Clinical Development

What we do

We develop novel delivery systems to effectively deliver nucleic acid therapeutics in a clinical setting. Scaled-up synthesis procedures have been implemented to produce delivery systems in large quantities. Efforts to produce GMP-grade materials are ongoing. We are committed to undertaking the required safety and toxicological studies to facilitate clinical testing of the delivery systems.

Our R&D focus

The major area of focus for RJH Biosciences is to implement RNA interference (RNAi) via delivery of short interfering RNA (siRNA). Our therapeutic focus is oncology indications involving blood and solid cancers and immuno-inflammatory diseases. We recognize that the RNAi can be implemented for treatment of a large range of human diseases and we are eager to work with other companies to explore new indications. 


Another focus area is direct administration of plasmid DNA (pDNA) and mRNA to express therapeutic proteins, with applications in immunotherapy.


We have been adopting our delivery systems for treatment of patient-derived cells as well as preclinical animal models. 


A sampling of our work can be found below. Click on the images to go to the online publications. 

Studies with Patient Cells
Andrew Wu et al., 2022. Multiple gene knockdown strategies for investigating the properties of human leukemia stem cells and exploring new therapies
PMID: 34157051
Mahsa Mohseni et al., 2021. Therapeutic delivery of siRNA with polymeric carriers to down-regulate STAT5A expression in high-risk B-cell acute lymphoblastic leukemia (B-ALL)
Juliana Valencia-Serna et al., 2019. siRNA-mediated BCR-ABL silencing in primary chronic myeloid leukemia cells using lipopolymers
Studies in Preclinical (Animal) Models
Bindu Thapa et al., 2022. TRAIL Therapy for Breast Cancer Treatment by Employing Lipopolymer mRNA Delivery
PMID: 36839732
Huijing Wang et al., 2023. Safe and Effective Delivery of mRNA Using Modified PEI-Based Lipopolymers
Juliana Valencia-Serna et al., 2018. siRNA/lipopolymer nanoparticles to arrest growth of chronic myeloid leukemia cells in vitro and in vivo

Why targeting blood cancers with nucleic acid therapeutics?

There are three types of blood cancers: leukemia, lymphoma, and myeloma. Leukemia is characterized by highly proliferating, abnormal white blood cells [1]. Lymphoma and myeloma are respectively cancers of the lymphatic system and plasma cells, which greatly affect the immune system [2,3]. These cancers are difficult to treat and current drugs are limited in efficacy, especially at the end stage of the disease, with overall 5-year survival rates ranging between 38% and 90%.

The use of nucleic acid-based therapeutics can facilitate treatment of these cancers with RNAi technology and cell-based immunotherapy. Polynucleotides such as siRNA has facilitated downregulation of cancer-driving oncogenes and can be designed to target specific abnormalities in individual patients, making it a ‘personalized’ approach to therapy [4]. The possibility of inducing apoptosis and related cell-death mechanisms in malignancy with the use of RNAi is providing a fresh approach to managing drug resistant cancers.



The immunotherapy approach to treat blood cancers have more recently relied on using engineered cells, whereby the patients’ own immune cells (T-/B-/NK-cells) are modified to engage various innate mechanisms in the host [5]. This strategy is highly advantageous as immune cells can ‘seek’ and destroy the malignant cells. As the foundation of immunotherapy relies on introduction of nucleic acids into patient cells, we are RJH are tailoring our delivery systems for efficient nucleic acid delivery into primary immune cells.


To realize the potential of nucleic acid therapeutics, we look forward to working with other pharmaceutical and biotechnology companies in specific clinical indications. Our delivery systems were shown to be effective for a range of nucleic acids and we are confident that we can implement nucleic acid therapies in diverse clinical indications. Efforts to scale-up the preparation of delivery systems are underway and typical performance of the delivery systems from multiple scales of production is summarized below. Great reproducibility is evident for scaled-up batches of delivery systems with regards to binding of nucleic acids (left), particle size and charge (middle) and silencing efficiency with a therapeutic siRNA (right).

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  1. Jean, C. and Dick, J. (2005) Cancer stem cells: lessons from leukemia. Trends in cell biology. 15, 494-501.
  2. Woods, N. et al. (2006) Therapueti gene causing lymphoma. Nature. 440, 1123.
  3. Mahindra, A. et al. (2012) Latest advances and current challenges in the treatment of multiple myeloma. Nature Reviews Clinical Oncology. 9, 135-143.
  4. Uludağ, H. et al. (2016) Current attempts to implement siRNA-based RNAi in leukemia models. Drug Discovery Today. 21, 1412-1420.
  5. Zou, W. (2006) Regulatory T cells, tumour immunity and immunotherapy. Nature Reviews Immunology. 6, 295-307.