Innovation Insights: siRNA gene silencing

Innovation Insights: siRNA gene silencing

Summary

siRNA gene silencing is a powerful technique used in molecular biology to selectively silence the expression of specific genes. Small interfering RNAs (siRNAs) are short double-stranded RNA molecules that can bind to complementary messenger RNA (mRNA) sequences and trigger their degradation, preventing the translation of the corresponding protein. This process, known as RNA interference (RNAi), allows researchers to study the function of individual genes and potentially develop new therapeutic strategies for treating genetic diseases or cancer. By designing siRNAs that target specific genes, scientists can effectively silence their expression and investigate the resulting phenotypic changes in cells or organisms. The use of siRNA gene silencing has revolutionized the field of genetics and has the potential to lead to significant advancements in medicine and biotechnology.

siRNA, or short interfering RNA, is a non-coding RNA sequence that can inhibit gene expression, making it a key component in various therapeutic innovations. While the discovery of siRNA for targeted therapies dates back over a decade, current efforts are focused on improving stability and targeted delivery. The gene silencing capabilities of siRNA have led to its widespread use in novel therapies, with a significant number of patents set to expire in the near future.

Recent patent analysis shows a growing trend in siRNA innovation, with a particular focus on metabolic diseases, infectious diseases, neurological diseases, and cardiovascular diseases. The majority of patent activity is driven by universities and research institutions, with over 100 new entities filing patents in the past two years.

The innovation landscape is primarily dominated by companies in Southeast Asia, particularly in China, Japan, and South Korea. Among publicly listed companies, leaders in siRNA gene silencing include Alnylam, Thermo Fisher (Revvity/Dharmacon), and Sirnaomics. Additionally, there are over 50 startups in this space, with Chinese companies like Suzhou Ribo Life Sciences and Gangzhou Ribo Bio leading the way.

In the drug landscape, there are currently 799 siRNA gene silencing drugs in development, with a focus on oncology and metabolic disorders therapies. The most common route of administration is subcutaneous, and major players like Alnylam Pharmaceuticals Inc and Sirnaomics Ltd are leading the way in drug development, particularly in Phase I and Phase II trials.

Alnylam Pharmaceuticals Inc leads the way in clinical trials for siRNA gene silencing drugs, with 79 trials, followed by Arrowhead Pharmaceuticals Inc with 48 trials. Metabolic disorders trials are the most prevalent, with Hepatitis B being the top indication.

In terms of deals, there have been 334 deals totaling US$107 billion in the siRNA gene silencing sector. The United States and Ireland stand out with two major M&A deals worth $54.9 billion. The highest deal volume was observed in 2023, while the highest deal value was seen in 2020. The slight decline in 2021, followed by a slight increase in 2022, may indicate market adjustments and strategic consolidations. The United States is the major geography for deals, with a deal value of US$54.9 billion and the highest deal volume since 2020.

Key Highlights

• siRNA Gene Silencing report is a high-impact innovation area in the Pharma industry, with a growing number of patents being filed and a focus on new indications such as Oncology and Infectious diseases.
• Big pharma companies like Merck and co dominate the patent activity in Membrane protein targeting and modulation with a dip in new companies filing patents in recent years.
• Competitive intelligence shows that Alnylam leads in terms of portfolio size followed by Hanmi Science and Eli Lilly&Co. are among the leaders with strong innovation portfolios in siRNA gene silencing.
• Startups like Suzhou Ribo Life Sciences, Ractigen and Curigin are leading in terms of innovation impact and focus in the siRNA gene silencing.
• The drug landscape for siRNA gene silencing is dominated by innovator drugs, with a focus on Oncology and Metabolic disorders, and major players like Alnylam Pharmaceuticals Inc and sirnaomics Ltd leading in drug development.
• Clinical trials in siRNA gene silencing are dominated by Metabolic disorders trials, with the Alnylam Pharmaceuticals Inc leading in the number of trials.

• Innovation Insights: innovation examples by each use cases segment of various sectors to present key trends.
• Key player: This represents a sample list of key players in each use case highlighted in the report.
• Startups: This represents a sample list of emerging starups in each use case highlighted in the report.
• University: This represents a sample list of leading universities in each use case highlighted in the report.

• Drive Innovation with Our siRNA Gene Silencing Report:
• Emerging technologies like siRNA gene silencing are revolutionizing healthcare, and staying ahead requires actionable insights. Our report offers comprehensive analysis of patents, drug pipelines, clinical trials, and market dynamics, empowering strategic planning and R&D decisions. Stay informed on regulatory changes and clinical advancements critical for navigating the evolving healthcare landscape. Identify investment opportunities through detailed financial deal and funding trend analyses. Whether you're a pharmaceutical leader, biotech innovator, or academic researcher, this report delivers essential competitive intelligence to guide data-driven decisions and ensure you remain at the forefront of gene silencing innovation.