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Our mRNAi GOLD™ Platform

Our mRNAi GOLD™ is a platform for precision engineered therapies designed to accurately target and ‘silence’ specific disease-associated genes in liver cells by using the body’s natural process of RNA interference (RNAi).

  • Improves molecular design
  • Maximizes efficacy
  • Minimizes off-target effects
  • Stabilizes molecules
  • Optimizes manufacturing
  • Robust and growing IP estate
High-quality discovery programs

The Opportunity for Our mRNAi GOLDTM
Platform is Substantial

Existing siRNA programs have only scratched the surface of the liver target space

Only ~1% of genes expressed in the liver have been targeted by publicly known siRNAs

one percent

Opportunity to identify new GaINAc mRNAi drugs targeting many of the remaining 99% (~14,000) of liver-expressed genes

Maximizing Output through Our mRNAi GOLD TM Platform

  • High quality target identification using transational genomics
  • Lower attrition rates in discovery enabled by machine learning
  • GaINAc strategic partnerships to enhance pipeline opportunities (e.g. target selection)
Targeting 2-3 INDs/yr from 2023 through our proprietary and partnered GaINAc programs