FDA grants Aspen RMAT designation for personalized dopamine neuron replacement therapy in Parkinson’s disease

FDA grants Aspen RMAT designation for personalized dopamine neuron replacement therapy in Parkinson’s disease

On July 30, Aspen Neuroscience announced that the FDA had granted RMAT designation to its sasineprocel (ANPD001) program. RMAT designation is intended for regenerative therapies that treat serious diseases and allows developers to discuss development plans with the FDA earlier and more frequently. ANPD001 is made from an individual patient’s skin cells and implanted into the part of the brain involved in movement control.

In Parkinson’s disease, the neurons that produce dopamine die. This chemical signal helps initiate and coordinate movement. To produce ANPD001, physicians take a small skin biopsy, return the cells to a stem cell state, and then use them to grow dopamine neuron progenitors.

The ASPIRO trial registry describes bilateral implantation of these cells into the putamen, a brain region where dopamine helps control movement. The procedure is performed with MRI guidance. For each participant, the team must produce a new batch of cells, grow them, verify their quality, and deliver them into the brain.

ASPIRO is designed to enroll nine participants. During the first year, researchers will primarily record health problems that occur after the procedure. They will also monitor movement and dopamine system function at the cell implantation site. The program is testing the entire personalized procedure, not only the cells themselves.

According to Aspen’s announcement, RMAT designation will give the team earlier and more frequent meetings with the FDA. If the program continues to meet the relevant requirements, the designation may provide access to expedited review pathways. The FDA will now discuss with Aspen how to obtain each patient’s cells, control their quality, and perform the surgery as one reproducible procedure.