Clinical trials
Follow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AAVrh.10hCAT(KANL⁻)
- Development stage
- Preclinical
- Lead developer
- Weill Cornell Medicine
- Modality
- Gene Therapies
- Administration
- Intravitreal, Intravenous
01
Overview
AAVrh.10hCAT(KANL⁻) is an adeno-associated virus (AAV) serotype rh.10-based gene therapy designed for the treatment of dry age-related macular degeneration (AMD). Developed by researchers at Weill Cornell Medicine, the vector utilizes a CAG promoter to drive the expression of a modified human catalase (hCAT) gene. The modification involves the deletion of the C-terminal KANL sequence (amino acids 524-527), which normally serves as a peroxisomal targeting signal. By removing this sequence, the resulting catalase enzyme is secreted from the cell rather than being sequestered intracellularly. This creates a persistent extracellular antioxidant shield in the retina, neutralizing hydrogen peroxide (H2O2) and protecting retinal pigmented epithelium and photoreceptor cells from oxidative stress-induced degeneration.
- Other names
- AAVrh.10-CAG-hCAT(KANL⁻)
02
Targets
Sulfated N-acetyllactosamine/keratan sulfate-like cell-surface glycanNeuraminidase
Beyond the preview
Go deeper on AAVrh.10hCAT(KANL⁻).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Explore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Trace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Explore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Compare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Connect 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 AAVrh.10hCAT(KANL⁻).