Aspen Neuroscience is a clinical-stage biotechnology company headquartered in San Diego, California, focused on developing autologous cell therapies for Parkinson's disease. The company's lead product, ANPD001, is a patient-specific dopaminergic neuron replacement therapy derived from the patient's own induced pluripotent stem cells (iPSCs).
| Program |
Target/Mechanism |
Indication |
Phase |
Status |
| ANPD001 |
Autologous iPSC-derived dopamine neurons |
Parkinson's Disease |
Phase 1/2 |
Active (dRIP Trial) |
| ANPD002 |
Allogeneic iPSC-derived dopamine neurons |
Parkinson's Disease |
Preclinical |
Research |
| ANPD003 |
Encapsulated cell therapy |
Parkinson's Disease |
Preclinical |
Research |
Aspen's cell therapy platform includes:
- Autologous iPSC Therapy: Patient-specific cells derived from their own skin or blood cells
- Directed Differentiation: Precise protocol to differentiate iPSCs into midbrain dopaminergic neurons
- Gene Editing: CRISPR-based correction of genetic mutations in patient-derived cells
- Personalized Medicine: Tailored treatment for each patient
Aspen's cell therapy approach aims to replace the dopaminergic neurons lost in Parkinson's disease:
- Patient's cells are collected and reprogrammed into iPSCs
- iPSCs are differentiated into midbrain dopaminergic neurons
- Cells are quality-tested for safety and potency
- Cells are transplanted into the patient's brain
- Transplanted neurons integrate and restore dopamine production
- First-in-human trial of autologous iPSC-derived neurons for Parkinson's
- Surgical delivery to the putamen (region with dopamine neuron loss)
- Primary endpoints: Safety and tolerability at 1 year
- Secondary endpoints: Motor function improvement (MDS-UPDRS)
- No immunosuppression required (patient's own cells)
- Reduced risk of immune rejection
- Personalized treatment
- Potential for better engraftment
- 2024: Raised $150M Series B financing
- 2022: Raised $70M Series A financing
- Strategic partnership with Novartis for manufacturing
- NIH grants for Parkinson's disease cell therapy research
- Publicly traded (NASDAQ: APNE)
Aspen has built an integrated cGMP manufacturing facility in San Diego:
- Scalable autologous cell production
- State-of-the-art quality control laboratories
- Directed differentiation process optimization
- Dr. Damian B. (CEO and Co-founder)
- Dr. Andres M. (Co-founder and Chief Scientific Officer)
- Dr. Philippe G. (Chief Medical Officer)
- Dr. Jeanne F. (President of R&D)
The company's clinical approach includes:
- Screening of Parkinson's patients for suitability
- Personalized cell manufacturing for each patient
- Specialized neurosurgical delivery
- Long-term follow-up for safety and efficacy