"The need for new medicines in
"We are committed to advancing new RNA-targeted therapeutics, like RNAi and antisense, to patients with high unmet medical need in
Following the identification of a development candidate, Ribo may exercise its option to license each drug by paying Ionis a license fee. For each drug that Ribo licenses, Ribo will be responsible for all development and commercialization activities and costs in
Ribo will provide Ionis a royalty-free license to the data and intellectual property created under the collaboration.
ABOUT RNAi AND ssRNAi
As it is known to occur in nature, the RNAi pathway is mediated by short double-stranded RNA oligonucleotides called "small interfering RNAs" or "siRNAs." To date, efforts aimed at harnessing this pathway to silence disease-causing proteins have used double-stranded siRNAs. Double-stranded siRNA drugs require complex formulations or special conjugates to achieve sufficient delivery for systemic activity. This requirement has severely limited the development of safe and effective drugs that work through the RNAi pathway. In contrast, single-stranded RNA-like antisense drugs can be administered subcutaneously and distribute to tissues without the need for complex formulations or special conjugates. Using its expertise in oligonucleotide chemistry and design, Ionis has discovered strategies for designing single-stranded oligonucleotides that act through the RNAi mechanism. With further development, these chemically modified, single-stranded, RNA-like oligonucleotides have the potential for improved properties for systemic administration while harnessing the advantages of the RNAi mechanism.
ABOUT SUZHOU RIBO LIFE SCIENCE CO., LTD.
Ionis is the leading company in RNA-targeted drug discovery and development focused on developing drugs for patients who have the highest unmet medical needs, such as those patients with severe and rare diseases. Using its proprietary antisense technology, Ionis has created a large pipeline of first-in-class or best-in-class drugs, with over three dozen drugs in development. SPINRAZA™ (nusinersen) is a drug that has been approved in the U.S. for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. Biogen is responsible for commercialization of SPINRAZA. Drugs currently in Phase 3 development include volanesorsen, a drug Ionis is developing and plans to commercialize through its wholly owned subsidiary,
IONIS' FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding Ionis' collaboration with Ribo, and the discovery, development, activity, therapeutic and commercial potential and safety of drugs under Ionis' relationship with Ribo. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs 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. Ionis' 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 Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended
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Investor and Media Contact: D. Wade Walke, Ph.D., Vice President, Corporate Communications and Investor Relations, 760-603-2741