Aspen Neuroscience scientists are showcasing their progress in developing automated manufacturing processes for induced pluripotent stem cell (iPSC)-derived neuron replacement therapies at two major scientific conferences this May. The presentations highlight advancements in the company's lead investigational program, ANPD001, an autologous cell therapy for Parkinson's disease currently in Phase 1/2a clinical trials.
The company's scientific team, led by Chief Scientific Officer Xiaokui Zhang, Ph.D., is presenting at both the International Society for Gene & Cell Therapy (ISCT) Annual Meeting (May 7-10) and the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting (May 13-17) in New Orleans, Louisiana.
Automated Manufacturing Platform for Personalized Cell Therapies
At the ISCT meeting, Aspen is focusing on its development of automated production systems for iPSC-derived cell therapies. The company is expanding capacity for autologous manufacturing at its San Diego-based GMP facility and incorporating machine learning to optimize cell quality.
Will Ansari, Ph.D., Associate Director of Technology Development at Aspen, received the US Host Region (US West) Abstract Award for his presentation on "Development of a Protocol and Machine Learning Algorithms for the Derivation of Autologous Human iPSC on an Automated Platform to Enable Clinical Semi-autonomous Manufacturing."
"We are excited to present our progress in automating the manufacture of iPSC-derived therapies," said Dr. Zhang, who is co-chairing a session on building frameworks using iPSC products and moderating a roundtable on automating manufacturing of iPSC-based therapies at ISCT.
Personalized Approach to Neuron Replacement
At the ASGCT meeting, Dr. Zhang will present during the Scientific Symposium "Next Generation Strategies for Evading Immunity in Stem Cell Therapies" on May 16. Her presentation will cover recent developments in personalized, autologous cell therapy research and development, including ANPD001.
The company's approach uses patient-derived induced pluripotent stem cells to create dopaminergic neurons that can be transplanted back into the same patient, potentially avoiding the need for immunosuppressive drugs that would be required with donor-derived cells.
Addressing the Unmet Need in Parkinson's Disease
Parkinson's disease affects approximately 10 million people worldwide and is characterized by the progressive loss of dopamine-producing neurons in the brain. Current treatments focus on symptom management rather than addressing the underlying neurodegeneration.
Aspen's investigational therapy ANPD001 aims to replace the lost dopaminergic neurons with healthy, patient-derived cells. This approach could potentially restore normal dopamine production and improve motor function in patients with Parkinson's disease.
Integrating Advanced Technologies
What distinguishes Aspen's platform is its integration of cell biology with machine learning and genomic approaches. The company has developed comprehensive in-house capabilities spanning bioinformatics, manufacturing, and quality control.
At ISCT, Aspen scientist Jeanne Drucks is presenting a poster titled "Functional characterization of GBA1 mutations in iPSC-derived midbrain dopaminergic neurons," highlighting the company's work in understanding genetic factors that may influence treatment outcomes.
"Our vision is to translate the potential of autologous therapies into safe and effective medicines to improve patients' lives by restoring lost function without the need for immunosuppressive drugs," explained Dr. Zhang.
Looking to the Future of Cell Therapy Manufacturing
The presentations at both conferences underscore the importance of manufacturing innovation in advancing cell therapies to clinical application. Automated production systems could potentially increase consistency, reduce costs, and improve accessibility of these complex treatments.
As Aspen continues to develop its platform and advance ANPD001 through clinical trials, the company's work represents a significant step forward in the field of regenerative medicine for neurodegenerative diseases.
