Apertura Gene Therapy and Scientists from the Broad Institute to Share Data on TfR1 CapX™, a Novel Capsid Targeting the Central Nervous System (CNS), at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

— Presentations show that TfR1 CapX^™ is being manufactured at clinical scale, a major hurdle in gene therapy development, and illustrate the importance of human-relevant preclinical models for gene therapy development —

— Presentations on Open Aperture, Apertura's material transfer agreement program, describe how open academic access to TfR1 CapX can unlock innovation and collaboration across the field —

NEW YORK, April 27, 2026 /PRNewswire/ -- Apertura Gene Therapy, a biotechnology company developing next-generation AAV capsids for delivering genetic medicines, along with scientists from the Broad Institute of MIT and Harvard, will present advancements related to TfR1 CapX^™, a leading AAV capsid that leverages human transferrin receptor 1 (hTfR1) to enable intravenous delivery to the brain and spinal cord, at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), held May 11–15, 2026 in Boston, MA.

Data presented by Apertura at ASGCT show that TfR1 CapX can be manufactured at clinically relevant scale, helping reduce one of the biggest bottlenecks in bringing therapies to patients. The company will also present on the utility of different animal models for the preclinical evaluation of novel AAV capsids such as TfR1 CapX, emphasizing that more human-relevant models are critical for more accurately predicting clinical translation. The company will also share how it is expanding access to its innovative TfR1 CapX capsid through its Open Aperture program, which provides academic researchers free access and a practical development playbook aimed at accelerating discovery and enabling more breakthroughs across the field.

"We look forward to sharing Apertura's latest updates with the broader gene therapy community at ASGCT this year," said Andrew Steinsapir, Acting CTO at Apertura and Director & Gene Therapy Program Lead at Deerfield Management. "The progress we continue to see with TfR1 CapX is a reflection of the dedication of our collaborators, partners, and patient advocacy groups who are all working toward the same goal: advancing new treatment options for patients with serious neurological diseases."

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, will highlight the versatility of TfR1 CapX across therapeutic approaches. These include 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.

"Our recent work shows the versatility of TfR1 CapX with a wide array of gene expression and editing payloads, enabling rapid blood-brain barrier crossing and efficient delivery to cells throughout the central nervous system," Deverman said. "We are excited to share our findings and help foster a community of open sharing amongst groups using TfR1 CapX to develop gene therapies for the CNS." 

Apertura Presentation Details

Title: Defining pharmacologically relevant animal models for characterizing human transferrin receptor-binding AAVs (#1519)
Presenter: Chris Davis
Poster Presentation: Tuesday May 12, 2026, 5:00 PM - 6:30 PM ET

Title: Establishing scalable TfR1 CapX manufacturing platform to accelerate multiple CNS gene therapy programs (#2237)
Presenter: Andrew Steinsapir
Poster Presentation: Wednesday May 13, 2026, 5:00 PM - 6:30 PM ET

Title: Open Aperture: An express MTA framework enabling academic evaluation of an hTfR1-targeting AAV capsid for systemic CNS delivery (#2236)
Presenter: Von Wiltman
Poster Presentation: Wednesday May 13, 2026, 5:00 PM - 6:30 PM ET

Broad Institute Presentation Details
Title: In vivo base editing extends the lifespan of a humanized mouse model of prion disease Nature Medicine 2025
Presenter: Jessie Davis
Oral Presentation: Tuesday May 12, 2026, 11:33 AM – 11:59 AM ET

Title: Progress towards a one-time epigenetic editing therapy for prion disease (#195)
Presenter: Fiona Serack
Oral Presentation: Wednesday May 13, 2026, 10:15 AM – 10:30 AM ET

Title: A modular capsid engineering platform to confer neutralizing antibody evasion to CNS and muscle targeted AAVs (#317)
Presenter: Simon Pacouret
Oral Presentation: Thursday May 14, 2026, 11:30 AM – 11:45 AM ET

Title: An improved human transferrin receptor-targeted AAV capsid mediates brain-wide silencing of Prnp (#3037)
Presenter: Ken Chan
Poster Presentation: Thursday May 14, 2026, 5:00 PM - 6:30 PM ET

About TfR1 CapX^™
TfR1 CapX^™ is a leading IV-administered, BBB-crossing capsid. Multiple for-profit and non-profit organizations have validated and licensed TfR1 CapX, additional groups are in discussions to license the technology. Clinical readiness has been supported by several preclinical development programs, including regulatory engagement and manufacturing by contract development and manufacturing organizations (CDMOs). TfR1 CapX is a proprietary, second-generation capsid that demonstrates superior CNS delivery compared to Apertura's first-generation capsid, BI-hTFR1. Research on the first-generation BI-hTFR1 capsid was published in Science.

About Apertura Gene Therapy 
Apertura Gene Therapy develops genetic medicines and next-generation AAV capsids that engage human-relevant receptors, aiming to enable more effective and selective gene delivery. The company's lead capsid, TfR1 CapX^™, leverages human transferrin receptor 1 to enable intravenous delivery to the brain and spinal cord. This established transport mechanism has a strong clinical track record in pediatric and geriatric populations, expanding its potential to treat serious neurological and genetic diseases. Apertura has licensed its next-generation capsids to multiple partners, with several programs expected to enter clinical trials over the next 12 months. Founded in 2021 on technology from the Broad Institute and supported by Deerfield Management, Apertura Gene Therapy is headquartered in New York City. Learn more at aperturagtx.com and follow us on LinkedIn. 

Contact:

Apertura Gene Therapy:
[email protected] 

SOURCE Apertura Gene Therapy