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Other Research Partners
- Families of SMA/NINDS Grant – In October 2009, Paratek and Families of SMA announced a five-year cooperative agreement
with National Institute of Neurological Diseases and Stroke (NINDS) to identify and fund pre-clinical drug development up to the filing of an
Investigational New Drug (IND) Application to the FDA for Paratek’s novel small molecule RNA splicing modifier drugs for use in the rare genetic
disease spinal muscular atrophy (SMA). Over 5 years more than $4 million is available with success of the drug candidate being evaluated at key
stages of development using quantitative go/no-go milestones that have been established. The Krainer Laboratory at Cold Spring Harbor
Laboratory and the Hastings Laboratory at Rosalind Franklin University are also key collaborators in the program. The compounds under investigation
at Paratek are intended to correct RNA splicing of a low functioning back-up gene to SMN1 called SMN2, which will in turn increase SMN protein
levels. The leading drug candidate has been shown to accomplish this in a number of tissues in animal models of the disease and the activity of
optimized leads is being confirmed in vivo.
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