Aspen Neuro Adds $70M to Test Parkinson’s Cell Therapy in the Clinic

Xconomy San Diego — 

Parkinson’s disease patients experience progressive breakdown of their neurons, cells essential to movement and mental function. Aspen Neuroscience, a biotech working to develop a therapy that would reprogram those patients’ own living cells in ways that would allow the cells to reconstruct their damaged neural networks, has raised $70 million in a Series A financing round.

The 33-person company managed to land the funding amid tenuous economic times by shifting into high gear directly after the JP Morgan Healthcare Conference in San Francisco in January, the same month the first COVID-19 cases in the US were reported, CEO Howard Federoff (pictured) said in a phone interview.

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“I have a better understanding of how these emerging infections might impact on infrastructure,” he said. “The whole team came together in such a tightly aligned manner that there was no opportunity that we missed to be able to interact, accelerate, and, where possible, get investors on board.”

OrbiMed led the funding for the San Diego-based company, which raised a seed round of $6.5 million late last year.

Aspen is advancing two product candidates to treat Parkinson’s patients. The company’s lead program, ANPD001, is in preclinical development as a treatment for sporadic, or spontaneous, forms of Parkinson’s disease—the most common type.

The company’s second product candidate, ANPD002, combines autologous neuron therapy with gene editing to treat inherited forms of the condition. About 15 percent of people with Parkinson’s disease have a family history of the disorder, and a number of genes have been identified as causing it if mutated.

Parkinson’s affects more than 1 million people in North America and more than 4 million people worldwide, according to the National Institutes of Health. In the United States, the disease occurs in about 13 per 100,000 people, and about 60,000 new cases are identified yearly.

Aspen says the fresh cash will allow it to finish preclinical work on ANPD001 and, with the FDA’s blessing, move it through Phase 1 testing. The financing will also allow it to recruit and screen Parkinson’s patients for trial enrollment, make the cells they will need for those patients, and get enough data to advance into a Phase 2 study. Some of the money will also support ongoing research and development, including the ANPD002 program and R&D into possible treatments for neuroinflammatory disorders.

Available treatments for Parkinson’s disease focus primarily on restoring dopamine, the chemical produced by the cells that are degraded as the disorder progresses. Aspen aims to reprogram cells collected from a patient into immature cells, or iPSCs, further engineer them to become predisposed to mature into neurons, then return those cells to the brain. Using a cell therapy product developed autologously, or from the patients’ own cells, would avoid the risk of an immune system reaction that could occur with the use of donor cells.

Aspen says it employs proprietary genomics tools that use machine learning to reduce the time and cost of the manufacturing process needed to produce its personalized cell therapies.

The work that underpins the company was conducted in the lab of Jeanne Loring, the company’s co-founder and chief scientific officer, who also directs the Center for Regenerative Medicine at the Scripps Research Institute.

Led by OrbiMed, Aspen’s Series A round included investment from ARCH Venture Partners, Frazier Healthcare Partners, Domain Associates, Bill Maris’s Section 32, and Sam Altman, the former president of Y Combinator and CEO of OpenAI.

As part of the deal, OrbiMed’s Peter Thompson and ARCH Venture’s Tom Daniel join its board of directors. Aspen also announced that Faheem Hasnain, a veteran biotech executive who most recently co-founded San Diego’s Gossamer Bio (NASDAQ: GOSS), has joined the board as its chairman.