The products, ANPD001 and ANPD002, are the first-ever Parkinson’s treatments designed to use a patient’s own cells as a replacement to restore neuronal function and to modify disease progression. This approach is referred to as an autologous neuron replacement therapy.
“Our mission at Aspen Neuroscience is to develop a restorative, disease modifying autologous neuron therapy for people suffering from Parkinson disease,” Howard Federoff, MD, PhD, Aspen’s CEO, said in a press release. “We are dedicated to using a person’s own cells for replacement therapy and bringing best-in-class treatments to people suffering from Parkinson disease as rapidly as possible.”
In autologous neuron replacement therapy a patient’s own cells (usually skin cells) are reprogrammed back into a stem cell-like state, which allows the development of an unlimited source of almost any type of human cell needed, including dopamine-producing neurons, which are those mainly affected by Parkinson’s.
Because these cells are derived from patients, they do not carry the risk of being rejected once re-implanted, eliminating the need for immunosuppressive therapies, which carry serious side effects such as infections, and possibly limiting therapeutic potential.
In theory, replacing lost dopaminergic neurons with new stem cell-derived dopamine-producing ones potentially could ease or reverse motor symptoms associated with the disease.
The $70 million raised will fund the completion of studies needed to finish the IND application to the U.S. Food and Drug Administration (FDA), the application submission itself, and will help Aspen design a Phase 1 clinical trial. This includes the recruitment of patients for the trials, as well as the collection and manufacturing of their cells.
The funding also will contribute toward the collection and analysis of the data from the Phase 1 trial and will lay the groundwork for a Phase 2 trial.
Aspen also is developing a gene-editing treatment (ANPD002) for familial forms of Parkinson’s, starting with the most common genetic variant in the GBA gene, which provides instructions to make the enzyme beta-glucocerebrosidase.
This treatment differs from ANPD001 in that it also involves the genetic modification of the cells after they are collected from patients. This is done through technology that is proprietary to Aspen.
The funds raised also will contribute to the continued research and development of ANPD002.
The $70 million fundraising goal was achieved by investments from OrbiMed, ARCH Venture Partners, Frazier Healthcare Partners, Domain Associates, Section 32, and Sam Altman. OrbiMed was the primary investor during this round of fundraising.
“We are impressed by the progress Aspen has made to date against its goals to develop innovative therapies to treat Parkinson disease and encouraged by the broader investment community’s support of the company,” said Jonathan Silverstein, a managing partner of OrbiMed.
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