BOSTON — JCR Pharmaceuticals reported early preclinical data in Boston showing that its experimental gene therapy delivery platform may improve the movement of treatments into the central nervous system while reducing exposure in the liver, a key challenge for some gene therapy approaches.
The Hyogo, Japan-based biopharmaceutical company presented the data at the American Society of Gene and Cell Therapy’s annual meeting, held May 11-15. The research focused on JCR’s JUST-AAV platform, an investigational adeno-associated virus technology designed to target the brain, muscle and other tissues more selectively than conventional approaches.
AAV-based gene therapies have shown promise for rare genetic diseases, but getting therapies across the blood-brain barrier and into the central nervous system remains difficult. Liver accumulation is also a safety concern in the field. JCR said its preclinical studies suggest JUST-AAV may help address both issues.
“The presented preclinical results demonstrate that JCR’s novel capsid platform is able to deliver therapeutic agents to the central nervous system more efficiently than conventional AAV9, while reducing liver accumulation,” said Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer at JCR Pharmaceuticals. “These data represent an advancement toward potential new treatment options for previously challenging CNS diseases. This research is an important step forward in our ongoing commitment to developing innovative solutions for complex healthcare challenges, including neurodegenerative disorders.”
One study examined a potential gene therapy approach for GM1 gangliosidosis, a rare inherited disorder that affects the nervous system. Researchers tested an AAV9-based therapy designed to produce a lysosomal enzyme capable of crossing the blood-brain barrier through a transferrin receptor-targeting mechanism.
In mouse models, the therapy reduced disease-related buildup in the brain, improved neurological function and extended survival, according to the company. Researchers also reported no apparent toxicity tied to liver expression or systemic exposure to the enzyme.
JCR also presented data from studies in mouse models of neuronal ceroid lipofuscinosis, including CLN1 and CLN2, rare neurodegenerative lysosomal storage disorders. In the CLN1 study, mice treated with JUST-AAV-PPT1 lived nearly as long as healthy control animals, while animals treated with a conventional AAV9 approach had shorter survival gains.
The company said treated mice also showed reduced markers of inflammation and lysosomal storage, higher enzyme activity in the brain and preservation of motor function and retinal thickness.
In a CLN2 model, JCR said its JUST-AAV-TPP1 therapy produced a strong survival benefit and showed evidence of enzyme activity in brain tissue after systemic administration.
Alexion, AstraZeneca Rare Disease also presented preclinical data from a collaboration using JCR’s JUST-AAV technology. That research evaluated a transferrin receptor-targeted capsid designed to broaden central nervous system delivery while reducing liver exposure. Studies in mice and non-human primates showed higher brain-to-liver exposure ratios compared with AAV9, according to the companies.
The findings remain early-stage and preclinical, meaning the approaches have not yet been tested in human clinical trials. Study limitations were not discussed in the release.
