Aspen Neuroscience, a pioneering force in regenerative medicine, has received clearance from the U.S. FDA for its Investigational New Drug (IND) application for ANPD001. ANPD001 is a novel autologous (“self-derived”) cell therapy that has the potential to treat Parkinson’s Disease by replenishing lost dopamine neurons. This green light from the FDA propels Aspen Neuroscience into historic territory, initiating a clinical trial for ANPD001 in human patients.
Specifically, Aspen will be undertaking a Phase 1/2a clinical trial targeting individuals who have moderate to severe Parkinson’s Disease (PD). This IND approval follows the company’s 2022 Trial-Ready Screening Cohort Study, where cells were manufactured for potential patient candidates. This multicenter trial will mark the first Phase 1/2a trial of an autologous iPSC-derived therapy on U.S. soil.
Aspen’s inventive approach revolves around utilizing induced pluripotent stem cells (iPSCs) generated from the patient’s own skin cells. These iPSCs are harnessed to craft dopamine neuronal precursor cells (DANPCs). A stringent quality control regimen, bolstered by Aspen’s proprietary AI-driven genomic assessments, ensures the potency of these cells before implantation.
On the manufacturing front, Aspen’s three-step process starts with a small sample of the patient’s own skin cells. These cells are then reprogrammed into iPSCs and subsequently transformed into DANPCs. Via surgical intervention, these DANPCs are transplanted into the patient’s body, with the intent to restore cells that have been lost or impaired due to Parkinson’s Disease. The quality of each individual’s cells is assessed during each stage of manufacturing using Aspen’s state-of-the-art AI-driven genomic assessments.
The upcoming ANPD001 clinical trial will be a Phase 1/2a study, operating as a single-arm, open-label investigation. This dose-escalation trial will meticulously examine the safety, tolerance, and preliminary efficacy of ANPD001 in individuals grappling with moderate to severe Parkinson’s Disease.
Headquartered in San Diego, California, Aspen Neuroscience is a clinical-stage, privately company that is operating at the forefront of personalized (autologous) regenerative medicine. Fueled by patient-derived iPSC technology, the company is committed to crafting individualized cell therapies that can combat diseases with substantial unmet medical needs.
This landmark approach to PD treatment stems from the collaborative efforts of Aspen’s co-founders, Dr. Jeanne Loring, Professor Emeritus and Director of The Center for Regenerative Medicine at the Scripps Research Institute, and Dr. Andres Bratt-Leal, Aspen’s Senior Vice President of Research and Development. To read the full press announcement, click here.
To learn more about the market for iPSCs, view the “Global Induced Pluripotent Stem Cell (iPS Cell) Industry Report – Market Size, Trends, and Forecasts.”
