Aspen Neuroscience

Aspen Neuroscience is a clinical-stage biotech company working to develop personalized replacement cells for the dopamine-producing (DA) neurons that are lost in neurodegenerative aging of the Parkinson’s type (PD). It recently completed a Phase I trial in which patients received transplants of replacement cells derived from their own skin cells.

We depend on a tiny, fragile population of neurons in a part of your brain called the substantia nigra sub compacta (SNc) for control of fine movements. This specific subpopulation of neurons specializes in signaling with the now-famous messenger molecule dopamine. While dopamine is popularly known as a molecule involved in motivating us to seek out things that were intensely positive in the past, these neurons use dopamine to both initiate vigorous movements and to keep our movements from “overshooting.” Throughout our lives, DA neurons slowly die as a result of aging processes, and when too many of them are lost, we develop PD. The most obvious symptoms of PD involve the loss of this fine control, causing people’s heads or hands to shake throughout their waking hours and their faces to freeze into an expressionless mask.

The most direct potential solution to this problem is to transplant new neurons to replace those lost to aging and PD. Researchers have been working on this goal since the 1980s, starting with cells derived from the early brain tissue of aborted fetuses. A few of the volunteers in these early trials experienced improvements so remarkable that they stopped taking their medications for years, and a subset of the transplanted neurons continued producing dopamine for up to 24 years after transplant. But many got little or no benefit, and some experienced side effects from the abnormal function of the cells or from non-DA neurons being transplanted as part of the crude fetal tissue material, leading to involuntary jerky movements that are more typical of late-stage PD.

Aspen Neuroscience grew out of a crowdfunded nonprofit effort by Scripps Institute regenerative neuroscientist and erstwhile SENS Research Foundation member Jeanne Loring. The nonprofit’s goal was to fund trials of patient-specific immature DA cells created via reprogramming technology from each patient’s own cells. Aspen scientists take deep-layer skin cells from people with PD and use reprogramming biotechnology and a proprietary platform to derive and validate DA neuronal progenitors intended for transplantation. These cells, which they refer to as ANPD001, are thus a perfect immunological match for each patient, eliminating the need to suppress the immune system.

After successful animal testing in 2003 using cells derived from several different human PD patients, ANPD001 was quickly granted fast-track status by FDA, and soon entered early-stage clinical testing in patients with moderate to severe PD.

The ASPIRO Phase 1/2a trial of ANPD001 is still ongoing, but in May 2025, the company announced that it had collected 6-month data from the first 3 patients, and Aspen scientists reported the results at a scientific conference and released ameeting abstract in a scientific journal. Aspen scientists delivered 5 million DA progenitors into the brains of these first three patients, using MRI to guide the implantation. This was substantially more cells than had been used in previous trials of reprogrammed or embryonic stem cell-derived DA neurons.

And it seems to have been a good investment: just six months after transplantation, volunteers enjoyed a remarkable 45% average improvement in their MDS-UPDRS part III PD score during the period when their medications were wearing off. (The MDS-UPDRS part III score is one of the more objective tests of PD function). While this result is in a mere 3 subjects, this appears to be a much greater improvement in just six months than was seen in much longer trials by other groups, and with a hint that the patients were still enjoying improvements at the time the trial was reported.

There were no serious side effects from the surgery, though two out of the three transplant recipients’ tongues became swollen.

In September 2025, Aspen announced that they had dosed their first subject in Cohort Three in ASPIRO, which for the first time used their commercial formulation of ANPD001, which enables scalable, consistent biomanufacturing of the therapeutic cell product for future use in larger clinical trials and eventually for commercial production.

In November 2025, Aspen announced that they had completed a $115 million Series C financing round co-led by OrbiMed, ARCH Venture Partners, and others. This brought their total capital raised to date to over $340 million, including an $8 million grant from the  California Institute for Regenerative Medicine (CIRM). The funding will be used to support ongoing clinical trials with ANPD001, scale their biomanufacturing capabilities, and advance their pipeline of autologous reprogrammed cell therapies for additional neurological indications.

In January 2026, Aspen announced that it had completed construction of its current GMP manufacturing facility, providing it with a scalable, consistent supply of cells produced as a cryopreserved “thaw‑and‑inject” drug product, which can be ready for dosing upon arrival at the surgical center rather than requiring additional processing before surgery. Additionally, company scientists had completed dosing Cohort Three in ASPIRO.