JUST-AAV
What is JUST-AAV
JUST-AAV is JCR’s proprietary gene therapy platform based on adeno-associated virus (AAV), with the potential to be applied to a wide range of diseases.
The name reflects the initials of JCR, Ultimate destination of organ, Safeguarding against off-target delivery, and Transformative technology.
Key Features of JUST-AAV
- Enables efficient gene delivery
to target tissues or organs while reducing distribution
to non-target sites, supporting an improved safety profile. - Target tissues or organs can be redirected
by modifying the tag (binder or miniaturized antibody)
expressed on the AAV capsid surface.
Expectations for JUST-AAV
AAV is widely known as a non-pathogenic virus and is commonly used as a gene therapy vector to deliver therapeutic genes. However, challenges remain, including liver accumulation that may lead to adverse effects and difficulty achieving efficient delivery to specific tissues such as the brain and muscle.
JUST-AAV is JCR’s proprietary engineered AAV. A miniaturized antibody displayed on the capsid surface helps direct the vector efficiently to target tissues and organs. With promising non-clinical results, JCR has succeeded in reducing liver accumulation by modifying part of the gene that encodes the capsid. Together, these approaches are expected to reduce liver toxicity risk while enhancing therapeutic gene expression in target tissues and organs.