Novotech, a globally recognized full-service clinical research organization (CRO) and scientific advisory company, has released its newest preclinical report exploring the evolving landscape of small interfering RNA (siRNA) therapeutics. This report provides insights for biotech and pharmaceutical companies leveraging RNA-based precision medicine to develop next-generation treatments for oncology, metabolic disorders, and rare genetic diseases.  

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Unlocking the Potential of siRNA for Drug Development  

siRNA therapeutics represent a paradigm shift in targeted drug development by enabling precise gene silencing of disease-causing genes. With the FDA approval of multiple siRNA-based drugs, including ONPATTRO™ (Patisiran) and Inclisiran, the field is witnessing rapid advancements in delivery platforms, stability, and tissue-specific targeting. Novotech’s report outlines the current research and regulatory landscape, highlighting how siRNA technologies are expanding the druggable genome, addressing previously undruggable targets.  

Key highlights  

• Clinical and Regulatory Landscape: Overview of the six FDA-approved siRNA therapies and their expanding indications in cardiovascular, hepatic, and rare genetic diseases.
• Advancements in siRNA Delivery: Innovations in lipid nanoparticles (LNPs), GalNAc conjugates, and polymeric nanoparticles enhancing targeted tissue delivery and minimizing immune-related challenges
• Expanding Applications in Oncology: siRNA’s role in silencing oncogenes, overcoming drug resistance, and targeting tumor microenvironments, positioning it as a promising alternative to traditional cancer treatments.
• Clinical Trial Growth & Future Outlook: Analysis of the 150+ industry-sponsored siRNA clinical trials conducted between 2020 and 2024, with a 79.5% CAGR reflecting significant industry investment.

Partnering for Success in siRNA Clinical Development  

As a leading global provider of end-to-end clinical trial services, Novotech partners with biotech and pharmaceutical innovators to accelerate the development of siRNA therapeutics. With expertise in RNA research and over 25 years of experience in clinical trial execution, Novotech provides the comprehensive regulatory guidance, strategic site selection, and expert trial management to navigate the complexities of siRNA drug development-ensuring efficiency, compliance and success at every phase.  

Download the Full Report  

About Novotech Novotech-CRO.com  

Novotech is a globally recognized full-service clinical research organization (CRO) and scientific advisory company that provides biotech and small- to mid-sized pharma companies an accelerated path to market since 1997. With a global footprint spanning 30+ offices across the Asia-Pacific region, North America, and Europe, and partnerships with 5,000+ trial sites, Novotech offers unparalleled access to key clinical trial destinations and diverse patient populations. Novotech leverages its therapeutic and regulatory expertise, client-centric service model, local market insights, and advanced analytical tools to expedite patient recruitment, enhance trial efficiencies, and bring life-changing therapies to market faster. This work has been recognized by awards such as the Frost & Sullivan CRO Company of the Year, which Novotech has received for 19 consecutive years.  

For more information or to speak to an expert team member visit www.Novotech-CRO.com  

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