- Here are my quick notes from ALNY at Rodman and Renshaw 9/13/11:
- TTR: over 100 mutations described, our approach is to target all of these as well as wild-type protein [differs from Pfizer/FoldRx drug]
- 2 delivery approaches:
2) Conjugate - very excited about translating conjugate technology into pipleine as part of 5x15 as well [but first 4 of 5 programs announced use LNP...]
Question and Answer session-
- Cash sufficient for a long time. We have given no specific guidance on runway. We will be partnering ex-US for these programs, and expect a lot of costs to be shared with partners. Goal is to build US commercial infrastructure... another reason to target orphan indications
- both LNP and conjugate particles are taken up by receptor mediated endocytosis (but via different receptors), both get access to cytoplasm via "leakiness"...this is an area of research and optimization
- We believe that executing on current business plan is right way to build shareholder value...vs M&A, inlicensing, etc. based on where our technology can go