Apertura Gene Therapy Unveils Breakthrough AAV Capsid Progress at ASGCT 2025
Apertura Gene Therapy Unveils Breakthrough AAV Capsid Progress at ASGCT 2025
Apertura Gene Therapy, a pioneering biotechnology company specializing in gene therapy innovations, recently made significant strides in the realm of targeted therapies for neurological disorders during the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), which took place from May 13-17, 2025, in New Orleans, LA. The company presented notable advancements in their human transferrin receptor-targeted adeno-associated virus (AAV) capsid known as TfR1 CapX.
Significant Developments at ASGCT
At the ASGCT meeting, Apertura's team focused on the preclinical and regulatory progress associated with TfR1 CapX, which aims to enhance gene therapy delivery to the brain. The company highlighted positive feedback received from the FDA concerning their TSC1 therapeutic program, which endorsed a streamlined preclinical development pathway focused on using human transferrin receptor knock-in mice as the pivotal model for evaluating safety and biodistribution for TfR1 CapX therapies.
Furthermore, the FDA's acknowledgment included the dismissal of non-human primate studies for this context due to the unique binding affinity between TfR1 CapX and the human transferrin receptor. This endorsement is pivotal for both accelerating research and reducing associated costs, while laying down a strategic roadmap for the translational development of future gene therapies.
Advancements in TSC2 Program
Alongside the updates on TSC1, Apertura also introduced findings from their TSC2 research program. A cell-based potency assay targeting the mTOR pathway—a critical pathway inhibited in Tuberous Sclerosis Complex (TSC)—revealed that neither previously documented nor internally engineered TSC2 minigene constructs succeeded in restoring mTOR activity. Owing to the large size of the TSC2 gene, Apertura is working on developing multiple functional TSC2 split intein payloads to effectively restore mTOR functionality in laboratory settings.
The inherent capability of TfR1 CapX to cross the blood-brain barrier and enable central nervous system (CNS) delivery is paramount in simplifying this novel split intein strategy. Apertura's innovation stands poised to not only tackle TSC but also open new avenues in treating other CNS ailments that necessitate the delivery of sizeable genes or novel gene editing technologies.
Expert Insight
Andrew Steinsapir, the Director of Gene Therapy Program Lead at Deerfield Management and Acting CTO at Apertura Gene Therapy, emphasized the company's commitment to leading the charge in neurological therapeutics. "Our goal is to address the critical issues surrounding translational aspects and manufacturability early on, ultimately demonstrating how TfR1 CapX can facilitate the development of gene therapies targeting CNS disorders," he stated.
The presentations at ASGCT underscored TfR1 CapX's potential as a versatile delivery vehicle for CNS applications and reinforced Apertura's intention to revolutionize the gene therapy landscape for neurogenetic disorders. With ongoing IND-enabling studies and an established regulatory pathway, TfR1 CapX is set to unveil novel treatment modalities across various neurometabolic and neurodevelopmental conditions.
Presentation Highlights
Apertura showcased a series of presentations at the ASGCT, aiming to expand on their findings and share important data with the scientific community. Some highlighted presentations included:
- - Redefining TSC2 Gene Therapy Development - Focused on a novel split intein payload and its translational potency assay.
- - Fit-for-Purpose Analytics for Engineered Capsids - An exploration into enhanced precision in AAV characterization.
- - Revolutionizing Neurometabolic Gene Therapy - Discussing the TfR1 CapX platform and the regulatory pathway devoid of non-human primates.
- - Intravenous Delivery of CNS-Penetrant AAV Gene Therapy - Provided insights into survival benefits observed in a TSC1 disease model.
- - FDA-Aligned Strategy for TSC1 Gene Therapy Development - Detailed on advancing preclinical development.
- - Machine Learning in AAV Gene Therapy Manufacturing - Highlighted how optimization efforts can be enhanced through novel analytical techniques.
Apertura Gene Therapy is uniquely positioned within the biopharmaceutical landscape, committed to developing groundbreaking therapies aimed at treating conditions previously deemed intractable. Founded in 2021 on the technological resources from the Broad Institute and backed by Deerfield Management, Apertura continues to push forward with its mission of unlocking access to genetic treatments across multiple disease scenarios.
For further information about Apertura Gene Therapy, explore their official site at www.aperturagtx.com and connect with them on LinkedIn.
Topics Health）
【About Using Articles】
You can freely use the title and article content by linking to the page where the article is posted.
※ Images cannot be used.
【About Links】
Links are free to use.