Drug intelligence / Profile preview

rAAV-miniBEND

Development stage
Preclinical
Lead developer
Peking University
Modality
Gene Therapies
Administration
Intravenous, Parenteral
01

Overview

rAAV-miniBEND is a recombinant adeno-associated virus (rAAV) vector system optimized for highly specific and efficient gene delivery to brain endothelial cells (brainECs). Developed by researchers at Peking University and the Chinese Institute for Brain Research, the system utilizes a modular design featuring a truncated version of the Tek (Tie2) promoter and optimized cis-acting elements. This truncation engineering creates a compact transcriptional regulatory unit that saves approximately 2.8 kb of packaging space compared to standard promoters. The system is integrated with engineered capsids and has been validated through multiple delivery routes, including local intracranial injection and systemic intravenous administration. rAAV-miniBEND is primarily used as a research tool for cerebrovascular disease modeling, including the recapitulation of cerebral cavernous malformations (CCM) and brain arteriovenous malformations (bAVM) in rodent models.

Other names
rAAV-based mini-system for brain endothelial cells
02

Targets

TEK (Angiopoietin-1 receptor)

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