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Modulators of Survival Motor Neuron Production

Posted Dec 08 2010 7:00pm

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.

Therapeutic developments for Spinal Muscular Atrophy

Small molecule (series of analogs can be derived in search of improved performance)

Development Status:
  • Pre-clinical; no animal data
  • In vitro data available

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)

Patent Status:
HHS, Reference No. E-109-2010/0
US, Application No. 61/323,963 filed 14 Apr 2010

Licensing Status:
Available for licensing

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 for more information.

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
Phone: 301-435-4074
Fax: 301-402-0220

Ref No: 2204

Updated: 12/2010

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