Aspen Neuroscience, Inc., a private biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies, including the first autologous neuron replacement for Parkinson’s disease (PD), announced that this month it will launch the first patient screening study of its kind, working with multiple clinical screening sites in the U.S.
The company’s Trial-Ready Cohort Study is a preliminary step to filing an Investigational New Drug (IND) application with the US Food & Drug Administration for ANPD001, its first therapeutic development candidate for potential treatment of idiopathic Parkinson’s disease (PD). The Trial-Ready Cohort Study will provide information to and screen potential patient candidates for the company’s planned Phase 1/2A first-in-patient clinical trial of ANPD001. The company will announce multiple U.S. screening sites throughout the spring.
“This is an historic moment for patients and for the Aspen Neuroscience team, as we open our first screening study to expedite our investigation of iPSC-derived cell replacement therapies for Parkinson’s disease,” said Damien McDevitt, Ph.D., president and chief executive officer. “We are excited and very humbled to begin this next phase during Parkinson’s Awareness Month. This is a significant step forward for the patient community, for health care providers and the neuroscience field.”
PD is the second most common neurodegenerative disease, affecting around one million Americans and more than ten million people worldwide. Even with the current standard of care therapy, patients can eventually develop debilitating motor complications due to loss of dopamine neurons in the brain; approximately 50% are lost even before diagnosis.
Aspen Neuroscience is the leading company developing a personalized cell replacement that can eliminate the need for immunosuppressive therapy. The approach utilizes a patient’s own skin cell-derived iPSCs to produce replacement dopamine neurons for transplantation back into the same patient. Developed from a simple skin biopsy, each patient’s cells will be evaluated for potential effectiveness using proprietary AI-based genomics tools, before being transplanted for clinical use.
