researchers free access to TfR1 CapX along with a practical development playbook aimed at accelerating discovery and enabling important research across the field of gene therapy.