#USA #Boston #Broad Institute #Apertura Gene Therapy #TfR1 CapX

Apertura Gene Therapy to Present at ASGCT 2026

Apertura Gene Therapy, a trailblazer in biotechnology, is set to unveil groundbreaking advancements at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) in Boston, MA, from May 11 to 15, 2026. The focus will be on their innovative AAV capsid, TfR1 CapX™, which targets the central nervous system (CNS) and utilizes the human transferrin receptor 1 (hTfR1) for efficient delivery.

During this esteemed event, Apertura will reveal how TfR1 CapX™ can be manufactured at a clinically relevant scale. This achievement addresses one of the critical bottlenecks in the development of gene therapies, thereby accelerating the pathway for bringing these therapies to patients in need. Along with showcasing their production capabilities, Apertura will highlight the importance of employing human-relevant preclinical models for more accurate predictions during clinical translation of gene therapies.

Andrew Steinsapir, Acting CTO at Apertura, expressed excitement about sharing the company's latest progress with the broader gene therapy community. He emphasized the collective efforts of collaborators, partners, and patient advocacy groups all geared towards advancing treatment options for neurological diseases. Apertura’s presentations will reflect this collaborative spirit and the pressing need for innovation in the sector.

The meeting will also see contributions from scientists at the Broad Institute of MIT and Harvard, namely Dr. Sonia Vallabh and Dr. Ben Deverman. Their research underscores the versatility of TfR1 CapX™ and its application in several therapeutic strategies, including epigenetic silencing and base-editing methods aimed at mitigating prion protein levels in the brain. This innovative capsid marks a significant leap from Apertura’s first-generation capsid, BI-hTFR1, showcasing improved delivery mechanisms to navigate challenges such as neutralizing antibodies that limit therapeutic efficacy.

Apertura's commitment extends beyond mere presentations as the company introduces the Open Aperture program—an initiative offering academic researchers complimentary access to TfR1 CapX™ along with a practical playbook. This effort is designed to foster innovation and collaboration within the scientific community, promoting breakthroughs that could redefine gene therapy for CNS conditions.

Key Presentations

During the ASGCT meeting, Apertura will feature several presentations, including:

• - Defining pharmacologically relevant animal models for characterizing human transferrin receptor-binding AAVs by Chris Davis (Poster Presentation: May 12, 2026)
• - Establishing scalable TfR1 CapX manufacturing platform to accelerate multiple CNS gene therapy programs by Andrew Steinsapir (Poster Presentation: May 13, 2026)
• - Open Aperture: An express MTA framework enabling academic evaluation of an hTfR1-targeting AAV capsid for systemic CNS delivery by Von Wiltman (Poster Presentation: May 13, 2026)

Additionally, the Broad Institute will feature its research on prion disease therapies, including strategies for extending the lifespan of a humanized mouse model and advancing epigenetic editing techniques. The presentation titles reflect the cutting-edge nature of this research, showcasing the dynamic and rapidly evolving landscape of gene therapy.

About TfR1 CapX™

TfR1 CapX™ stands out as a promising AAV capsid with IV administration capabilities, which also crosses the blood-brain barrier. Its robust clinical readiness is validated through numerous preclinical developments and successful engagements with regulatory bodies. This proprietary capsid demonstrates enhanced CNS delivery compared to earlier models and is set to address significant neurological and genetic conditions.

Apertura Gene Therapy, founded on breakthrough technology from the Broad Institute and backed by Deerfield Management, aspires to revolutionize genetic medicine. By leveraging human receptors for gene delivery, Apertura aims to provide targeted therapies with enhanced efficacy for both pediatric and geriatric populations suffering from severe neurological disorders.

For more information about their work, audience members are encouraged to visit their website at aperturagtx.com and connect with them on LinkedIn to keep up with their latest endeavors and collaborations in the field of gene therapy.