This press release includes forward-looking statements regarding Isis' plans for discovery and development of antisense drugs for Huntington's disease and other neurodegenerative disorders, and about its collaboration with CHDI. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs, including the planned commercialization of mipomersen is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2010 and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.
Morpholino oligos have excellent antisense properties compared to other gene knockdown systems . Microinjection or electroporation of Morpholino oligos into the embryos of frogs, zebrafish, chicks, sea urchins and other organisms successfully and specifically shuts down the expression of targeted genes, making Morpholinos an indispensable tool of developmental biologists. Morpholinos have also proven their versatility and efficacy in cultures of primary or immortal cells when delivered by Endo-Porter , electroporation or Vivo-Morpholinos . Usually, Vivo-Morpholinos are used to bring the specificity and efficacy of Morpholino oligos to experiments requiring systemic delivery in adult animals. The list of over 8500 publications using Morpholinos is growing daily and is maintained on-line in a browseable database.
Cath Stanley, Chief Executive of the Huntington’s Disease Association of England and Wales, welcomed the news that the first doses of IONIS-HTT Rx had been given to Huntington’s patients. “As well as being desperate for good news, the Huntington’s community is uniquely well-informed and engaged with progress in research across the world,” she said. “RNA-targeting approaches are especially exciting because they tackle the problem at its source – the production of the mutant huntingtin protein. The IONIS-HTT Rx trial has been eagerly awaited for many years and we hope that the news from the trial continues to be positive.”