Novosom AG (HALLE, Germany) announced that it has purchased an exclusive option from Isis Pharmaceuticals, Inc (CARLSBAD, California) that, if exercised within 6 months, enables the company to acquire an exclusive, worldwide license to antisense inhibitors targeting the CD40 receptor for all indications. CD40 is a membrane protein expressed on antigen-presenting cells (APCs), and is a validated target for inflammatory diseases and B-cell cancers. CD40 is also a receptor for CD154, expressed on T-cells. The binding of CD40 to CD154 activates the APCs and produces a variety of downstream effects. CD40/CD154 signaling also occurs between T- and B-cells, stimulating the latter for proliferation and differentiation into antibody-secreting plasma cells. Novosom plans to target CD40 for indications such as Crohn's disease, transplant, rheumatoid arthritis (RA), multiple sclerosis (MS), and cancer. If acquired, the license from Isis would include rights to the target and to oligonucleotides targeting CD40 and incorporating Isis' second generation antisense chemistry, 2' MOE (2'-O-methoxy ethyl/phosphorothioate) oligonucleotides, in exchange for financial considerations including an upfront exercise fee, milestone payments, and royalties.

Novosom is using its proprietary Smarticles® technology to enable systemic delivery of antisense inhibitors targeting CD40. The company has completed a series of preclinical studies that establish the in vivo safety of the formulation as well as the superior efficacy relative to TNF-α antibodies in a variety of inflammatory disease models (Crohn's disease, RA, and transplant). This encapsulated antisense approach has demonstrated targeted delivery to specific cell types with a rapid onset of action and enhanced in vivo efficacy. Novosom's Smarticles/CD40 antisense combination has achieved targeted delivery that is specific to APCs. The company plans to conduct further preclinical investigational new drug studies in models of psoriasis, MS, and cancer.

Novosom is working with its partners to develop unique antisense, DNAi- and siRNA-based therapeutics with a current focus on inflammation, oncology, and liver diseases. Smarticles liposomal vectors allow intracellular delivery of their patrners' active substance (siRNA, antisense, and decoy agents) for topical or systemic applications.

—A. Techman

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