SirnaGen Therapeutics: An RNAi Drug Development Company
Explore the scientific approach of SirnaGen Therapeutics, a company using RNA interference to create a distinct class of gene-silencing medicines.
SirnaGen Therapeutics is a biotechnology company focused on creating treatments using RNA-based therapies. Founded in 2019, the company operates to address chronic diseases by leveraging its specialized knowledge in gene interference technology. SirnaGen has locations in both Cambridge, Massachusetts, and Daejeon, South Korea, positioning itself within prominent biotechnology ecosystems.
Understanding RNA Interference Technology
RNA interference, or RNAi, is a natural process within cells that helps regulate which genes are active. Genes in our DNA contain the instructions for building proteins, the molecules that perform a vast array of tasks in the body. To build a protein, a gene’s instruction is copied into messenger RNA (mRNA), which then travels to the cell’s protein-making machinery. RNAi technology uses small, specially designed RNA molecules, known as small interfering RNAs (siRNAs), to intercept specific mRNA molecules.
The process works with high precision. An siRNA molecule is engineered to match a specific sequence on a target mRNA molecule. When the siRNA is introduced into a cell, it binds to this matching mRNA. This binding action signals to the cell’s own machinery that the targeted mRNA should be destroyed. By destroying the mRNA, the production of the specific protein it codes for is stopped, or “silenced.”
This ability to selectively turn off the production of harmful or disease-causing proteins gives RNAi a broad therapeutic potential. Many diseases are caused by the overproduction of a particular protein. By designing siRNAs that target the mRNA responsible for that protein, it is possible to reduce its levels and thereby treat the underlying cause of the disease.
SirnaGen’s Drug Development Platform
A primary challenge in RNAi therapy is getting the siRNA molecules to the correct cells and tissues in the body without being degraded. SirnaGen’s technology is designed to overcome these delivery hurdles, allowing for effective treatment of diseases located outside of the liver, which has traditionally been an easier target for RNAi drugs.
The company’s platform, known as SAMiRNA™, uses modular chemistry to enhance the stability and targeting of its therapeutic candidates. This approach allows for the creation of siRNA molecules that can be administered in various ways, including:
- Subcutaneous injection
- Intravenously
- Topically
- Via an inhaler
This flexibility in administration routes enables the company to pursue treatments for a wider range of conditions, including those affecting inflamed tissues, the lungs, and the brain.
Therapeutic Focus and Pipeline
SirnaGen Therapeutics is directing its drug development efforts toward immunology and central nervous system (CNS) diseases, with a particular focus on fibrotic and immunological conditions. The company is building a pipeline of potential therapies aimed at treating immunodeficiencies, autoimmune disorders, and transplant-related complications.
The company’s lead drug candidate is SRN-001, which is being developed for the treatment of idiopathic pulmonary fibrosis (IPF), a chronic and progressive lung disease. This candidate has shown promise in addressing fibrotic diseases. While specific details on the complete pipeline and the development stages of all its candidates are not extensively publicized, the focus remains on chronic diseases where there is a significant need for new treatment options.
Key Milestones and Company Progress
The company was selected to join the 2022-2023 cohort of Endless Frontier Labs (EFL) at New York University’s Stern School of Business. This inclusion in the Life Sciences Track provides the company with mentorship from experienced entrepreneurs, scientists, and investors to help scale its operations and refine its strategy.
SirnaGen has also secured support from other industry incubators and innovation hubs, including Johnson & Johnson Innovation – JLABS and the Seoul Biohub. These affiliations provide resources and networking opportunities. While SirnaGen has not yet announced major funding rounds, its participation in these competitive programs indicates external validation of its technology and potential. The company continues to advance its programs with the goal of moving its therapeutic candidates toward clinical trials.