The FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to sasineprocel (ANPD001), Aspen Neuroscience’s lead investigational cell therapy for Parkinson disease (PD). The designation, which follows an earlier Fast Track designation for the therapy, provides increased FDA guidance and expedited development support, including potential eligibility for priority review and accelerated approval.
Trials show safety and functional gains. The RMAT designation is based on results from the ongoing phase 1/2a ASPIRO trial (NCT06344026), an open-label, first-in-human study evaluating bilateral intracranial delivery of sasineprocel, an autologous induced pluripotent stem cell (iPSC)-derived dopaminergic neuron precursor cell therapy, in patients with PD aged 50 to 70 years.
An earlier analysis of the first 3 treated patients at 6 months found no hemorrhages, serious intraoperative or postoperative complications, or severe graft-induced dyskinesia, with swollen tongue the most common adverse event; that analysis also reported a 45% mean improvement in MDS-UPDRS Part III OFF scores and a 71% improvement in Part II scores.
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A subsequent 12-month analysis of the first 8 treated patients, presented at the 2026 AD/PD International Conference in Copenhagen, found continued safety with no serious surgical adverse events, no severe graft-induced dyskinesia, and no symptomatic hemorrhages or infarctions. Good ON time increased by a mean of 2.1 hours in the lower-dose cohort and 2.4 hours in the higher-dose cohort, while mean MDS-UPDRS Part III OFF scores improved by 15.5 and 13.5 points, respectively; FDOPA PET imaging showed evidence of graft survival and engraftment in both cohorts.
“These findings support the potential for sustained clinical benefit without the need for chronic immunosuppression,” trial investigator Chad Christine, MD, said in a statement at the time of the reported data.
Sasineprocel is manufactured from a patient’s own skin fibroblasts, which are reprogrammed into iPSCs and differentiated into dopaminergic neuron precursor cells before image-guided delivery into the putamen. Because the cells are autologous, the approach is designed to avoid the chronic immunosuppression required for allogeneic cell therapies.
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Preclinical data supporting the program, presented at the 2023 World Parkinson Congress, showed that dopaminergic neuron precursor cells manufactured from 6 PD patient skin biopsies survived and functioned in a 6-OHDA rodent lesion model without migration or tumor formation.
Aspen is very encouraged by the results of these studies, which demonstrate the ability to manufacture and produce personalized, patient specific DANPCs in support of a future clinical study, senior author Andrés Bratt-Leal, PhD, senior vice president of R&D and cofounder of Aspen Neuroscience, said at the time.
Aspen has said it plans to advance sasineprocel into phase 3 testing later in 2026, a stage the company has described as critical for determining whether the early ASPIRO signals can be replicated in a larger, controlled population. While the current data remains promising, moving to a randomized, controlled phase 3 study is a standard requirement for the FDA to consider approval for a disease-modifying treatment.