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ATM gene therapy
- Development stage
- Preclinical
- Lead developer
- IRCCS Ospedale San Raffaele
- Modality
- Molecular Glues → Targeted Protein Degraders (TPDs) → Small Molecules, Gene Therapies
- Administration
- Intravenous, Intracerebellar
01
Overview
ATM gene therapy is an experimental therapeutic approach aimed at treating Ataxia Telangiectasia (AT), a multisystem genetic disorder caused by mutations in the ATM gene. Due to the large size of the ATM coding sequence, which exceeds the capacity of standard viral vectors, researchers at San Raffaele Hospital and the National Research Council of Italy developed the STRING (System for Trans-splicing and Reconstitution of Integrated Gene) platform. This technology utilizes intein-based trans-splicing enhanced by an orthogonal molecular glue to reconstitute the full-length ATM protein from two or three fragments delivered via lentiviral or AAV vectors. The therapy is designed to restore ATM's role in DNA damage repair and cellular stress sensing, with development focusing on hematopoietic stem cell therapy to treat immunodeficiency and direct cerebellar delivery to prevent neurodegeneration.
- Other names
- ATM protein trans-splicing therapyATM dual lentiviral systemATM triple AAV system
02
Targets
ATM (Ataxia telangiectasia mutated protein)
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