Unixell’s off-the-shelf stem cell therapy cleared for US clinical trials

Clinical trials of UX-DA003, an experimental stem cell therapy for Parkinson’s disease developed by Unixell Biotechnology, can now begin in the U.S. following clearance from the Food and Drug Administration (FDA).

The therapy aims to replace dopaminergic neurons, which are the nerve cells lost in Parkinson’s. It uses induced pluripotent stem cells (iPSCs) derived from mature cells of a healthy donor and reprogrammed in the lab into a stem-cell-like state. The company is also developing UX-DA001, a similar treatment that uses the patient’s own cells to obtain iPSCs.

In both cases, the iPSCs are differentiated into dopaminergic neuron precursor cells, which are implanted into the patient’s brain, where they are expected to develop into dopaminergic neurons.

Recommended Reading

Dec. 17, 2024 Columns by Jamie Askari

How others (and I) can best respond to a difficult diagnosis

Simultaneous global development

The FDA’s decision comes shortly after a similar approval was granted by the regulatory agency in China, allowing for parallel clinical development of the therapy in both countries. UX-DA001 is currently being tested in a Phase 1 clinical trial (NCT06778265) in China.

“The FDA IND clearance of UX-DA003 is a critical step in our global clinical layout,” Lanlin Wu, Unixell’s CEO, said in a company press release. “We will continue to advance parallel clinical development in China and the U.S., and push forward the global commercialization of innovative iPSC-based therapies for neurodegenerative diseases.”

Parkinson’s is caused by the gradual loss of dopaminergic neurons, which produce dopamine, a signaling molecule involved in motor control. As these neurons die, patients typically develop motor symptoms such as tremors, rigidity, and slowed movements, as well as nonmotor symptoms.

Replacing these neurons could help delay disease progression and alleviate symptoms. One approach, such as UX-DA003, uses iPSCs derived from a healthy donor and is known as an allogeneic therapy. This enables a scalable, off-the-shelf treatment because cells from a single donor can treat multiple patients.

In contrast, UX-DA001 uses iPSCs derived from the patients themselves, which is called an autologous therapy. This personalized approach reduces the risk that the patient’s immune system will reject the treatment.

Both therapies build on Unixell’s technology called Single-Cell Split Barcoding lineage tracing platform, which allows researchers to track the fate of individual progenitor cells, as well as a high-precision stem cell differentiation platform, according to the company’s website.

Preclinical studies show that the proportion of functional dopaminergic neurons surviving after transplantation reaches 50% to 60%, with promising long-term safety. The cells demonstrated a low proliferation rate of 0.23% after six months, indicating a lower risk of the uncontrolled growth that can lead to tumor formation, which is a potential complication of stem cell therapies.

Additionally, because of optimized cell purity, the therapy required a 50% to 80% lower dosage than other cell therapies.

“Autologous and allogeneic iPSC therapies target distinct clinical scenarios for Parkinson’s disease. Our dual-pathway strategy not only provides flexible, individualized treatment options for patients but also accelerates the scalable industrialization of iPSC regenerative medicine,” Wu said.