An Evolved Therapeutic Ecosystem

SmartPharm Therapeutics is a second-generation gene-encoded therapeutics company that has benefited from the first decade of learning on GETs.

SmartPharm has a number of key distinctives to help make gene-encoded therapeutics a clinical reality in the near future.

Some companies build their efforts around a single technology platform. However, different specific applications benefit from different types of nucleic acids. Therefore, SmartPharm utilizes both RNA and DNA platforms as well as delivery technologies that help us get the right nucleic acid to the right target to make the optimal therapeutic protein:

  • Our DNA platforms are minimally immunogenic and capable of significant and prolonged expression
  • Our RNA platforms can produce large quantities of protein for days and weeks if needed
  • We utilize a range of delivery and targeting technologies to guide nucleic acids to target cells

SmartPharm designs the GET that works best for a specific indication. We select a nucleic acid platform for the type of release criteria desired and couple that with tissue-specific targeting and release timing for optimized yield and minimal off-target effects.

A Platform Technology

Our therapies are customized for each indication.

SmartPharm focuses on technologies with reduced regulatory risk of barriers to clinical implementation

We have learned from our experience with multiple regulatory agencies (FDA, Health Canada, and China FDA) to avoid these approaches that will make it more difficult to achieve market approval with a gene-encoded therapeutic:

  • Use of viral vectors or other approaches requiring pharmaceutical-based immune suppression
  • The use of electroporation as a delivery technology
  • Complex non-viral delivery technologies that face extensive regulatory hurdles
  • Expensive or highly complex manufacturing processes

SmartPharm implements an accelerated development ecosystem

We also utilize an established network of best-in-class development partners to speed development of new therapeutics into clinical testing. We tap networks and approaches that have already proved to be able to move new candidates into clinical testing faster and less expensively than industry standards.