May 18, 2026 —
JCR Pharmaceuticals presented new preclinical data at the 29th American Society of Gene & Cell Therapy Annual Meeting, highlighting the potential of its JUST-AAV platform to improve systemic gene delivery to the central nervous system. The platform is designed to address one of the major limitations of neurological gene therapy: crossing the blood-brain barrier efficiently while reducing off-target accumulation in organs such as the liver.
The JUST-AAV platform uses engineered AAV capsids that incorporate miniaturized antibodies designed to bind transport receptors on the blood-brain barrier, including the transferrin receptor. By engaging this natural transport pathway, the vectors are intended to ferry genetic payloads into the brain and spinal cord after systemic administration. JCR also highlighted additional capsid modifications designed to reduce liver tropism, aiming to improve the therapeutic index of CNS-directed AAV gene therapy.
According to the data presented, brain-targeting JUST-AAV vectors achieved substantially higher CNS gene expression compared with conventional AAV9 in preclinical models, while markedly reducing liver accumulation. This combination of enhanced brain delivery and reduced peripheral exposure could be especially important for rare neurological and lysosomal storage diseases, where broad CNS distribution is needed but systemic AAV dosing can raise safety concerns.
JCR presented disease-focused preclinical results in GM1 gangliosidosis and neuronal ceroid lipofuscinosis, also known as Batten disease. In GM1 models, systemic administration of a JUST-AAV vector delivering a therapeutic enzyme led to high enzyme levels in the brain, reduced toxic substrate accumulation, improved neurological function, and extended survival. In CLN1 and CLN2 disease models, a single systemic dose of JUST-AAV therapy produced broad CNS enzyme delivery, reduced neuroinflammation, preserved motor function, and extended survival toward levels observed in healthy animals.
The platform has also gained strategic validation through JCR’s collaboration with Alexion, AstraZeneca Rare Disease. Under a 2025 license agreement, Alexion gained access to JCR’s proprietary capsids for up to five genomic medicine programs. At ASGCT, Alexion also presented supportive preclinical data using TfR-targeted JUST-AAV capsids, showing broad CNS biodistribution and favorable brain-to-liver exposure profiles in animal models.
Together, the ASGCT data position JUST-AAV as a potentially important next-generation AAV delivery platform for CNS disorders. While the technology remains preclinical and will require clinical validation, the findings suggest that receptor-targeted, liver-detargeted AAV capsids may help unlock new therapeutic opportunities for neurological diseases that have historically been difficult to reach with conventional gene therapy vectors.
