Description of Invention: This technology discloses compounds that modulate the amount of Survival Motor Neuron protein (SMN). Low levels of SMN protein are associated with Spinal Muscular Atrophy (SMA), which constitutes a group of inherited diseases that cause progressive muscle degeneration leading to death. Consequently, therapeutic inventions have focused on increasing SMN protein levels. This invention discloses novel arylthiazolyl piperidines which are shown to be modulators of SMN production. This invention also discloses methods of treating SMA by administering SMN protein modulators.
Applications: Therapeutic developments for Spinal Muscular Atrophy
Advantages: Small molecule (series of analogs can be derived in search of improved performance)
Pre-clinical; no animal data
In vitro data available
Inventors: Juan J Marugan (NHGRI) Jingbo Xiao (NHGRI) Steven Titus (NHGRI) Noel T Southall (NHGRI) Wei Zheng (NHGRI) Elliott J Androphy (University of Massachusetts Medical School) Jonathan Cherry (University of Massachusetts Medical School)
Collaborative Research Opportunity: The NIH Chemical Genomics Center (NCGC), National Human Genome Research Institute, is seeking statements of capability or interest from parties interested in collaborative research to further develop, evaluate, or commercialize these SMN modulator compounds. Please contact Dr. Juan Marugan at email@example.com for more information.
Portfolios: Internal Medicine Internal Medicine - Therapeutics Central Nervous System Central Nervous System - Therapeutics
For Licensing Information Please Contact: Steven Standley Ph.D. NIH Office of Technology Transfer 6011 Executive Blvd. Suite 325, Rockville, MD 20852 United States Email: firstname.lastname@example.org Phone: 301-435-4074 Fax: 301-402-0220