JCR Pharmaceuticals showcased its innovative J-Brain Cargo® technology at the ASGCT 28th Annual Meeting in New Orleans, emphasizing its efficiency in delivering adeno-associated virus (AAV) gene therapies to the central nervous system (CNS).

The presentation highlighted preclinical data demonstrating the proprietary technology's ability to effectively cross the blood-brain barrier, a significant hurdle in CNS gene therapy.

Dr. Hiroyuki Sonoda underscored the importance of these findings, noting their potential to treat challenging CNS diseases more effectively than traditional methods.

Research findings included a study where a brain-targeting AAV vector achieved a remarkable 77-fold increase in green fluorescent protein expression in mouse brains compared to the conventional AAV9, while also reducing liver tropism by 99%.

In a mouse model of neuronal ceroid lipofuscinosis, the application of the JUST-AAV vector resulted in symptom disappearance and an extended lifespan, suggesting its potential as a functional cure.

The JUST-AAV platform not only enhances targeted delivery but also minimizes liver tropism, thereby improving the safety and efficacy of gene therapies for CNS applications.

In monkey studies, the bispecific vector demonstrated improved gene delivery efficiency to the brain, while also significantly reducing infections in undesirable tissues like the liver by over 90%.

Yuhei Ashida's oral presentation detailed the development of a brain-targeting AAV vector that significantly increased gene delivery to the brain while minimizing liver accumulation.

JCR Pharmaceuticals is committed to leveraging its scientific expertise to develop next-generation therapies, with ongoing investigations targeting several rare diseases.

The ASGCT serves as a premier organization for professionals in gene and cell therapy, promoting the knowledge and application of these therapies to combat human diseases.

Founded 50 years ago, JCR Pharmaceuticals is dedicated to developing therapies for rare and genetic diseases and is pursuing a global expansion strategy.

The ASGCT 28th Annual Meeting took place from May 13-17, 2025, gathering experts to discuss advancements in gene therapy.