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AAV-SIDT1 + AAV-DTWD2
- Development stage
- Preclinical
- Lead developer
- Hannover Medical School
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV-SIDT1/DTWD2 is an investigational adeno-associated virus (AAV)-based gene therapy combination designed to restore the expression of SID1 transmembrane family member 1 (SIDT1) and DTW domain containing 2 (DTWD2). These two proteins are key mediators of glycosylated non-coding small RNA (glycoRNA) biosynthesis. In patients with metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH), the expression of glycoRNAs and their biogenesis mediators, SIDT1 and DTWD2, is significantly reduced, leading to increased fatty acid load and inflammation. By delivering functional copies of the SIDT1 and DTWD2 genes via AAV vectors, this therapy aims to restore glycoRNA biogenesis, thereby attenuating hepatic steatosis, inflammation, and fibrosis. Preclinical studies in mouse models of MASH have demonstrated that AAV-mediated restoration of SIDT1 and DTWD2 expression significantly improves liver pathology.
- Other names
- AAV-DTWD2AAV-DTWD-2AAV-DTWD 2AAV-SIDT1AAV-SIDT-1AAV-SIDT 1AAV-SIDT1/DTWD2
02
Targets
DTWD2 (tRNA-uridine aminocarboxypropyltransferase 2)SIDT1 (SID1 transmembrane family member 1)
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