People with Parkinson’s disease lack adequate amounts of neurotransmitters, which play a key role in the brain’s ability to control movement and motor coordination. While drugs can be used to address this deficiency, their effectiveness diminishes over time. Aspen Neurosciences aims to treat Parkinson’s disease through cell therapy.Biotechnology now has $147.5 million In the new funding, as it prepares for its first test in humans, this could show not only how its technology works, but also whether it has advantages over Bayer’s cell therapy already in the clinic.
The Series B round, announced Monday, was co-led by GV, LYFE Capital and Revelation Partners.
The key brain chemical that Parkinson’s patients 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 replace these neurons using cell therapy to restore dopamine release and rebuild neural networks.
Aspen uses induced pluripotent stem cells (iPSCs) for cell therapy, called ANPD001, that have the potential to become virtually any type of human cell. Biotechnology induces these cells to become dopamine-producing neurons. Just before these cells mature, they are transplanted into patients.
Aspen’s approach is a personal approach. Stem cells are derived from a patient’s skin biopsy. Aspen uses artificial intelligence to assess the potential effectiveness of the cells before transplanting the autologous cell therapy back into the patient. The company claims its process reduces the time and cost of manufacturing personalized cell therapies. Aspen also claims another advantage: avoiding drugs that suppress the immune system. Because the therapy is derived from the patient’s own cells, immunosuppressive drugs are not required to prevent the immune system from rejecting the cell therapy.
Bayer is taking a similar approach to Parkinson’s. Like Aspen, the pharmaceutical giant is developing a cell therapy that will be implanted in patients to restore dopamine production. However, Bayer’s cell therapy is allogeneic — iPSCs come from healthy donors. This off-the-shelf approach is designed to offer its own advantages. Personalized cell therapy, such as CAR T therapy for cancer, requires a lengthy, multi-step manufacturing process that takes a long time. But off-the-shelf approaches may yield therapies that are easily accessible from more scalable manufacturing processes.
The experimental Bayer Parkinson’s cell therapy DA01 comes from BlueRock Therapeutics, a 2016 biotech joint venture between the pharmaceutical giant and Versant Ventures. In 2019, Bayer Complete acquisition of BlueRock $240 million upfront and another $360 million for milestones.
Bayer advanced Last summer, BlueRock Parkinson’s cell therapy entered human trials, a Phase 1 clinical trial that enrolled about a dozen patients. These patients will take drugs that partially suppress their immune systems for a year to prevent the body from rejecting the transplanted cells. Study participants will then be monitored for two years to assess tolerance to treatment and survival of transplanted cells. Preliminary clinical data from the study could be released next year, Christian Rommel, head of research and development at Bayer’s pharmaceutical unit, told MedCity News on the sidelines of the JPMorgan Healthcare conference in January.
According to Aspen, its laboratory and preclinical studies have shown that its method can generate dopamine-releasing neurons. The company is preparing to submit an investigational new drug application to the FDA. If the agency accepts the application, the company can proceed with patient screening and a planned Phase 1/2a clinical trial. In a prepared statement, Aspen President and CEO Damien McDevitt said the Series B financing will also support the development of other therapies in the company’s pipeline, including autologous gene-correcting cells and programs exploring treatments for other neurological diseases.
Since its inception in 2018, Aspen said it has raised more than $220 million. Other recent funding participants disclosed include new investors Newton Investment Management, EDBI, LifeForce Capital, Medical Excellence Capital Partners, Mirae Asset Capital, NS Investment. Early investors OrbiMed, Arch Venture Partners, Frazier Life Sciences, Section32 and Alexandria Venture Investments also participated in the Series B round.
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