World Parkinson’s Day is observed annually on April 11. In 2021, the number of people living with Parkinson’s disease (PD) in China reached 5.077 million, accounting for 38.08% of the global total. The disease is characterized by resting tremor, bradykinesia, muscle rigidity, and postural instability. Although Parkinson’s disease remains incurable, early diagnosis, standardized medication, and scientific management can effectively alleviate symptoms, slow disease progression, and help patients maintain a high quality of life.
The core pathological change in PD is the loss of dopaminergic neurons in the substantia nigra‑striatum pathway, leading to reduced cerebral dopamine levels and imbalanced neurotransmission. The primary principle of pharmacotherapy is to restore dopaminergic function. Commonly used clinical agents include: levodopa combinations, non‑ergot dopamine receptor agonists, monoamine oxidase type B inhibitors (MAO‑BIs), catechol‑O‑methyltransferase inhibitors (COMTIs), anticholinergics, and amantadine. While these drugs relieve motor symptoms, they cannot halt neuronal damage, and long‑term use may cause side effects such as motor complications (dyskinesia) and psychiatric symptoms.
New Drug R&D for Parkinson’s Disease: Diverse Therapies in Parallel
With further elucidation of PD pathogenesis and rapid advances in stem cell and gene therapy technologies, new drug development has entered a phase of rapid growth. Multiple Parkinson’s drugs are currently in progress, spanning small molecules, cell therapy, peptides, antibodies, gene therapy, and nucleic acid drugs.
• Small Molecule Drugs
AbbVie’s tavapadon has advanced rapidly, with a New Drug Application (NDA) submitted in September 2025. It selectively activates D1/D5 dopamine receptors on medium spiny neurons in the direct striatal pathway, avoiding cardiovascular and motor side effects associated with overstimulation of D2/D3 receptors.
Vertero Therapeutics’ VT‑5006 targets the gut microbial amyloid protein CsgA, interfering with CsgA‑driven α‑synuclein aggregation and inflammation, thereby reducing brain pathology and inflammation, improving motor function, and slowing disease progression. A Phase Ⅰ clinical trial was initiated in December 2025.
• Cell Therapy
Shize Bio’s XS411 Cell Injection is an allogeneic “off‑the‑shelf” cell therapy derived from iPSC‑based dopaminergic neural progenitor cells. By replenishing lost dopaminergic neurons, it reconstructs neural function at the pathological source. It is under clinical investigation in both China and the US, with Phase Ⅱ completion expected in 2026.
Bayer’s bemdaneprocel is an off‑the‑shelf allogeneic stem cell‑derived therapy. Surgically implanted dopaminergic neurons from human embryonic stem cells target specific brain regions to restore damaged neural circuits. It is in Phase Ⅲ trials; 36‑month Phase Ⅰ data demonstrated favorable safety and durable efficacy.
• Peptide Drugs
Prodegrade Therapeutics’ PDR‑001 Injection is a gene therapy using recombinant adeno‑associated virus (AAV) targeting α‑synuclein accumulation. This strategy represents a shift from symptomatic relief to pathological targeting, making it the world’s first first‑in‑class peptide degrader in clinical development.
Herantis Pharma’s HER‑096 mimics the activity of cerebral dopamine neurotrophic factor (CDNF) to promote neuronal survival and functional recovery. Phase Ib results showed good tolerability and blood–brain barrier penetration, avoiding risks of direct intracranial infusion. A PhaseⅡ trial is planned for 2026.
• Antibody Drugs
Roche’s prasinezumab is an IgG1 humanized monoclonal antibody against α‑synuclein. It clears pathological α‑synuclein aggregates, prevents their accumulation and cell‑to‑cell propagation, and slows disease progression. A Phase Ⅲ study in early Parkinson’s disease was initiated in China in January 2026.
• Gene Therapy
VGN‑R08b, developed by VGN Bio, is an AAV‑based gene therapy administered via intracerebroventricular injection to deliver functional GBA1 to neurons and glia. It restores functional glucocerebrosidase (GCase) to degrade accumulated glucosylceramide, recovering neuronal function and alleviating CNS involvement. It is in clinical trials in both China and the US.
Belief BioMed’s BBM‑P002 Injection uses stereotaxic brain delivery with a CNS‑targeted engineered AAV vector to express optimized dopamine‑synthesis genes in the bilateral putamen. It aims to achieve long‑term therapeutic effects with a single administration.
The rapid advancement of new drugs relies on in‑depth mechanistic research, breakthroughs in diverse therapies, and a highly translatable preclinical evaluation system.
Kylin Lab Empowers Anti‑Parkinson Drug R&D with Scientific Strength
Kylin Lab specializes in preclinical R&D services for central nervous system (CNS) drugs. We have built a multi‑dimensional, high‑clinical‑translation drug evaluation platform focused on PD mechanisms and targets, supporting numerous preclinical PD programs with integrated, end‑to‑end technical solutions:
•Disease Animal Models: Established standardized PD models including MPTP‑induced, 6‑OHDA‑induced, and AAV‑α‑synuclein models, supporting mechanistic research and drug screening.
•Efficacy Evaluation Platform: Integrates behavioral testing, biomarker analysis, and electrophysiological monitoring to objectively quantify improvements in motor function and neuroprotection.
•Humanized Model Platform: Uses human iPSC‑derived dopaminergic neurons and brain organoids to recapitulate authentic PD pathology, enhancing clinical translation success.
•Full‑Chain Service System: Covers model construction, target screening, functional validation, in vivo efficacy, and mechanistic research, providing one‑stop support from early discovery to IND filing.
References:
1. China Parkinson’s Disease Report 2025
2. Chinese Guidelines for the Treatment of Parkinson’s Disease (4th Edition) [J]. Chinese Journal of Neurology, 2020, 53(12): 973–981
3. Guidelines for Primary Care of Parkinson’s Disease (2019) [J]. Chinese Journal of General Practitioners, 2020, 19(1): 5–17
Kylin Lab
CNSxplore | 提供一站式神经系统药物研发解决方案
Kylin Lab is a preclinical CRO company specializing in central nervous system (CNS) diseases, dedicated to offering one stop solutions for CNS drug discovery. With a portfolio of fully-validated cellular and animal disease models, combined with comprehensive research and analytical capabilities, we empower clients to accelerate the development of innovative therapies and reduce clinical trial risks.
Kylin Lab boasts an experienced technical team well-versed in international regulations, along with high-standard experimental platforms. Our core technologies include AI-driven phenotypic screening, humanized stem cells and organoids, electrophysiology, high-throughput electroencephalography (EEG), histology, and molecular biology. Our expertise spans a broad range of areas including Alzheimer’s disease, Parkinson’s disease, depression, schizophrenia, spinal muscular atrophy (SMA), pain, and stroke etc.
Since its establishment, Kylin Lab has adhered to the philosophy of “Pioneering R&D, Leading in technology Quality-first, Customer-centric.” We have served hundreds of clients, successfully completed numerous thematic research projects and IND submissions, and established a high-quality, stable, and forward-looking efficacy evaluation system.
