US EditionApertura Gene Therapy Showcases the Progress of its Novel Human Transferrin Receptor AAV Capsid, TfR1 CapX, and Preclinical Programs for Tuberous Sclerosis Complex (TSC) at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
- On May 7, 2025, Apertura Gene Therapy announced it will present progress on its TfR1 CapX capsid and TSC programs at ASGCT's 28th Annual Meeting in New Orleans.
- Apertura developed TfR1 CapX, a BBB-penetrant AAV capsid supported by the FDA, to enable gene therapy delivery to the CNS for tuberous sclerosis complex.
- The FDA recommended employing mice engineered to express the human transferrin receptor for TfR1 CapX biodistribution and safety evaluations, favoring this model over non-human primates and enabling a more efficient development process.
- Andrew Steinsapir, Acting CTO at Apertura, explained that their ASGCT presentations this year will focus on addressing key challenges in translation and manufacturing, demonstrating how TfR1 CapX can enhance and accelerate the creation of gene therapies targeting disorders of the central nervous system.
- Apertura's presentations will emphasize TfR1 CapX's potential to advance treatments for neurogenetic disorders and unlock therapies for large genes requiring CNS delivery.
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Apertura Gene Therapy Showcases the Progress of its Novel Human Transferrin Receptor AAV Capsid, TfR1 CapX, and Preclinical Programs for Tuberous Sclerosis Complex (TSC) at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
TfR1 CapX, a BBB-penetrant and uniquely human-focused AAV capsid targeting the central nervous system (CNS), demonstrates robust, brain-wide delivery via intravenous injectionFDA-supported TfR1 CapX development: TfR1 CapX receives FDA feedback and alignment on a clear, consistent, and streamlined development path…
·Parsons, United States
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