Parkinson's disease (PD), as the second most common neurodegenerative disease worldwide, has long relied on dopamine replacement therapy for treatment, but it cannot delay the progression of the disease. In recent years, with breakthroughs in technologies such as gene editing, cell regeneration, and new preparations, global research teams have made multiple milestone advancements in the treatment of Parkinson's disease. The following are the six most representative achievements from 2024 to 2025, covering the entire chain of innovation from preclinical research to new drugs on the market.

① China's first "off-the-shelf" iPSC cell drug has been approved for clinical trials simultaneously in China and the United States

The world's first allogeneic universal IPSC-derived dopaminergic neural precursor injection (XS-411) developed by Shize Biotech was approved by the Food and Drug Administration of China and the United States in January 2025 to conduct registration clinical trials. This therapy achieves "ready-to-use" supply through HLA matching optimization and immunomodulatory techniques. After transplantation, the cells can survive for a long time in the patient's brain and establish functional synaptic connections. Early clinical data show that the abnormal fluctuation time during the "switching period" of patients has been shortened by 60%, and the MDS-UPDRS score has decreased by 40%. It is expected that the new drug marketing application will be submitted in 2028.

② The 6-month follow-up data of autologous iPSC therapy is impressive

The ANPD001 therapy of Aspen Neuroscience in the United States reprograms the patient's autologous skin cells into IPscs, differentiates them into dopaminergic neural precursor cells and then transplants them. The phase I/IIa data released in 2025 showed that the patients' motor function scores improved by 45%, their activities of daily living increased by 71%, and they did not require immunosuppressants, demonstrating excellent safety.

Gene therapy: AAV vectors achieve "one-time administration, long-term effectiveness"

The gene therapy drug BBM-P002 injection of Belief Medicine was granted tacit approval for clinical trials by the NMPA of China in May 2025. This therapy delivers the dopamine synthesis gene to the putamen of the patient's brain through an engineered AAV vector to achieve continuous expression. Preclinical studies have shown that its delivery efficiency to the nervous system is significantly higher than that of similar products, with a lower dosage, and it is expected to break through the limitation of traditional drugs that need to be taken for a long time.

Long-acting sustained-release microsphere formulation: The world's first weekly formulation launched on the market

Luye Pharma's "Jinyouping ®" (rotigotin microspheres for injection) was approved for marketing in China in June 2024. This drug was developed based on the concept of continuous dopaminergic stimulation (CDS). It is administered once a week, and the fluctuation of blood drug concentration is reduced by 80% compared with the traditional daily dosage. Phase III clinical trials showed that the patient's motor symptoms continued to improve, and the duration of the "critical period" was significantly shortened. n protein: Initial efficacy of immunotherapy has emerged

The UB-312 antibody drug developed by Vaxxinity in the United States successfully reduced the level of α-synuclein protein aggregates in cerebrospinal fluid by 20% in early clinical trials (only 3% in the placebo group). This therapy selectively eliminates toxic protein clumps while retaining functional monomers, providing a new direction for "etiological treatment" targeting pathological mechanisms.

Natural product research: The antioxidant potential of Curry leaf Extract

In May 2025, an animal experiment found that the extract of Murraya koenigii leaves significantly improved the motor function of rotenone-induced PD mouse models and increased the dopamine level in the striatum by activating the Nrf-2/Sod-2 pathway. At a dose of 200 mg/kg, the damage of dopamine neurons in the substantium nigra was reduced by 58%, laying the foundation for the clinical application of natural antioxidants.

Basic research: The neuroprotective mechanism of the USP2-FOXC1 axis

A team from Zhengzhou University discovered that the deubiquitinating enzyme USP2 inhibits neuronal apoptosis, inflammation and oxidative stress by stabilizing the transcription factor FOXC1. In the MPP+ -induced PD cell model, overexpression of USP2 increased cell survival rate by 37% and reduced ROS levels by 58%, providing a new target for the development of small molecule activators.

Outlook: From "treating the symptoms" to "addressing the causes", the treatment of Parkinson's disease has entered a new era

Currently, the treatment of Parkinson's disease is shifting from single symptom management to multi-dimensional intervention:

- Cell and gene therapy → Targeting nerve regeneration and functional reconstruction;
- Long-acting preparations and immunotherapy → Optimizing treatment compliance and pathological mechanism intervention;
- Research on natural products and targets → Expanding the source of drug development.

Conclusion

Although some therapies still need to be verified through large-scale clinical trials, these breakthroughs undoubtedly ignite hope for over 10 million patients worldwide. In the next decade, Parkinson's disease is expected to transform from an "irreversible" terminal illness into a chronic disease that can be delayed or even partially reversed.