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HdAd-ATM

Development stage: Preclinical
Lead developer: University of North Carolina at Chapel Hill
Modality: Gene Therapies
Administration: Intrathecal

Overview

HdAd-ATM is a gene therapy candidate designed to treat Ataxia-Telangiectasia (A-T), a multi-system disorder caused by mutations in the ATM gene. Because the ATM coding sequence is approximately 9.2 kb, it exceeds the packaging capacity of standard adeno-associated virus (AAV) and lentiviral vectors. HdAd-ATM utilizes a helper-dependent adenoviral (HdAd) vector, which has a large packaging capacity of approximately 36 kb, to deliver a full-length, functional copy of the ATM gene. This approach aims to restore ATM-mediated DNA double-strand break (DSB) repair signaling and genomic integrity, particularly in Purkinje neurons, to prevent the progressive cerebellar degeneration characteristic of A-T. Preclinical data presented at ASGCT 2026 demonstrated that HdAd-ATM vectors using the Ubiquitin C (UBC) promoter successfully restored DNA damage response recruitment in patient-derived fibroblasts and murine cortical neurons without observed cytotoxicity.

Other names: Helper-Dependent Adenoviral Delivery of Full-Length ATM

Targets

ATM (Ataxia telangiectasia mutated protein)

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