Technology
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Pioneering the RNAi Revolution
RNA interference (RNAi) technology is the genetic medicine modality with the highest degree of human validation. This powerful pharmaceutical technology has experienced a rapid transition from a Nobel Prize-winning discovery to marketed products that reversibly silence disease-driving genes. SanegeneBio operates under the singular vision that RNAi therapeutics will become standard-of-care across a broad range of chronic diseases, with the potential to improve quality and longevity of life for patients worldwide.
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The medical, scientific and patient communities are now witnessing RNAi at a crossroads – from benefiting small populations with genetically-defined diseases to benefiting large numbers of patients with chronic diseases. SanegeneBio has developed a fully integrated RNAi technology platform comprising four proprietary pillars: optimized RNAi payload, proprietary GalNAc technology, extrahepatic LEAD™ technology, and bispecific siRNA technology. This integrated suite enables us to systematically develop safe, potent and durable RNAi medicines which can be self-administered as infrequently as twice per year.
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Optimized RNAi Payload
Petunia: Our proprietary siRNA design tools
Standard and novel ribose and backbone chemistries
Validated by high performing clinical programs
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Proprietary GalNAc Platform
Differentiated GalNAc linker and configuration
Outperformed industry benchmarks in clinical programs
Cost and scalability advantages
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Extrahepatic LEADTM Platform
Tissue-selective, potent, and durable gene silencing outside the liver
Unique receptor ligand and enhancer chemistries
Powers internal and partnered extrahepatic pipeline
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Bispecific siRNA Technology
Combines two siRNAs in one molecule for enhanced efficacy
Accelerates nonclinical and clinical development
Compatible with both GalNAc and LEAD™-based delivery
LEAD™ (Ligand and Enhancer Assisted Delivery) Platform
for Extrahepatic Delivery
Tailored Enhancer
PK Modulation
Receptor Affinity
Intracellular Trafficking
* Composition and placement of the ligand and enhancer are unique to each LEAD™ embodiment
New Ligands
Small Molecules
Peptides
Antibodies
Targeted
Delivery
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Muscle
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Adipose
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Macrophage
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CNS
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Best-in-class potency and durability
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Defined receptor-mediated internalization mechanisms for exquisite cell-type selectivity
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Clinically validated chemistries for de-risked safety profile
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Line-of-sight to commercial-scale manufacturing
Our Pipeline
We are advancing a pipeline of RNAi therapeutics with best-in-class and first-in-class potential, targeting autoimmune, cardiovascular, obesity and metabolic diseases, and beyond.