P.S.K Biosciences Presents Engineered Exosome RNA Delivery Platform at BIOCHINA Global Forum @ USA
Forum @ USA, a global biopharma exchange event held alongside the BIO International Convention. The forum brought together innovative biotech companies, investors, pharmaceutical BD leaders, and industry experts to promote cross-border collaboration and global translation of China’s biopharmaceutical innovation.
During the forum, Dr. Tinghe Wu, Founder and CEO of P.S.K Biosciences, presented the Company’s latest progress in engineered exosome-based RNA delivery, with a focus on brain-penetrating delivery for central nervous system (CNS) diseases and extrahepatic tissues.
Addressing the CNS Delivery Challenge
Nucleic acid therapeutics, including siRNA, ASO, mRNA, and gene-editing technologies, have rapidly advanced over the past decade. However, delivery remains one of the most critical barriers to clinical translation, particularly for CNS and extrahepatic applications.
Existing delivery technologies, including lipid nanoparticles, viral vectors, and conjugated delivery systems, have achieved important progress in selected indications. Yet they continue to face challenges in CNS delivery, including limited brain distribution, insufficient tissue penetration, restricted repeat dosing, potential immunogenicity, and long-term safety concerns.
For neurodegenerative and CNS-related diseases such as Alzheimer’s disease, Parkinson’s disease, ALS, and post-stroke repair, therapeutic targets are often located within brain parenchymal cells or specific brain regions. This makes efficient, repeatable, and controllable delivery across the blood-brain barrier a key challenge for next-generation CNS drug development.
Engineered Exosomes as a Next-Generation RNA Delivery Platform
P.S.K Biosciences is developing an engineered exosome-based RNA delivery platform designed to overcome key delivery barriers in the CNS and extrahepatic tissues.
Exosomes are naturally secreted nanoscale vesicles with favorable biocompatibility, complex membrane structures, and intrinsic potential for cellular uptake and tissue distribution. Compared with synthetic nanoparticles, exosomes may offer advantages in cellular internalization, biodistribution, and repeat dosing. Compared with viral vectors, exosomes may also provide a non-viral delivery approach with potential advantages in safety and CMC controllability.
Built on the natural delivery properties of exosomes, P.S.K Biosciences further enhances targeting, payload loading, and therapeutic delivery through engineering strategies. During the presentation, Dr. Wu highlighted that the platform has achieved an approximately 100,000-fold improvement in nucleic acid loading efficiency, with a BP ratio of 2.0, and demonstrated robust target gene knockdown across multiple brain regions with repeat dosing feasibility.
These results support the potential of engineered exosomes not only as natural vesicle carriers, but also as a programmable, scalable, and functionally validated RNA therapeutic delivery platform.
Platform Reproducibility Across Multiple Targets
For a delivery platform, success in a single target is not sufficient. A truly scalable platform must demonstrate consistent performance across different RNA sequences, therapeutic targets, and disease settings.
P.S.K Biosciences’ technology has shown strong consistency and reproducibility across multiple targets, including both nucleic acid loading efficiency and target gene silencing performance. This supports the platform’s potential for multi-program expansion and continued technology iteration.
Rather than developing a single exosome-based therapeutic product, P.S.K Biosciences is building a platform capable of carrying multiple RNA modalities and adapting to a broad range of disease-relevant targets.
From Technical Innovation to CMC-Ready Development
The clinical success of nucleic acid delivery technologies depends not only on early efficacy signals, but also on whether the platform can support scalable manufacturing, quality control, and regulatory development.
P.S.K Biosciences has integrated CMC considerations into the design of its engineered exosome platform from the beginning. By controlling producer cells, engineering design, RNA loading, purification processes, potency assays, and critical quality attributes, the Company aims to establish a biologics-like manufacturing and quality control framework for exosome-based therapeutics.
This was one of the key messages delivered during the BIOCHINA Global Forum: engineered exosome delivery should not remain only a scientific concept, but should be developed into a next-generation therapeutic platform with clear CMC logic, quality systems, and industrial scalability.
Supporting Long-Term Treatment for Chronic Diseases
CNS disorders and many chronic diseases often require long-term treatment and repeat dosing. Therefore, delivery systems must support safety, controllability, repeat administration, and consistent manufacturing.
The repeat dosing feasibility demonstrated by P.S.K Biosciences’ engineered exosome platform provides an important foundation for chronic neurological diseases, long-term RNA therapy, and broader extrahepatic delivery applications. With biologics-like CMC and quality control advantages, the platform is well positioned for long-term treatment strategies and industrial-scale development.
Advancing Global Collaboration
BIOCHINA Global Forum @ USA provided an important opportunity for P.S.K Biosciences to present its engineered exosome RNA delivery platform to the international biopharma community. The Company’s participation reflects its continued commitment to advancing China-originated delivery innovation toward global collaboration and clinical translation.
Looking ahead, P.S.K Biosciences will continue to develop its engineered exosome RNA delivery platform for CNS and extrahepatic applications, accelerate preclinical validation and translational development, and actively explore partnerships with global pharmaceutical companies, research institutions, and industry partners.
Through continued innovation in engineered exosome delivery, P.S.K Biosciences aims to help address major delivery bottlenecks in nucleic acid therapeutics and provide new solutions for CNS and chronic disease treatment.
P.S.K Biosciences
Corporate Communications
June 21, 2026