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RNAi Start-Ups Continue Seeking, Attracting Support: Private Investors Like Nucleonics' Focus

This article was originally published in Start Up

Executive Summary

Nucleonics, like other companies competing in the new field of RNAi, started out with big ambitions. Even before its Series A financing ran out, the company decided to focus on clinical goals. Nucleonics has just received a $41 million financing meant to see two drug candidates, presently still in preclinical testing, get through Phase II. Investors like its focus. Meanwhile, Alnylam still has big plans, which it's now hoping to finance through the public markets.

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Nucleonics Inc.

Nucleonics Inc. has turned to plasmid DNA delivery technology as an answer to the siRNA delivery challenge. The company believes this approach could be an effective mechanism for selectively targeting viruses, and offers significant advantages over other methods for generating a gene silencing response in targeted cells

RNAi-Based Drug Development

Two issues dominate discussions over the business prospects for RNA interference-based therapeutics: the first is a basic pharmacological challenge: can a double-stranded RNAi be delivered inside a cell, and can it survive entry through the cell membrane with the physical integrity necessary to bind its target RNA and shut down protein production before it starts? The second is determining who has freedom to operate, in an area where patents enabling human therapeutic uses for RNAi are relatively fresh, and clinical data does not exist.

Alnylam Pharmaceuticals Inc.

Alnylam is a leader in the emerging field of RNA interference-based therapeutics development, and the first to land a deal with a Big Pharma (Merck). Its focus is to discover and develop therapies for a range of diseases including viral infection, oncology, and autoimmune diseases.

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