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RNAi-Based Drug Development

This article was originally published in Start Up

Executive Summary

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.

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Galenea Corp.

Galenea Corp. was founded to develop new treatments for schizophrenia based on modulation of the calcineurin pathway. With funding for this long-term program secured at inception, to balance its portfolio, the start-up also licensed technology for developing a flu vaccine using RNA interference directed to conserved regions of the influenza virus--a nearer-term development proposition.

Galenea Corp.

Galenea Corp. was founded to develop new treatments for schizophrenia based on modulation of the calcineurin pathway. With funding for this long-term program secured at inception, to balance its portfolio, the start-up also licensed technology for developing a flu vaccine using RNA interference directed to conserved regions of the influenza virus--a nearer-term development proposition.

RNAi Enters the Clinic for Macular Degeneration

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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