Clinical Trial Assesses Safety and Tolerability of Escalating Doses of ANPD001 Autologous, Dopaminergic Neuronal Cell Replacement Therapy
SAN DIEGO, Jan. 13, 2025 /PRNewswire/ — Aspen Neuroscience, Inc. has successfully completed dose escalation and the first two cohorts of patients in the ASPIRO study, an ANPD001 Phase 1/2a clinical trial in Parkinson’s Disease (PD).
The Autologous-derived Study of a Parkinson’s Investigational Regenerative therapy in an Open-label trial (ASPIRO) was launched in 2024 to assess ANPD001 in patients with moderate to severe PD.
ANPD001 is a Personalized Dopamine Neuron Cell Replacement Therapy being Studied in Parkinson’s Disease
The study is examining escalating doses, with the primary endpoint being safety and tolerability of ANPD001. Secondary endpoints include improvement in “on” time, when patients experience periods of symptom control, and improvements in motor symptoms and quality of life based on standard PD rating scales.
“This is a major milestone for the first multi-patient, multi-center clinical trial of an autologous therapy for Parkinson’s disease,” said Edward Wirth III, MD, PhD, Chief Medical Officer of Aspen Neuroscience. “We are happy to announce that to date, ANPD001 and its delivery have been well tolerated, and no serious adverse events have been observed in the first two cohorts of patients in the ASPIRO study. All patients were discharged within 48 hours, per protocol. We are now advancing the program to investigate our new commercial formulation.”
ANPD001 is the most advanced autologous investigational new cell therapy in the United States for treating Parkinson’s disease. More information about the Phase 1/2a trial is available at clintrials.gov (NCT06344026).
The study trial includes patients 50–70 years of age, and excludes patients with cognitive impairment and other comorbidities that could preclude treatment. All enrolled patients are under the care of a movement disorder specialist.
About ANPD001
ANPD001 is an investigational autologous dopaminergic neuronal precursor cell (DANPC) therapy being studied as a regenerative treatment for PD. Aspen’s personalized approach means that patients do not require immunosuppressive drugs to counteract the body’s immune response against foreign cells.
Aspen’s manufacturing process starts from a small sample of the patient’s own skin cells, followed by reprogramming to induced pluripotent stem cells (iPSCs) and then differentiation of the iPSCs into DANPCs. These DANPCs are transplanted into the putamen, replacing cells that were lost or damaged due to disease. The quality of each person’s cells is assessed at every manufacturing stage using Aspen’s proprietary machine learning-based genomics tests.
ANPD001 has received Fast Track designation by the U.S. Food & Drug Administration (FDA).
About Aspen Neuroscience
Headquartered in San Diego, Aspen Neuroscience, Inc. is a clinical development-stage, private company focused on autologous regenerative medicine. The company’s patient-derived iPSC platform is used to create personalized therapies to address diseases with high unmet medical needs, beginning with autologous neuron replacement for PD.
Aspen combines cell biology with the latest machine learning and genomic approaches to investigate patient-specific, restorative cell treatments. The company has developed a best-in-class platform to create and optimize pluripotent-derived cell therapies, which includes in-house bioinformatics, manufacturing and quality control.

Check out our AAV CDMO service to expedite your gene therapy research
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
Related News
Navega Therapeutics Receives $4 Million CIRM Grant to Advance Epigenetic Gene Therapy for Chronic Pain
SAN DIEGO, CA – February 4, 2025 – Navega Therapeutics, a pioneering biotechnology company developing cutting-edge epigenetic gene therapies, today announced a significant milestone with the receipt of a $4 million Translational Science grant from the California...
Akribion Therapeutics Secures €8 Million in Seed Financing to Advance Novel RNA-Guided Cell Depletion Technology
ZWINGENBERG, Germany, February 4, 2025 – Akribion Therapeutics, a biotechnology company pioneering a unique, RNA-guided, nuclease-based technology for programmable cell depletion, today announced the closing of an €8 million Seed financing round. The round was led by...
UF-Kure19 CAR-T Cell Therapy Demonstrates High CR Rates, Low Toxicity in R/R NHL
Treatment with UF-Kure19, a rapidly manufactured CAR T-cell therapy, led to complete responses (CR) and low toxicity in patients with relapsed/refractory non-Hodgkin lymphoma, according to data from a single-arm, mult-center phase 1 study (NCT05400109) presented at...
Opinion: Companies Vie to Develop a Hunter Syndrome Therapy That Reaches the Brain
Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio—have products in the pipeline that could improve treatment options for this rare disease. Hunter syndrome is a rare, X‐linked disease caused by a deficiency of the lysosomal enzyme...
Related Services

AAV Packaging Services
READ MORE

Off-the-Shelf AAV Products
READ MORE