NatureCell Submits U.S. IND for Stem Cell Autism Therapy
Company plans early-stage clinical trial of MSC-based therapy and aims for FDA RMAT designation to accelerate development
NatureCell announced on March 16 that its U.S. subsidiary, NatureCell America, submitted an Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) on March 13 for a stem cell therapy targeting autism spectrum disorder (ASD).
The proposed study is an early-stage clinical trial evaluating a mesenchymal stem cell (MSC)-based therapy. The treatment is designed to address ASD by regulating neuroinflammation and restoring immune balance observed in patients.
Autism spectrum disorder is a neurodevelopmental condition characterized by impaired social interaction and communication, along with repetitive behaviors. In the United States, drugs such as risperidone and aripiprazole are used to manage behavioral symptoms, but no therapies have been approved to treat the disorder’s core symptoms.
Citing data from the U.S. Centers for Disease Control and Prevention (CDC), NatureCell noted that one in 36 children in the United States is diagnosed with ASD. The global patient population is estimated to exceed 75 million, with the related treatment and management market valued at around $28 billion.
The company added that the Biostar Stem Cell Technology Research Institute has been providing stem cell–based regenerative treatments in Japan under the country’s Act on the Safety of Regenerative Medicine. Clinical practice has reported cases suggesting potential symptom improvement, prompting NatureCell to pursue more systematic evaluation through U.S. clinical trials.
If the IND is approved, NatureCell plans to seek RMAT (Regenerative Medicine Advanced Therapy) designation from the FDA, which provides closer regulatory consultation and can accelerate clinical development and review.
A company official said, “Autism spectrum disorder remains a major unmet medical need worldwide. Based on our experience with regenerative medicine treatments in Japan, we aim to scientifically validate the potential of stem cell therapy through U.S. clinical trials and offer a new treatment option.”