SK Biopharmaceuticals: Parkinson's Disease Treatment Candidate Effectively Removes Causative Protein

Full-scale Development of Next-Generation Disease-Modifying Therapy Begins

SK Biopharmaceuticals has reported research results showing that its investigational treatment candidate for Parkinson's disease effectively removes the causative protein.

On June 9, SK Biopharmaceuticals announced that it had presented its latest research findings on the development of innovative Parkinson's disease therapies in an oral presentation at the international academic conference 'GBA1 Meeting 2025', held in Montreal, Canada, from June 5 to June 7 (local time).

This conference focuses on mutations in the GBA1 gene, a major genetic risk factor for Parkinson's disease, and brings together GBA1 experts and leading industry players from around the world to share the latest research trends and therapeutic strategies. SK Biopharmaceuticals was selected for the oral presentation session, which is limited to only 16 out of all poster presentations, demonstrating the quality and significance of its research.

Parkinson's disease is a representative neurodegenerative disorder in which the progressive loss of dopamine-producing cells in the brain leads to impaired motor function. It is known that approximately 5 to 15 percent of Parkinson's disease patients are closely associated with mutations in the GBA1 gene. When a mutation occurs in the GBA1 gene, the function of the GCase enzyme (which breaks down cellular waste in brain cells) is reduced. As a result, the toxic protein 'alpha-synuclein (α-synuclein, a Parkinson's disease-related toxic protein)' accumulates excessively, leading to the death of dopamine cells and worsening symptoms.

SK Biopharmaceuticals is developing 'SKPD', a next-generation Parkinson's disease treatment candidate that works by activating the GCase enzyme to suppress the accumulation of alpha-synuclein. According to the presentation, SKPD restored motor function to near-normal levels in various animal models, and its effects were sustained for up to three months after treatment ended, confirming its potential as a disease-modifying therapy (DMT) that addresses the root cause of the disease.

Additionally, SKPD was shown to effectively remove alpha-synuclein aggregates, a major cause of Parkinson's disease, by activating lysosomes (which break down cellular waste in brain cells) and autophagy (the process that removes damaged cellular components). This mechanism is attracting attention as a key factor in slowing the progression of Parkinson's disease.

Currently, SK Biopharmaceuticals is developing SKPD as an oral DMT, initially aiming to improve clinical accuracy in patients with GBA1 gene mutations. The company also plans to expand the indication to patients with Parkinson's disease who do not have GBA1 mutations in the future.

Lee Donghun, CEO of SK Biopharmaceuticals, stated, "To address the unmet medical needs in Parkinson's disease treatment, we are accelerating the development of innovative therapies that directly target the root causes of the disease," adding, "We will continue to lead the development of next-generation new drugs based on our global competitiveness."

This content was produced with the assistance of AI translation services.