Aiming to treat Parkinson’s disease by replacing neurons, Aspen Neuroscience raises $147 million

Patients with Parkinson’s disease lack sufficient amounts of a neurotransmitter that plays a key role in the brain’s ability to control movement and motor coordination. Although drugs are available to fill this gap, their effectiveness decreases over time. Aspen Neurosciences aims to treat Parkinson’s disease with cell therapy. Biotechnology now has $147.5 million in new funding as it prepares for a first-in-man test that could show not only how its technology works, but also whether it offers an advantage over a Bayer cell therapy already in the clinic.

The Series B funding round announced on Monday was co-led by GV, LYFE Capital and Revelation Partners.

The main brain chemical that patients with Parkinson’s disease lack is dopamine, which comes from dopamine-producing neurons. According to Aspen, about half of these neurons are lost before a patient’s Parkinson’s disease is diagnosed. The San Diego-based biotech aims to use cell therapy to replace these neurons to restore dopamine release and rebuild neural networks.

Aspen makes its cell therapy, called ANPD001, from induced pluripotent stem cells (iPSCs), which have the potential to grow into almost any type of human cell. Biotechnology induces these cells to become dopamine-producing neurons. Just before these cells mature, they are transplanted into the patient.

Aspen’s approach is personalized. The stem cells come from a skin biopsy of the patient. Before transplanting the autologous cell therapy into the patient, Aspen uses artificial intelligence to assess the potential effectiveness of the cells. The company claims that its process reduces the time and cost of manufacturing a personalized cell therapy. Aspen also claims another benefit: avoiding immunosuppressive drugs. Since the therapy is derived from the patient’s own cells, no immunosuppressive drugs are needed to prevent the immune system from rejecting the cell therapy.

Bayer is taking a similar approach to treating Parkinson’s disease. Like Aspen, the pharmaceutical giant is developing a cell therapy that would be implanted in the patient to restore dopamine production. However, Bayer Cell Therapy is allogeneic – iPSCs come from healthy donors. This standard approach is intended to offer its own benefits. Personalized cell therapies, such as CAR T therapies for cancer, require a long, time-consuming, multi-step manufacturing process. But an off-the-shelf approach could lead to therapies being readily available from a more scalable manufacturing process.

Bayer’s experimental cell therapy for Parkinson’s, DA01, spun out of BlueRock Therapeutics, a biotech joint venture the pharma giant formed with Versant Ventures in 2016. In 2019, Bayer Fully Acquired BlueRock for $240 million upfront and an additional $360 million tied to milestone achievement.

Bayer Advanced BlueRock Parkinson’s cell therapy in human testing last summer, a phase 1 clinical trial enrolling a dozen patients. These patients will take drugs to partially suppress their immune system for a year to prevent the body from rejecting the transplanted cells. Study participants will then be followed for two years to assess treatment tolerance and survival of the transplanted cells. Christian Rommel, head of research and development for Bayer’s pharmaceuticals division, told MedCity News during an interview at the JP Morgan Health Care conference in January that preliminary clinical data from the study could arrive later. next year.

According to Aspen, his laboratory and preclinical studies have demonstrated that his approach can produce dopamine-releasing neurons. The company is preparing to submit an Investigational New Drug Application to the FDA. If the agency accepts the case, the company could proceed with patient screening and a planned phase 1/2a clinical trial. Damien McDevitt, President and CEO of Aspen, said in a prepared statement that the Series B funding will also support the development of other therapies in the company’s pipeline, including autologous gene-corrected cells and programs that explore the treatment of other neurological diseases.

Since its founding in 2018, Aspen claims to have raised more than $220 million. Other disclosed participants in the latest funding include new investors Newton Investment Management, EDBI, LifeForce Capital, Medical Excellence Capital Partners, Mirae Asset Capital, NS Investment. Previous investors OrbiMed, Arch Venture Partners, Frazier Life Sciences, Section32 and Alexandria Venture Investments also participated in the Series B round.

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