SRPT $30 ON RNA splicing modulator, The most advanced antisense drug, mipomersen, marketing approval in Europe
Splicing & Its Role in Disease
Splicing is a normal mechanism that the cell uses in order to produce many
different, but closely related proteins from a single gene by varying the processing
of the RNA. It is estimated that of the approximately 25,000 genes in the human
genome, approximately 90 percent have alternative splice forms. In some cases,
alternative splicing results in diseases, such as SMA, where alternative splicing
inhibits the production of a functional protein necessary for normal motor function.
Antisense drugs are small (12-21 nucleotides) DNA- or RNA-like compounds that
are chemically modified to engineer good drug properties. Isis’ antisense drugs
have been evaluated extensively in both animals and man with more than 5,000
subjects dosed with Isis’ antisense drugs. Isis has conducted approximately 80
clinical trials in more than a dozen different patient populations from
cardiovascular to cancer. The most advanced antisense drug, mipomersen,
completed a broad Phase 3 program in patients with high cholesterol and is
currently being evaluated for marketing approval in Europe.
Isis’ drug, ISIS-SMNRx
is designed to modulate the splicing of the SMN2 gene to
significantly increase the production of functional SMN protein. In previously
published results, researchers showed that ISIS-SMNRx produced sustained
activity in mouse models of SMA and that target tissue concentrations were
achieved in non-human primates following a single intrathecal injection.
Isis Pharmaceuticals is a leader in antisense technology to discover and develop novel drugs. Isis’ broad drug pipeline consists of 24 drugs in development designed to treat a wide variety of diseases with an emphasis on cardiovascular, metabolic, severe and rare/neurodegenerative diseases and cancer.