Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
CRISPR-Cas9 adeno-associated virus (apolipoprotein E4)
- Development stage
- Preclinical
- Lead developer
- Columbia University
- Modality
- CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intravenous
01
Overview
CRISPR-Cas9 adeno-associated virus (apolipoprotein E4) is an experimental gene therapy candidate designed for the treatment of Alzheimer's disease. It utilizes an all-in-one adeno-associated virus (AAV) vector to deliver CRISPR-Cas9 components (specifically SaCas9 and single-guide RNAs) that target and knock down the expression of the apolipoprotein E (APOE) ε4 allele, which is the primary genetic risk factor for Alzheimer's. Developed by researchers at Columbia University, the therapy is administered intravenously and relies on focused ultrasound (FUS)-mediated blood-brain barrier opening (BBBO) to achieve targeted delivery to the central nervous system. Preclinical data indicates that this approach effectively reduces APOE4 mRNA and protein levels, decreases neuroinflammatory markers (astrogliosis and microgliosis), and reduces amyloid-beta accumulation in the brain.
- Other names
- AAV-CRISPR-Cas9 (APOE4)AAV-SaCas9-sgAPOE4AAV-SaCas-9-sgAPOE4AAV-SaCas 9-sgAPOE4All-in-one CRISPR-Cas9 AAV
02
Targets
APOE (Apolipoprotein E)
Beyond the preview
Go deeper on CRISPR-Cas9 adeno-associated virus (apolipoprotein E4).
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 CRISPR-Cas9 adeno-associated virus (apolipoprotein E4).