Aspen Neuroscience Gets CLIN2 Grant for ANPD001 from CIRM

27 June 2024

Aspen Neuroscience, Inc., a private biotechnology firm based in California, has been awarded an $8 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM). This significant funding will support clinical trials aimed at developing a novel treatment for Parkinson's disease (PD) using personalized regenerative therapies.

The awarded grant is notable as it marks the first time an autologous therapeutic approach for degenerative neurological conditions has received such support. The funds will be used to advance ANPD001, an investigational therapy that involves replacing damaged dopaminergic neurons with those derived from induced pluripotent stem cells (iPSCs).

This project represents the first U.S. multi-center, multi-patient Phase 1/2a clinical trial of an autologous neurological therapy. ANPD001 is currently being tested in a Phase 1/2a clinical trial to determine its safety and tolerability in patients with moderate to advanced Parkinson's disease. This trial is groundbreaking as it uses the autologous approach across multiple patients and centers for the first time.

Dr. Abla Creasey, Vice President of Therapeutics Development at CIRM, highlighted the significance of this clinical award. "This grant represents a pivotal step forward in Parkinson's disease treatment by advancing a personalized therapy that could potentially restore motor function in patients affected by this debilitating condition," said Dr. Creasey.

Damien McDevitt, President and CEO of Aspen Neuroscience, echoed these sentiments. "We are honored to receive support from CIRM, a true sponsor of innovation. Developing and studying ANPD001 in California is a significant milestone. Providing patients with dopamine neurons made from their own cells is a monumental advancement for personalized medicine and could have far-reaching implications for the treatment of neurodegenerative disorders."

Parkinson's disease affects over a million Americans, causing severe motor and balance issues. Current therapies only alleviate symptoms without addressing the root cause, leaving a substantial unmet need for more effective treatments. PD is characterized by the loss of dopamine neurons in the midbrain, and by the time of diagnosis, most patients have already lost a significant number of these neurons, leading to a continuous decline in motor and neurological functions.

Edward Wirth III, Chief Medical Officer of Aspen Neuroscience, elaborated on the trial's progress. "Our Phase 1/2a study has completed enrollment, and the first patient has already been treated. We'll continue dosing patients throughout the year," he said.

Aspen Neuroscience is leveraging its expertise in cell biology, machine learning, and genomics to create and optimize pluripotent-derived cell therapies. The company's platform is designed to develop personalized treatments starting with autologous neuron replacement for Parkinson's disease. They have built a comprehensive system that includes in-house bioinformatics, manufacturing, and quality control to ensure the efficacy and safety of their therapies.

The California Institute for Regenerative Medicine (CIRM) was established to accelerate stem cell treatments for patients with unmet medical needs. With a funding pool of $5.5 billion and more than 150 active stem cell programs, CIRM is one of the world's largest institutions dedicated to advancing regenerative medicine. The partnership with Aspen Neuroscience aligns with CIRM's mission to fast-track promising stem cell technologies and bring innovative treatments to patients.

In conclusion, the $8 million grant awarded to Aspen Neuroscience by CIRM is a significant step forward in the fight against Parkinson's disease. The development and testing of ANPD001 could pave the way for groundbreaking treatments that address the underlying causes of neurodegenerative disorders, offering new hope to millions of patients.

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