FDA Clears Aspen Neuroscience’s Cell Therapy IND Application for Parkinson’s Disease
23 August 2023
Aspen is to initiate the first-in-patient multicenter Phase I/IIa clinical trial of an autologous iPSC-based therapy in the United States. The company is developing patient-derived iPSCs to create autologous cell therapies that have the potential to address diseases with high unmet medical needs, starting with autologous neuron replacement for Parkinson’s disease.
The clinical development-stage company, Aspen Neuroscience, focused on autologous regenerative medicine, announced the clearance of its investigational new drug application for ANPD001, an autologous cell therapy for the treatment of Parkinson’s Disease by replacing lost dopamine neurons. The company is developing patient-derived iPSCs to create autologous cell therapies that have the potential to address diseases with high unmet medical needs, starting with autologous neuron replacement for Parkinson’s disease.
“The IND clearance of ANPD001 sets in motion a path toward a new treatment for the more than one million Americans and 10 million people worldwide with Parkinson’s disease,” said Damien McDevitt, PhD, Aspen president and CEO.
Pioneered by Aspen’s co-founders, Jeanne Loring, PhD, Professor Emeritus and Director of The Center for Regenerative Medicine, Department of Molecular Medicine at the Scripps Research Institute, and Andres Bratt-Leal, PhD, Aspen senior vice president, research and development, the autologous cell therapy approach uses induced pluripotent stem cells (iPSCs) developed from the patient’s own skin cells to manufacture dopamine neuronal precursor cells (DANPCs).
The patient’s iPSC-induced cells are then evaluated for potential activity using quality control assays, including Aspen’s proprietary artificial intelligence-based genomic tests, before being implanted via surgery. The DANPCs aim to replace the patient’s lost or damaged cells. The quality of each person’s cells is assessed at each stage of the 3-step manufacturing stage using Aspen’s proprietary artificial intelligence-based genomics tests.
“This is a major milestone in Aspen’s mission to develop and deliver personalized iPSC-derived cell replacement therapies for people with unmet medical needs, starting with Parkinson’s disease,” said Faheem Hasnain, chairman of the Aspen board of directors. “This is an exciting time for the Aspen team and the patients who have been so instrumental in enabling the company’s development.”
Aspen plans to commence a first-in-patient Phase I/IIa clinical trial for individuals with moderate to severe Parkinson’s Disease (PD).
“Our visionary team is working to make personalized regenerative medicine a reality, and we look forward to advancing this cell therapy for patients who are waiting,” added McDevitt.
To date, there is no disease-modifying therapy that can stop, replace, or prevent the loss of dopamine neurons or slow the progression of Parkinson’s Disease. By the time of diagnosis, it is common for people to have lost most cells that make dopamine in the brain. Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments.