Parkinson Disease iPSC-derived Therapy ANPD001 Cleared for Clinical Trial in the US


Aspen Neuroscience noted that the study will be the US’s first multicenter phase 1/2a clinical trial for an autologous iPSC-derived therapy.

Aspen Neuroscience has received clearance from the FDA for an investigational new drug (IND) application for ANPD001, an investigational autologous induced pluripotent stem cell (iPSC) derived therapy intended to treat Parkinson disease (PD).1

ANPD001 consists of dopamine neuronal precursor cells (DANPCs) produced from iPSCs that are in turn derived from samples of patients’ own skin cells. ANPD001 is implanted surgically and is intended to replace dopamine neurons that have been damaged or destroyed by the disease. Aspen noted that it will use a set of proprietary artificial intelligence-based whole genome sequencing and RNA sequencing tests to check for mutations and downstream functionality for the main stages of the therapy’s manufacturing process.1,2

“The IND clearance of ANPD001 sets in motion a path toward a new treatment for the more than 1 million Americans and 10 million people worldwide with Parkinson’s disease,” Damien McDevitt, PhD, the president and CEO of Aspen Neuroscience, said in a statement.1 “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.”

The company plans to conduct a first-in-human phase 1/2a clinical trial for ANPD001 at multiple sites in the United States for patients with moderate to severe PD. Notably, it will be the US’s first multicenter phase 1/2a clinical trial for an autologous iPSC-derived therapy. Prior to filing the IND application, in April 2022, Aspen initiated a "Trial-Ready Screening Cohort Study”in order to screen potential participants for the phase 1/2a trial and provide them with relevant information.1,3 The screening study also allowed for the initiation of advanced manufacturing of the therapy from candidate patient samples.

“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,” Faheem Hasnain, chairman of the Aspen board of directors, said in a statement.1 “This is an exciting time for the Aspen team and the patients who have been so instrumental in enabling the company’s development.”

In July, Andres Bratt-Leal, PhD, the cofounder and senior vice president of research and development at Aspen, presented data from preclinical efficacy and safety research regarding ANDPD001 at the 2023 World Parkinson Congress, held July 4 to 7, in Barcelona, Spain.2 The research utilized DANPCs differentiated from iPSCs derived from skin samples from 6 patients with PD. These DANPCs were evaluated for efficacy in a rodent model for PD; restoration of dopamine signaling defecits in this model were reported. Furthermore, in a separate Good Laboratory Practices rat model used for safety assessment, no indication of migration or tumorgenicity was observed.

“At the time of PD diagnosis, it is estimated that, for many patients, most of their dopaminergic neurons in the substantia nigra and their projections in the putamen are lost, resulting in the motor symptoms of the disease,” Bratt-Leal said in a July 2023 statement.2 “Aspen is very encouraged by the results of these studies, which demonstrate the ability to manufacture and produce personalized, patient specific DANPCs in support of a future clinical study.”

1. Aspen Neuroscience announces FDA clearance of investigational new drug application for ANPD001, autologous cell therapy for the treatment of Parkinson’s disease. News release. Aspen Neuroscience. August 8, 2023. Accessed August 11, 2023.
2.Preclinical safety and efficacy data for ANPD001 presented by Dr. Andres Bratt-Leal at 2023 World Parkinson Congress in Barcelona. News release. Aspen Neuroscience. July 6, 2023. Accessed August 11, 2023.
3. Aspen Neuroscience launches first patient screening study for planned clinical trial of personalized cell replacement in Parkinson’s disease. News release. Aspen neuroscience. April 12, 2022. Accessed August 11, 2023.
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