Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AAV8-hFIX-GRE-ITR
- Development stage
- Preclinical
- Lead developer
- Indiana University
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV8-hFIX-GRE-ITR is an experimental adeno-associated virus (AAV) gene therapy candidate designed for the treatment of hemophilia B. It utilizes an AAV8 capsid to deliver a transgene encoding the hyperactive Padua variant of human Factor IX (hFIX-Padua) under the control of a hepatocyte-specific promoter (such as the TTR promoter). A key innovation in this construct is the modification of the AAV inverted terminal repeats (ITRs) to include a glucocorticoid receptor-binding element (GRE). This modification is intended to enhance transgene expression efficiency and potentially allow for lower vector dosing, thereby reducing the risk of liver toxicity associated with high-dose AAV administration. Preclinical studies in hemophilic mice have demonstrated that the GRE-ITR modification leads to significantly higher levels of hFIX expression compared to wild-type ITR vectors.
- Other names
- scAAV8-hFIX-Padua-GRE-ITRscAAV-8-hFIX-Padua-GRE-ITRscAAV 8-hFIX-Padua-GRE-ITRAAV8-hFIX-Padua-GRE-ITRAAV-8-hFIX-Padua-GRE-ITRAAV 8-hFIX-Padua-GRE-ITR
02
Targets
Adeno-associated virus replication proteinF9 (Coagulation Factor IX)ASGPR (Asialoglycoprotein Receptor 1)GR (Glucocorticoid receptor)
Beyond the preview
Go deeper on AAV8-hFIX-GRE-ITR.
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 AAV8-hFIX-GRE-ITR.