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RNAi Enters the Clinic for Macular Degeneration

This article was originally published in Start Up

Executive Summary

RNAi has quickly gained a stellar reputation for its utility as a research tool, but its potential to yield safe and effective drugs has yet to be proven. Acuity Pharmaceuticals took the industry's first step toward establishing a clinical foothold in October, when its Cand5 anti-VEGF siRNA entered Phase I trials in age-related macular degeneration (AMD). Sirna followed suit in late November with a Phase I trial of Sirna-027, its own siRNA targeting VEGFR-1. IP issues remain to be settled.

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If 2006 was a breakout year for RNAi the concept, then 2007 could be a breakout year for RNAi drug development itself. In the past few years there has been a surge of newcomers to the space, in some cases sporting high-quality venture backers and boldfaced names in RNAi. Some companies have decided to play ball with the industry leaders, sublicensing Alnylam's IP around their chosen targets, for example; others have filed their own IP-either outside the Alnylam and Merck umbrellas or putting them on a relatively slow-motion collision course with the two power brokers; still others have argued that patenting novel delivery technologies will provide them with the necessary edge in licensing negotiations, should they come about, or with pharmaceutical partners.

In RNAi, Technology Proliferates Beyond the Big Two

If 2006 was a breakout year for RNAi the concept, then 2007 could be a breakout year for RNAi drug development itself. In the past few years there has been a surge of newcomers to the space, in some cases sporting high-quality venture backers and boldfaced names in RNAi. Some companies have decided to play ball with the industry leaders, sublicensing Alnylam's IP around their chosen targets, for example; others have filed their own IP-either outside the Alnylam and Merck umbrellas or putting them on a relatively slow-motion collision course with the two power brokers; still others have argued that patenting novel delivery technologies will provide them with the necessary edge in licensing negotiations, should they come about, or with pharmaceutical partners.

Merck Nabs Pole Position in RNAi With $1.1 Billion Sirna Buy

Merck's takeout of Sirna gives a boost to platform biotech investors and has allowed Merck to stake an expansive claim in RNAi. The deal caps a massive turnaround for Sirna--previously Ribozyme Pharmaceuticals--and rewards the investors who took an early chance on RNAi therapies. The move should pay off handsomely for Merck, now the leader in RNAi, provided the technology turns out to be the next Antibodies, not the next Antisense.

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