RubiusTherapeutics: Pathways & Mechanism

1. Biological Pathways and Mechanism (2-3 pages) RubiusTherapeutics Red Blood Cell Therapies

A. Discussion on the biological function of the protein/pathway affected by the drug/technologyI. Describe normal biological function the protein/pathway affected by the drug/technology.

1. What type of pathway is this?
2. What are the major proteins in this pathway?
3. Are there any other pathways that are related?

II. Describe how the biological pathway or function is affected in the disease indicated by the drug technology

1. What has happened in the disease state to this pathway?
2. Is the disease caused by a mutation of a gene, an immune reaction, oranother mechanism?
3. Why does disruption of the pathway lead to disease?

1. Discussion on mechanism by which the drug technology fixes the disease state of the pathway
   1. Describe how the malfunction of the protein targeted by the drug technology is involved in disease.
      1. What is the specific effect of a malfunctioned protein?
      2. Is the protein higher or lower in disease patients?
      3. Is the protein function normal or inhibited?
      4. Is the structure of the protein affected by the disease?
      5. Are the normal interactions of the protein affected by the disease?
   2. Describe how the drug/technology works to fix this malfunctioning.
      1. Does is repair the pathway? Shut it down?
      2. Does it work at the protein level? Gene level? Some other way?
      3. Does it correct or alter a key mechanism?
      4. Does it change the structure of any of the molecules involved?
2. Include 2-3 relevant figures. Figures showing the pathways you discussed and how the drug works are extremely helpful to the discussion in addition to any other relevant citations.

https://ir.rubiustx.com/news-releases/news-release…