Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AAV-SHMT2
- Development stage
- Preclinical
- Modality
- Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
- Administration
- Intracerebral
01
Overview
AAV-SHMT2 is an experimental gene therapy designed to deliver the Serine Hydroxymethyltransferase 2 (SHMT2) gene using an adeno-associated virus (AAV) vector. SHMT2 is a critical mitochondrial enzyme involved in the one-carbon metabolism pathway. Research indicates that SHMT2 is significantly downregulated in Huntington's disease (HD), leading to metabolic dysregulation, accumulation of homocysteine, and suppression of histone lactylation. By restoring SHMT2 expression, AAV-SHMT2 aims to normalize the metabolic-epigenetic axis, thereby attenuating the degeneration of medium spiny neurons (MSNs), reducing mutant huntingtin (mHTT) aggregation, and improving motor performance. It is currently being investigated in preclinical models of Huntington's disease, including human striatal organoids and transgenic mouse models.
- Other names
- Adeno-associated virus-SHMT2AAV-mediated SHMT2 overexpression
02
Targets
SHMT2 (Serine hydroxymethyltransferase 2)
Beyond the preview
Go deeper on AAV-SHMT2.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AAV-SHMT2.