Apertura Gene Therapy Collaborates with Broad Institute and Whitehead Institute to Advance Gene Therapy for Prion Disease

BOSTON and NEW YORK, Feb. 28, 2025 /PRNewswire/ – Apertura Gene Therapy, a biotechnology company pioneering next-generation gene therapy solutions, is supporting the Broad Institute of MIT and Harvard and the Whitehead Institute in the development of a novel gene therapy approach for prion disease. The project is led by the Vallabh-Minikel Lab at the Broad Institute, which is dedicated to finding a cure for prion disease. This groundbreaking initiative combines cutting-edge technologies from both the Broad and Whitehead Institutes, leveraging the CHARM platform—developed in Dr. Jonathan Weissman’s lab—and the TfR1 capsid, an engineered AAV vector designed in the lab of Dr. Ben Deverman, Director of Vector Engineering at the Broad Institute and scientific founder of Apertura.

Prion disease is a rare, fatal neurodegenerative disorder caused by misfolded proteins that lead to progressive brain damage. The new gene therapy is designed to target the root cause of the disease using CHARM (Coupled Histone tail for Autoinhibition Release of Methyltransferase) to silence the gene responsible for producing the disease-causing protein. This therapeutic payload will be delivered using Apertura’s TfR1 capsid, a next-generation adeno-associated virus (AAV) vector engineered to efficiently cross the blood-brain barrier (BBB) by binding to the human TfR1 receptor, a key transporter that facilitates iron uptake into brain cells. The combination of these two technologies offers a potentially transformative approach to treating central nervous system (CNS) diseases.

A Breakthrough Collaboration in Neurodegenerative Disease Research

“We are thrilled to see the progress being made in developing this innovative gene therapy for prion disease,” said Dr. Sonia Vallabh, co-leader of the Vallabh-Minikel Lab at the Broad Institute. “The collaboration between Apertura, the Broad Institute, and the Whitehead Institute marks a critical milestone in addressing one of the most devastating neurodegenerative disorders. By combining state-of-the-art gene-silencing technology with an advanced brain-penetrating AAV vector, we are taking an important step toward meaningful treatment options for patients.”

The engineered TfR1 capsid, developed by Dr. Deverman’s team and Apertura Gene Therapy, has demonstrated the ability to efficiently cross the blood-brain barrier, enabling targeted delivery of gene therapies to the CNS via intravenous administration. This innovation represents a significant leap forward in the field, offering a less invasive yet highly effective method for treating neurological disorders.

Apertura and the Broad Institute have worked closely throughout this project, with Apertura sharing preclinical data and regulatory insights from its genetic epilepsy program, where the TfR1 capsid has been a critical component. This collaborative knowledge-sharing has allowed the Broad Institute team to accelerate the timeline for clinical development, positioning the gene therapy for prion disease to move swiftly toward human trials.

Unlocking the Potential of the TfR1 Capsid for CNS Gene Therapy

“I believe the engineered TfR1-AAV capsid has immense potential as a highly effective delivery system for genetic medicines targeting the CNS,” said Dr. Ben Deverman, Director of Vector Engineering at the Broad Institute. “We are grateful to the team at Apertura for their continued support and access to their extensive data. The insights gained from their preclinical and regulatory experience serve as a valuable roadmap for the broader gene therapy community.”

The need for more efficient and less invasive methods to deliver gene therapies to the brain is a growing priority in the field. Apertura’s TfR1 capsid offers an alternative to traditional gene therapy delivery methods, such as direct injection into the brain or subretinal administration, which are highly invasive and limit patient accessibility.

“Apertura recognizes that many organizations are working to develop therapies for rare and ultra-rare diseases, where a more potent and non-invasive delivery platform like our TfR1 capsid could be a game-changer,” said Dr. Diego Garzón, Apertura’s Vice President of Corporate Development. “We are committed to collaborating with both for-profit and non-profit partners to explore pathways for making this cutting-edge technology widely available for CNS diseases.”

A Model for Future Gene Therapy Development

The progress of this gene therapy underscores the power of scientific innovation and strategic collaboration in tackling some of the world’s most challenging diseases. By bringing together Apertura’s expertise in gene therapy development with the Broad Institute’s pioneering research, the partnership aims to deliver transformative treatments for patients suffering from prion disease.

This initiative also sets an important precedent for how biotech companies, research institutions, and non-profits can work together to rapidly translate gene therapy discoveries into real-world therapeutic solutions. The work being done today could pave the way for future gene therapies targeting Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, and other CNS disorders that currently lack effective treatments.

About Apertura Gene Therapy

Apertura Gene Therapy is a biotechnology company dedicated to advancing gene therapies for severe, unmet medical needs. Founded in 2021 on technology developed at the Broad Institute, and backed by Deerfield Management Company, Apertura focuses on innovative capsid engineering and gene therapy platforms to improve delivery, safety, and efficacy in treating CNS and genetic disorders. Apertura is headquartered in New York City.

Source:
https://www.biospace.com/press-releases/apertura-gene-therapy-supports-the-broad-institute-in-development-of-gene-therapy-for-prion-disease-using-engineered-aav-capsid-targeting-tfr1-for-cns-delivery

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