Isis Pharmaceuticals towards an antisense drug

Isis Pharmaceuticals has exclusively licensed certain intellectual property from the University of Massachusetts to develop a potential new therapy for Spinal Muscular Atrophy (SMA). Funding support for the University of Massachusetts' research program responsible for creating this intellectual property was provided in part by Families of SMA.
ISIS recently announced that it had added a SMA drug candidate to its development pipeline, called ISIS-SMNRx. ISIS is developing ISIS-SMNRx as part of its strategy to discover and develop antisense drugs against neurodegenerative diseases.
ISIS' SMA program is part of its collaboration in neurodegenerative disease with Dr. Adrian Krainer at Cold Spring Harbor Laboratory and Genzyme, pursuant to which Genzyme has an exclusive option to license ISIS-SMNRx from ISIS.

The antisense therapeutic approach for SMA involves the use of a short, chemical structure, called an antisense drug, to increase the production of the protein SMN. SMN protein is reduced in SMA and is associated with normal motor function. SMN2 is a closely-related gene that normally produces a truncated and low-functioning form of SMN protein. Isis designed an antisense drug that binds to the SMN2 RNA and drives the production of SMN protein. Using this approach, Isis hopes to provide therapeutic benefit to patients with SMA.
“SMA is a terrible disease and the leading genetic cause of infant mortality. The ability of our drugs to specifically target RNA and drive the production of SMN may be able to compensate for the underlying genetic defect and offer some therapeutic benefit to patients with SMA. We are grateful for the support provided by Families of SMA in funding early research on SMA, which has significantly expanded the basic science of SMA and provided a roadmap to developing the first targeted therapy to treat SMA,” said Frank C. Bennett, Ph.D., Senior Vice President of Research at Isis Pharmaceuticals.

(source: FSMA website)