SAN DIEGO, July 6, 2023 /PRNewswire/ -- On Wednesday at the 2023 World Parkinson Congress in Barcelona, Aspen Neuroscience co-founder Andres Bratt-Leal, PhD, Senior Vice President Research & Development, presented preclinical safety and efficacy data for the first time for ANPD001, Aspen's iPSC-derived lead candidate for treatment of Parkinson's Disease (PD).
Dr. Bratt-Leal's poster presentation, "Preclinical Safety and Efficacy of Dopamine Neuron Precursor Cells Derived from PD Donor-Induced Pluripotent Stem Cells," featured GLP safety and efficacy data in well-established rodent models.
In the studies, dopamine neuron precursor cells (DANPCs) were generated from six different skin biopsies, donated by people diagnosed with Parkinson's Disease. Multiple induced pluripotent stem cell (iPSC) clones from each individual donor were generated and characterized. These autologous iPSCs were further differentiated to DANPCs, which were then tested for efficacy in a well-known rodent model of PD. A separate study was conducted to characterize safety, biodistribution and potential tumorigenicity of the DANPCs in an established rat model under Good Laboratory Practices (GLP).
The data suggest that after transplantation there is no evidence of migration or tumor formation, and that PD neurons function in vitro and restore dopamine signaling deficits in rodents. The results demonstrate the ability to manufacture patient specific DANPCs which are safe and efficacious in support of clinical investigation.
"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," said Dr. Bratt-Leal. "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."
Aspen is developing ANPD001, an autologous cell therapy derived from individual patients' own stem cells (patient-derivediPSCs) and designed to replace lost dopamine neurons. Towards this development, the company has established the manufacturing process to produce high quality neurons (DANPCs) from patient donors under GMP conditions. Whole genome sequencing (WGS) at each manufacturing stage ensures that genomic mutations are not present in cells selected for development. RNAseq is used to predict downstream functionality of both the iPSCs (PluriTest™) and the DANPCs (NeuriTest™).
Currently there is no disease-modifying treatment for PD, the second most common neurodegenerative disease affecting more than 10 million patients worldwide. One of the potential advantages of Aspen's autologous approach is that it will not require immunosuppressive drugs, which may not be well tolerated in some patient populations.
The World Parkinson Congress provides an international forum for scientists, clinical researchers, health care professionals, people with Parkinson's, and others to come together to discuss, learn, and engage in debate around the latest scientific discoveries, medical and comprehensive care practices related to Parkinson's disease.
About Aspen Neuroscience
Headquartered in San Diego, Aspen Neuroscience, Inc. is a development-stage, private biotechnology company focused on personalized (autologous) cell therapies. The company is developing patient-derived iPSCs to address diseases with high unmet medical needs, beginning with autologous neuron replacement for PD and extending across the brain and affected organs.
A leading iPSC platform company, Aspen combines stem cell biology with the latest artificial intelligence and genomic approaches to investigate patient-specific, restorative treatments. The company has developed a best-in-class platform to create and characterize pluripotent-derived cell medicines, which includes in-house bioinformatics, manufacturing, and QC. For more information and important updates, please visit https://www.aspenneuroscience.com.
SOURCE Aspen Neuroscience, Inc.