Structure-guided RNA loading engineered exosomes designed to enable systemic, functional RNA delivery to the central nervous system and extrahepatic tissues.
SGRL-Exo™ is P.S.K Biosciences’ engineered exosome-based RNA delivery platform designed to enable systemic, functional RNA delivery to the central nervous system and extrahepatic tissues.
The platform is built on a single-clone producer cell line architecture and an endogenous biogenesis-based RNA loading mechanism. By engineering RNA-binding proteins to selectively recognize RNA secondary structures, SGRL-Exo™ enables efficient and selective RNA enrichment into exosomes without post-production cargo loading or reformulation.
This design supports high payload density, reproducible product generation, controlled batch consistency, and scalable GMP-compatible manufacturing.
Solving two of the toughest challenges in CNS delivery: brain targeting and efficient cargo loading.
Through genetic engineering, brain-targeting peptides are displayed on the exosome surface, enabling preferential accumulation in the CNS following peripheral administration. This approach may reduce the need for intrathecal dosing.
By co-optimizing exosome-associated loading proteins and nucleic acid sequence design, P.S.K Biosciences enhances siRNA loading efficiency and stability while enabling functional delivery, achieving an approximately 100,000-fold increase in loading efficiency.
Reproducible, functional, and ready for chronic CNS indications.
Programs are advancing across neurodegenerative and rare neurological diseases.
Engineered exosome-mediated delivery of HTT-targeting nucleic acid constructs to multiple brain regions.
Systemic IV delivery of SOD1 targeting RNA therapeutics to motor neurons.
Brain-penetrant engineered exosome delivery targeting Alzheimer’s disease pathways.
Many CNS delivery approaches focus primarily on BBB entry. SGRL-Exo™ is designed to go beyond brain entry by enabling functional intracellular RNA delivery after reaching the brain.
The delivery system distributes across multiple brain regions
Co-localization with NeuN indicates neuronal uptake of siRNA cargo
Systemic administration of siRNA-tExo results in broad distribution across key brain regions, and neuronal uptake shown by carrier-delivered siRNA and NeuN co-localization.
With demonstrated CNS delivery, consistent gene knockdown across key brain regions, and repeat dosing potential, SGRL-Exo™ provides a plug-and-play platform for CNS indications, supporting rapid expansion across Alzheimer’s disease, Parkinson’s disease, ALS, Huntington’s disease, and other genetically or molecularly defined neurological disorders.
