San Diego-based Aspen Neuroscience, Inc. announced receipt of US FDA Regenerative Medicine Advanced Therapy (RMAT) designation for sasineprocel (ANPD001), an autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neuron precursor cell therapy, for the treatment of Parkinson's disease (PD). The RMAT designation, established under the 21st Century Cures Act, is reserved for regenerative therapies showing preliminary clinical evidence of potential to address unmet medical needs in serious conditions; it provides sponsors with early and frequent FDA interactions and potential eligibility for priority review and accelerated approval.
The designation was supported by data from the ongoing Phase I/IIa ASPIRO trial (NCT06344026), an open-label, multicohort, multicenter study evaluating the safety, tolerability, and preliminary efficacy of sasineprocel in patients with moderate-to-advanced PD. At the 12-month readout — presented at the AD/PD 2026 conference in March — eight treated patients across low-dose (5 million cells per hemisphere) and high-dose (5–10 million cells per hemisphere) cohorts demonstrated numerical improvements across motor and functional measures. Mean Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III OFF scores decreased by 15.5 points in the low-dose group and 13.5 points in the high-dose group. Mean MDS-UPDRS Part II scores fell by 5.3 and 2.3 points, respectively, and PDQ-39 quality-of-life scores improved by 51.6% and 28.5%. Mean Good ON time — the period of adequate symptom control — increased by 2.1 hours in the low-dose and 2.4 hours in the high-dose cohort. No p-values were reported; the study was not powered for formal statistical testing. FDOPA PET imaging confirmed cell survival and engraftment in the putamen.
The safety profile through 12 months showed no serious surgical adverse events, no severe graft-induced dyskinesia, and no symptomatic hemorrhages or infarctions. Several patients also reduced their levodopa equivalent daily dose (LEDD), which the company cited as consistent with a potential disease-modifying effect. The ASPIRO trial has since dosed 15 patients in total across four cohorts, with Cohorts 3 and 4 using a cryopreserved commercial-ready formulation designed to support scalable manufacturing.
The RMAT designation adds to a prior FDA Fast Track designation for sasineprocel and opens a more structured regulatory dialogue ahead of the company's stated plan to initiate a Phase III study. Aspen has not disclosed the design parameters of the planned registrational trial, including whether it will incorporate a sham-surgery control arm — a design question with precedent in the Parkinson's cell therapy field.