Korean biotech companies are ramping up efforts in the central nervous system (CNS) therapeutics field, with a particular focus on Alzheimer’s disease. Traditionally focused on oncology and metabolic diseases, the industry is now pivoting toward the complexities of neurodegenerative disorders, leveraging a variety of mechanisms and advanced blood-brain barrier (BBB) delivery technologies to compete globally.

Alzheimer’s disease, marked by multifactorial pathophysiology—including amyloid-beta plaques, tau protein phosphorylation, synaptic loss, inflammation, and BBB dysfunction—has seen limited therapeutic progress despite recent U.S. FDA approvals targeting amyloid clearance. In response, Korean companies are diversifying treatment strategies with novel targets and innovative delivery platforms.

As the BBB poses a major hurdle to CNS drug delivery, Korean biotechs are developing platforms that shuttle drugs across the barrier or stabilize its integrity.

ABL Bio is advancing Grabody-B, a BBB shuttle platform targeting the insulin-like growth factor 1 receptor (IGF1R) to enable receptor-mediated transcytosis of antibodies into the brain. Preclinical studies in aged mouse models demonstrated sustained brain penetration, and the data will be unveiled at the 2025 Alzheimer’s Association International Conference (AAIC). ABL Bio’s licensing deal with GSK, signed in April and valued up to $2.9 billion, validates the platform’s commercial potential.

LIS Cure has developed ExoPN-101, an exosome-based delivery platform capable of transporting ASOs, siRNAs, and small molecules with high biocompatibility and reduced immunogenicity. In non-human primate studies, ExoPN-101 showed superior brain delivery compared to other shuttle technologies. The company is in active collaboration and licensing discussions with global pharmaceutical firms, backed by material transfer agreements (MTAs).

Curacle is taking a different approach with CU71, a candidate that stabilizes the BBB and restores cerebrovascular integrity. By enhancing tight junction proteins and reducing amyloid accumulation and inflammation, CU71 showed preclinical superiority over donepezil in memory restoration. Curacle has filed for international patent protection and will present the findings at AAIC 2025.

With Alzheimer’s treatments requiring long-term dosing, Korean companies are prioritizing formulation innovation to improve patient adherence.

G2GBIO’s InnoLAMP platform enables high drug loading and extended-release delivery in a once-monthly subcutaneous injection. Its lead assets, GB-5001 and GB-5001A, completed Phase 1 trials in Canada and South Korea and aim to improve adherence by maintaining efficacy with a single monthly dose.

AriBio’s AR1001 is an oral small-molecule therapy that targets multiple Alzheimer’s pathways, including PDE5 inhibition and tau phosphorylation suppression. The drug showed cognitive improvement in a U.S. Phase 2 study and is currently in global Phase 3 trials across 13 countries. In advance of topline results expected in 2026, AriBio secured a $590 million licensing deal with a UAE sovereign fund affiliate, raising its total deal value to $1.4 billion.

Korea’s biotech sector is accelerating its push into Alzheimer’s therapeutics through differentiated approaches in BBB penetration and formulation technologies. As platforms mature and clinical milestones are met, global partnerships and trial outcomes will determine which firms can stake a lasting claim in the competitive CNS market.