As the world population ages, health experts expect a dramatic increase the incidence of cancer and fibrotic disease – a condition related to aging in which scar tissue builds up on internal organs such as the heart, lungs and liver and impairs their ability to function. Fortunately, advances in medical research are yielding new medications to treat these debilitating and deadly diseases. One promising technology is RNA interference (RNAi), which delivers small interfering RNA sequences (siRNAs) into cells to silence the expression of specific genes associated with the progression of disease.
Sirnaomics, based in Gaithersburg, Maryland along with operations in China, is leveraging the promise of RNAi to develop new treatments for cancer and fibrotic disease.
The discovery of RNAi dates back to 1998, when Andrew Fire and Craig Mello published their research on RNAi in worms. They received the Nobel Prize in Physiology or Medicine for their discovery in 2006. Soon after the mechanisms of RNAi were characterized, a number of scientists, pharmaceutical companies and investors began looking at it as a platform for developing novel therapeutics for a range of medical conditions. In the 20 years since Fire and Mello first shared their work with the world, there have been exciting advances in the understanding and application of RNAi technology. In 2018, US Food & Drug Administration approved the first RNAi therapeutic, Onpattro, to treat a rare yet fatal genetic disease.
The Brilliant Mind Behind Sirnaomics
One visionary to quickly grasp the potential of RNAi was Dr. Patrick Lu, a scientist with more than 25 years of experience in biomedical research and pharmaceutical drug development. As the Co-Founder and Executive Vice President of Intradigm Corporation, Lu led multiple siRNA therapeutic programs, including the first demonstration of the prophylactic and therapeutic efficacy of an siRNA drug in a non-human primate model to treat SARS. Earlier in his career, Dr. Lu served as a Lab Head and Senior Scientist in Novartis’ gene therapy division. Over the course of his career, Dr. Lu has authored more than 50 scientific papers, review articles and book chapters, and has generated a range of scientific innovations covered by more than 50 issued and pending patents.
Lu founded Sirnaomics in 2007 to realize his goal of using RNAi to develop treatments for a broader range of diseases affecting millions of people across the globe.
“With the introduction of Onpattro, RNAi therapeutics have become reality and represent a new era of targeted therapeutics,” said Dr. Lu. “Using RNAi technology to block the production of disease-causing proteins has the potential provide a drug modality with a different mechanism of action from large monoclonal antibodies and synthetic small molecule drugs.”
At Sirnaomics, Lu attracted a team of leading scientists and clinical experts, raised more than US$70 million in investment, and developed a portfolio of “first-in-class” siRNA therapeutics targeting a range of conditions. The company’s two lead compounds, STP705 and STP707( systemic version, not in the clinic), have entered clinical trials in the US and China for treatment of non-melanoma skin cancer, cholangiocarcinoma, a type of liver cancer, and hypertrophic scarring of the skin. STP705 has received Orphan Drug Designation from the US FDA for liver cancer and liver fibrosis.
A Unique Approach to RNAi Drug Delivery
Most companies working in the RNAi field are utilizing integrated lipid nanotechnology and chemical conjugation platforms to deliver siRNA drugs to target cells. However, these approaches are sometimes limited by inherent challenges in getting the drug into the target tissue and cells.
Sirnaomics is forging a difference path – applying its proprietary polypeptide nanoparticle technology (PNP) and its own lipid and chemical technologies to optimize the delivery of siRNA drugs into the tumor microenvironment (TME) and an array of liver cell type beyond hepatocytes – including Kupffer cells, liver sinusoidal endothelial cells (LSECs) and liver stellate cells – that are implicated in the progression of liver cancer and liver fibrosis.
The Current and Future Trends of RNAi Technology
Dr. Lu emphasizes that the guiding principles at Sirnaomics are “Innovation, Global Vision and Patient-Centered”.
When addressing the needs of patients, it is important to a balance between therapeutic efficacy and drug-related toxicity in order to limit the risk of drug-related adverse events. Advances of precision medicine and its PNP technology is allowing Sirnaomics to develop novel therapies that can potentially decrease the risk of adverse effects and potentially improve patient outcomes.
Lu and his colleagues are dedicated to the Company’s mission to develop novel therapeutics to alleviate human suffering and advance patient care in areas of high-unmet medical need. Sirnaomics is expanding its operations in both the US and China to help accelerate the development and commercialization of RNAi drugs that will meet with needs of a significantly large patient population.
“Every day we are working to make RNAi therapeutics a mainstream treatment option for many diseases, such as skin cancer, liver cancer, bladder cancer and fibrosis diseases,” Lu says.