Aspen gets RMAT tag for Parkinson’s cell therapy

The U.S. FDA has granted regenerative medicine advanced therapy (RMAT) designation to Aspen Neuroscience’s lead investigational product, sasineprocel, an iPSC-based cell therapy being evaluated for the treatment of Parkinson’s Disease.

The RMAT designation for sasineprocel is based on results from the ongoing phase 1/2a ASPIRO trial, which has demonstrated encouraging early clinical activity and a favorable safety profile. The study is an open-label, multicohort, multicenter trial evaluating the safety, tolerability and activity of the surgical delivery of autologous dopaminergic neuron precursor cells (DANPCs) into the putamen, the part of the brain where restoration of dopamine signaling is needed. Aspen’s personalized approach means that patients do not require immunosuppressive (IS) drugs to dampen the body’s immune response against foreign cells, which can help avoid IS‑associated adverse events, eliminate IS drug‑monitoring requirements, and enable dosing for those with contraindications to IS therapies.

Aspen closed a $115 million Series C financing round last year, which was used to fuel the continued development of sasineprocel.

Aspen is not the only company in the cell and gene space pursuing treatment for the progressive brain disorder. In April 2025, Bayer’s BlueRock Therapeutics, published positive 18-month data from its phase 1 clinical trial for its stem cell therapy, bemdaneprocel, for Parkinson’s disease. The cell therapy designed to replace the dopamine-producing neurons that are lost in Parkinson’s disease. The company treated the first patient in a pivotal phase 3 clinical trial, exPDite-2, in September 2025. MeiraGTx's phase 3-ready AAV-GAD program for Parkinson's disease delivers the GAD gene to the subthalamic nucleus via a one-time stereotactic infusion to boost GABA production and restore dysfunctional brain circuits. Commercial manufacturing is ongoing in-house. In a randomized, sham-controlled study, the high-dose group achieved an 18-point improvement on the unified Parkinson’s disease rating scale at 26 weeks, while both dose groups showed significant improvements in quality of life.