Innovation
Ingenuity with intention
Centered around curiosity and collaboration, we leverage our technologies and expertise to develop life-changing solutions for rare diseases before exploring how they can help with more widespread, but equally challenging conditions.
Research that questions conventions
We are firm believers of diving into the intricacies of science to find meaningful solutions, never settling for surface-level answers. After all, meticulous dedication is deeply ingrained in us.
But ingenuity does not happen because we follow the rules – it happens because we challenge them. That is why we encourage every employee to take ownership of their research, think creatively and push the boundaries of Research & Development. Our culture and unique approach have driven the creation of numerous breakthrough therapies already, helping us to transform the care of complex disease and shape the future of health.
Our focus
Lysosomal storage disorders (LSDs) are a key area of interest for us. Although treatments for LSDs do exist, their limited efficacy and significant side effects mean they do not improve a patient’s quality of life.
There is still an unmet need. We utilize our expertise to develop new and improved proprietary biotherapeutics (such as J-Brain Cargo®), providing patients and their families viable solutions that make a real difference.
We are also taking this approach beyond LSDs, expanding into therapies for central nervous system disorders.
J-Brain Cargo®
The first approved blood-brain barrier penetration technology
“Please save my son”
Four words that changed everything
“For years, delivering medicine to the brain was considered impossible. But that all changed when a mother desperately approached one of our researchers during a patient advocacy event, pleading to save her son. Inspired by her story and many others like hers, my team refused to accept those limits and persisted in finding an answer. Today, J-Brain Cargo® stands as a breakthrough platform, enabling medicines to cross the blood-brain barrier and target neurological areas once thought out of reach. For us, this is not just about science but delivering our promise to families still waiting for a solution.”
Hiroyuki Sonoda
Director, Senior Managing Executive Officer, Research, Executive Director, Research Division
TEMCELL®
The first allogeneic regenerative medicine in Japan
TEMCELL® HS Inj. derives from human mesenchymal stem cells (MSCs) and is developed using the bone marrow collected from healthy donors. Unlike traditional treatments, these cells can be given to any patient without the need for matching donor-recipient HLA. Due to the nature of MSCs, they can be produced and stored in advance, making them readily available for both treatment and emergencies.
Therapyareas
We are developing treatments for rare diseases, including therapeutic products for mucopolysaccharidosis type II (Hunter syndrome), MPS I, MPS IIIA, MPS IIIB and Fabry disease.
We offer therapeutic solutions for growth hormone deficiency and short stature.
We are advancing treatments for acute graft-versus-host disease with cutting-edge regenerative medical products.
Excellence in biomanufacturing
When it comes to producing multiple programs quickly, simultaneously and cost-effectively, our internal manufacturing capabilities are unparalleled.
Many companies typically use large bioreactors to produce biopharmaceuticals at scale. However, this method is not ideal when producing smaller batches (like orphan drugs) as they must be cleaned with large amounts of water after each cycle.
We knew that small-scale production would be our specialty from the start. So we built our own, more efficient system using industry-leading technology, enabling us to produce a stable supply of high-quality pharmaceuticals that meet both global standards and demand.