$7m milestone rounds off good 2004 for RNAi firm

The two companies first started collaborating on the development of so-called 'gene silencing' therapeutics based on small interfering RNA (siRNA) sequences in September 2003. This was followed last June by a more specific alliance seeking to develop an RNAi-based treatment for age-related macular degeneration, a leading cause of blindness.

The achievement that triggered this milestone payment was the development by Alnylam of in vivo​ RNAi technology to a pre-specified level of performance. This is significant, as delivery of RNAi therapeutics to their site of action in the body has been considered one of the primary obstacles to the development of this new technology.

The milestone payment included a $5 million equity investment and $2 million in cash.

John Maraganore, Alnylam's president and CEO, said the milestone payment rounded off a good year for the company, in which it completed its initial public offering, the initiation of the second collaboration with Merck and the publication in Nature of a study demonstrating systemic RNAi activity in vivo​.

According to market researchers Jain PharmaBioTech, the market for RNAi sequences is estimated to be currently $300 million and will increase to $400 million in 2005 and $850 million by the year 2010. The value of the drug discovery market based on RNAi - in which sequences are used for research tools in target and drug discovery, can be assessed at $500 million currently with an increase to $650 million in the year 2005 and further doubling to $1 billion in the year 2010.

Even if only a handful of therapeuticss get into the market by the year 2010, this market will expand to $3.5 billion based on revenues from sales of RNAi-based drugs.

Meanwhile Frost & Sullivan estimate target validation to generate $146.4 million or just under 50 per cent of total RNAi market revenues, followed by research with $97.6 and therapeutics with