Aspen bags US$147 mln funding to advance personalized Parkinson’s cell therapy

USA – Aspen Neuroscience, a biotech developing the first autologous neuron replacement therapy for Parkinson’s disease, has closed a series B financing of $147.5 million to help get its autologous neuron replacement into a phase 1/2a study.

This second round of funding has doubled to US$147.5 million, following a US$70 million round two years ago, and it was led by venture capital firm and Alphabet spin-off GV, LYFE Capital, and Revelation Partners.

Newton Investment Management, EDBI, LifeForce Capital, Medical Excellence Capital Partners, Mirae Asset Capital, and NS Investment are among the new investors.

The funds far outnumber Aspen’s initial US$70 million in 2020, but the biotech’s mission remains unchanged: to develop transformative treatments for difficult-to-treat neurodegenerative diseases.

Aspen Neuroscience’s therapy approach

Aspen’s cell therapy, ANPD001, is created from induced pluripotent stem cells (iPSCs), which have the ability to become almost any type of human cell.


Aspen’s induced pluripotent stem cell-derived cell therapy platform generates personalized cell replacements from a patient’s own cells, removing the need for immunosuppressive therapy.

The autologous cells are obtained through a skin biopsy — skin stem cells are active throughout life for healing — and induced into pluripotent stem cells, which have the ability to differentiate into specialized cells.

The biotechnology converts these cells into dopamine-producing neurons. The cells are transplanted into the patient just before they mature. Nerve cells have difficulty communicating with muscles in the absence of dopamine.

Prior to that, the effectiveness of each patient’s stem cell-derived dopamine neurons would be assessed using proprietary artificial intelligence-based genomics tools.

Parkinson’s disease affects approximately one million Americans and ten million people worldwide.

The funds will be used for future studies, including a patient screening study and a Phase 1/2a clinical trial, both of which are subject to FDA approval.

According to a May 9 press release, the biotech now has the funds to support planned studies of ANPD001, its lead candidate for Parkinson’s disease.

A patient screening cohort study was announced in April, with multiple clinical screening sites planned across the United States.

The screening initiative comes nearly six months after the biotech appointed Xiaokui Zhang, Ph.D., as chief scientific officer.

Prior to joining Aspen, she was the executive vice president and chief scientific officer at Celularity, and she previously worked at Celgene Cellular Therapeutics and Helicon Therapeutics.

Zhang will direct future platform and pipeline investments for the autologous platform company in addition to leading current scientific programs.

Bayer’s similar approach

Bayer is taking a similar approach to Parkinson’s treatment. The pharmaceutical giant, like Aspen, is working on a cell therapy that would be implanted into the patient to restore dopamine production.

 Bayer’s cell therapy, on the other hand, is allogeneic—the iPSCs are derived from healthy donors. This off-the-shelf approach is intended to provide its own benefits.

CAR T-therapies for cancer, for example, necessitate an expensive, multi-step manufacturing process that takes a long time.

However, an off-the-shelf approach could result in therapies that are readily available from a more scalable manufacturing process.

The experimental Bayer Parkinson’s cell therapy, DA01, is developed by BlueRock Therapeutics, a biotech joint venture formed by the pharmaceutical giant in 2016 with Versant Ventures.

Bayer fully acquired BlueRock in 2019 for US$240 million up front and another US$360 million contingent on meeting milestones.

Bayer began human testing of the BlueRock Parkinson’s cell therapy last summer, with a Phase 1 clinical trial enrolling about a dozen patients.

For one year, these patients will take medications to suppress their immune systems in order to prevent the body from rejecting the transplanted cells.

Participants in the study will then be followed for two years to assess treatment tolerability and the survival of the transplanted cells.

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