ProjektA Next-Generation Precision Therapy Development Platform for Splice-Modulating ASO Therapies in A-T, advanced by…
Grunddaten
Titel:
A Next-Generation Precision Therapy Development Platform for Splice-Modulating ASO Therapies in A-T, advanced by Novel Therapeutic Targets and Chemistry
Laufzeit:
01.11.2026 bis 31.07.2029
Abstract / Kurz- beschreibung:
PRECISION-AT aims to advance this patient-customized sASO therapy platform to a nextgeneration level by:
(1) expanding the therapeutic target space to include splice-disrupting ATM mutations (SDMs) near the canonical site, currently untreatable by ASOs;
(2) enhancing sASO potency and durability via novel high-potency, long-acting ASO backbone chemistries to reduce dosing frequency and patient burden;and
(3) combining SDM-targeting sASO strategies with high-potency ASO chemistries to maximize therapeutic effect sizes, particularly for hard-to-target ATM mutations.
(1) expanding the therapeutic target space to include splice-disrupting ATM mutations (SDMs) near the canonical site, currently untreatable by ASOs;
(2) enhancing sASO potency and durability via novel high-potency, long-acting ASO backbone chemistries to reduce dosing frequency and patient burden;and
(3) combining SDM-targeting sASO strategies with high-potency ASO chemistries to maximize therapeutic effect sizes, particularly for hard-to-target ATM mutations.
Schlüsselwörter:
Biomarker
biomarker
Ataxia telangiectasia
ASO treatment
Neurodegenerative Diseases
Beteiligte Mitarbeiter/innen
Leiter/innen
Medizinische Fakultät
Universität Tübingen
Universität Tübingen
Neurologische Universitätsklinik
Kliniken und klinische Institute, Medizinische Fakultät
Kliniken und klinische Institute, Medizinische Fakultät
Lokale Einrichtungen
Abteilung Neurologie mit Schwerpunkt Neurodegenerative Erkrankungen
Neurologische Universitätsklinik
Kliniken und klinische Institute, Medizinische Fakultät
Kliniken und klinische Institute, Medizinische Fakultät