Clinical Development

What we do

We are developing novel delivery systems to effectively deliver nucleic acid therapeutics in a clinical setting. Scaled-up synthesis procedures have been implemented to produce our 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 our 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 initial therapeutic focus is oncology indications involving blood and solid cancers. We recognize that the RNAi activity can be implemented in the treatment of a large range of human diseases and we work with other companies to explore new indications. 

 

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


Our R&D activity is at the preclinical stage and we are committed to undertaking the initial development of our therapeutic agents in select human disorders. We actively seek partners in blood cancer and immune cell based therapeutic areas for clinical collaborations. 

We have been adopting our delivery systems for treatment of patient-derived cells as well as preclinical animal models. Reach out to us if you desire to use our reagents in particular disease models.

Why study blood cancers and immunotherapies 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.

 

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 of malignant with the use of RNAi is providing a fresh approach to managing drug resistant cancers.

 

The immunotherapy approach to treat blood cancers involve use of antibodies, stem cell transplants, cytokines, small molecules among others [5]. A more recent approach relies 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.

 

Nanomedicine based on nucleic acid therapeutics is becoming an integral component of the personalized cancer therapies. RJH Biosciences strives to provide quality delivery systems to this end, whether it involves the delivery of our own nucleic acid candidates or our customers’.

 

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 a range of diseases.

References:

  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.