NatureCell America Achieves FDA IND Approval for Adult Autism Stem Cell Therapy ‘AstroStem-AU’

Initiation of Phase 1 Clinical Trials Signals Progress in Autism Therapy Development/ Advancing RMAT Designation and Accelerated Approval Pathways

LOS ANGELES, CA / ACCESS Newswire / April 15, 2026 / NatureCell Co., Ltd., a biotechnology company focused on adult stem cell therapies led by Chairman Jeong-chan Ra, announced that its U.S. subsidiary, NatureCell America, has received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration for AstroStem-AU, an autologous adipose-derived mesenchymal stem cell therapy for adults with Autism Spectrum Disorder (ASD).

This IND approval marks the company’s formal entry into clinical-stage development for adult ASD, an area with significant unmet medical need. Despite ASD affecting an estimated 1-2% of the adult population in the United States, treatment options for adults remain extremely limited, with no therapies addressing the underlying causes of the condition.

AstroStem-AU is derived from mesenchymal stem cells cultured from a patient’s own adipose tissue and is designed to be administered through a series of 10 intravenous infusions. Unlike conventional treatments that focus on symptom management, the therapy is intended to target the biological mechanisms underlying ASD.

NatureCell’s prior clinical experience supports this approach. The company has conducted FDA-authorized trials using a similar repeated intravenous dosing regimen in Alzheimer’s disease, establishing a meaningful safety profile. This foundation is expected to support the upcoming Phase 1 trial in adult ASD.

The Phase 1 study will primarily assess safety and tolerability, while also exploring early indicators of efficacy, including potential improvements in behavior, cognitive function, and overall quality of life. Positive early signals could help accelerate subsequent development phases.

In parallel, NatureCell America intends to pursue Regenerative Medicine Advanced Therapy (RMAT) designation. If granted, this status would enable closer regulatory engagement with the FDA and provide access to expedited development pathways, including the possibility of accelerated or conditional approval based on interim clinical data.

The autism treatment market continues to expand globally. Valued at approximately $25-30 billion in 2024, it is projected to grow steadily at an annual rate of 4-6%. In the United States alone, autism-related medical and societal costs exceed $250 billion annually. Therapies like AstroStem-AU, which aim to address disease mechanisms rather than symptoms alone, may establish a new premium segment within this market.

Looking ahead, NatureCell America plans to leverage the U.S. as a strategic entry point for expansion into Europe and Asia, further strengthening its global footprint in regenerative medicine.

Chairman Ra stated, “This IND clearance represents a pivotal step toward developing a treatment for adult autism.” He added, “Building on the safety data established in our Alzheimer’s studies, we are eager to evaluate both the safety and therapeutic potential of AstroStem-AU in this new indication.” He further emphasized the company’s commitment to rapidly pursuing RMAT designation and contributing to a new standard of care in autism treatment.

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