Seekingalpha.com did a conference call on Isis Pharmaceuticals' Q3 2011 results.
According to Isis Pharmaceuticals CEO Stanley Crooke, the company: "will begin our Phase I study in patients with spinal muscular atrophy very shortly, and we continue to move forward on our SOD1 study in patients with ALS."
Mr Crooke reconfirmed the information given in the article
Progress and Promise: The Current Status of Spinal Muscular Atrophy Therapeutics which stated that: "ISIS Pharmaceuticals plans to submit an IND (investigational new drug) in the coming months, with the goal of starting a phase 1 trial in SMA patients this winter using a single intrathecal ISIS SMNRx injection as a starting dose."
ISIS-SMNRx is an antisense drug designed to treat Spinal Muscular Atrophy (SMA). A genetic deletion of the survival motor neuron 1, or SMN1, gene is responsible for SMA. ISIS-SMNRx increases the production of the protein SMN by modulating the splicing of a closely related pre-mRNA, SMN2. Normal motor function is associated with normal levels of SMN. By altering splicing to produce SMN protein, ISIS-SMNRx may compensate for the underlying genetic defect. The FDA granted Orphan Drug designation for ISIS-SMNRx for the treatment of SMA.
Comments
Post a Comment