Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
PTB shRNA
- Development stage
- Preclinical
- Lead developer
- brainVTA
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics, Gene Editing → Gene Therapies
- Administration
- Intrathecal, Intratumoral, Intravenous
01
Overview
PTB shRNA is a research-stage RNA interference (RNAi) tool designed to silence the expression of Polypyrimidine Tract-Binding Protein 1 (PTBP1), an RNA-binding protein that regulates alternative splicing. By knocking down PTBP1, researchers aim to induce glia-to-neuron reprogramming, specifically converting astrocytes into functional neurons. This approach is being investigated for the treatment of neurodegenerative and psychiatric conditions, including Alzheimer's disease, Parkinson's disease, and depression, as well as spinal cord injury and certain cancers like ovarian cancer. The shRNA is typically delivered via viral vectors such as adeno-associated virus (AAV) or lentivirus. It is currently used as a laboratory reagent and experimental therapeutic candidate rather than an approved pharmaceutical product.
- Other names
- PTBP1 shRNAPTBP-1 shRNAPTBP 1 shRNAshPTBshPTBP1shPTBP-1shPTBP 1Polypyrimidine Tract-Binding Protein 1 shRNA
02
Targets
PTBP1 (Polypyrimidine tract-binding protein 1)
Beyond the preview
Go deeper on PTB shRNA.
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 PTB shRNA.