Intronn’s SMaRT™ technology – RNA therapeutics – represents an entirely new approach to gene therapy at the pre m-RNA level and has broad applications across medicine and biology. Principal applications include therapeutics, molecular imaging, molecular evolution and genomics. Pre-trans-splicing molecules (PTMs) are the core of SMaRT™ technology and Intronn has developed a high capacity screen to select the best PTM from millions of candidates using molecular libraries. Intronn’s technology has progressed considerably over the last three years and has recently shown in vitro and in vivo proofs of principle in the liver, in addition to its established positions in cystic fibrosis, human papilloma virus, AD and collagen disease.

Intronn’s main focus is its therapeutic pipelines, building a franchise in RNA therapeutics for the liver to address haemophilia, hypercholesterolemia, AAT deficiency (alpha 1-antitrypsin), and cystic fibrosis, as well as addressing substantial commercial opportunities in genetic diseases, cancer, and molecular imaging.

Proteome Sciences was a founder shareholder in Intronn LLC in 1996, an entity from which Intronn Inc. emerged following further US funding in 2001 and 2004. This funding should enable Intronn to finance its progress through to clinical trials, by which time it intends to enter into partnering programmes, where significant portions of clinical and commercial development, including upfront payments and sponsored research collaborations, will be provided by strategic partners.

The research at Intronn using SMaRT™ is advancing at a fast rate, in particular in relation to the RNA programmes for liver related disorders. Strong results have been generated from the high capacity screen developed last year which show considerable improvements in trans-splicing efficiency compared with traditional PTMs (pre-trans-splicing molecules). It has been particularly encouraging to see that a number of leading academic groups at Cornell and Duke in 2004 have independently used SMaRT™ successfully across a broad range of new applications and published scientific papers in the major journals. This has provided strong external validation for SMaRT™ technology.

Details on Intronn Inc. can be found at www.intronn.com