#USA #New York #Viralgen #Apertura Gene Therapy #TfR1 CapX

Collaboration to Manufacture Gene Therapy

On March 17, 2026, Apertura Gene Therapy and Viralgen announced a significant partnership aimed at the production of the TfR1 CapX, a cutting-edge AAV capsid specifically designed for gene therapies targeting central nervous system disorders. This strategic collaboration is set to enhance the efficiency and scalability of gene therapy development programs that leverage the innovative capabilities of Apertura's TfR1 CapX.

What is TfR1 CapX?

TfR1 CapX is an advanced AAV capsid that binds to the human transferrin receptor 1 (hTfR1), allowing it to cross the blood-brain barrier effectively. This mechanism is crucial for delivering genetic medicines to the central nervous system (CNS), which has historically been a significant challenge in the field of gene therapy. Early preclinical studies have demonstrated that TfR1 CapX shows remarkable selectivity towards CNS tissues, with robust transduction of neurons and astrocytes across various brain regions, as evidenced by independent tests.

Benefits of the Collaboration

Through this partnership with Viralgen, Apertura's licensees can access the TfR1 CapX plasmid efficiently via Viralgen's integrated AAV manufacturing platform. This is expected to streamline the scaling process and ensure a smooth transition from research phases to clinical development. Dr. Diego Garzón, Chief Commercial Officer at Apertura, expressed optimism about the collaboration, highlighting that multiple partners of Apertura are anticipated to advance their gene therapy programs into clinical phases within this year and the next.

Viralgen, known for its expertise in contract development and manufacturing for gene therapies, has already expanded its production capabilities for TfR1 CapX. With an established AAV manufacturing process that has proven successful in over 1,500 batches, Viralgen supports the generation of critical materials and provides integrated services necessary for developing effective gene therapies.

Dr. Jimmy Vanhove, CEO of Viralgen, emphasized the significance of a well-defined production framework that allows gene therapy programs to progress efficiently towards clinical development and the emergence of scalable, effective gene therapies. With their state-of-the-art facility in San Sebastián, Spain, which comprises several cGMP-certified production suites, Viralgen is poised to provide reliable and swift solutions for the gene therapy landscape.

Moving Forward

This collaboration is designed to facilitate the clinical manufacturing planning and regulatory engagement necessary for Apertura's ongoing gene therapy initiatives. As Jorge Santiago-Ortiz, VP of CMC and Regulatory Affairs at Apertura, noted, this partnership allows their licensees to benefit from a rapid and efficient supply of the TfR1 CapX plasmid, ensuring that clinical production timelines are met.

The strategic alliance between Apertura Gene Therapy and Viralgen marks a crucial step forward in the pursuit of innovative and effective gene therapies targeting serious neurological and genetic disorders. With TfR1 CapX's unique capabilities and this new manufacturing collaboration, the potential to bring life's transformative therapies to patients is more promising than ever.

About Apertura Gene Therapy

Founded in 2021 and headquartered in New York City, Apertura Gene Therapy focuses on developing next-generation genetic medicines and AAV capsids that interact with human receptors to enhance gene delivery. The company is planning to bring its therapeutic programs into clinical trials soon, with the backing of Deerfield Management Company and technologies from the Broad Institute.

About Viralgen

Established in 2017 as a subsidiary of AskBio, Viralgen specializes in contract development and manufacturing services for gene therapies based on AAV. With its advanced Pro10™ cell line and Aava™ manufacturing platform, Viralgen is leading the way in scalable, high-performance AAV manufacturing, enabling faster and more consistent delivery of transformative gene therapies to patients worldwide.