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Neurotrophic Agents for Treating Parkinson's Disaese

Award Information
Agency: Department of Health and Human Services
Branch: National Institutes of Health
Contract: 1R41NS089140-01A1
Agency Tracking Number: R41NS089140
Amount: $233,804.00
Phase: Phase I
Program: STTR
Solicitation Topic Code: 102
Solicitation Number: PA14-072
Timeline
Solicitation Year: 2015
Award Year: 2015
Award Start Date (Proposal Award Date): 2015-09-15
Award End Date (Contract End Date): 2016-08-31
Small Business Information
126 FILLY DRIVE
North Wales, PA 19454-4248
United States
DUNS: 961713588
HUBZone Owned: No
Woman Owned: No
Socially and Economically Disadvantaged: No
Principal Investigator
 SHAWN DEFREES
 (215) 315-9003
 sdefrees@neose.com
Business Contact
 SHAWN DEFREES
Phone: (267) 640-2583
Email: sdefrees@senebbio.com
Research Institution
 RBHS-NEW JERSEY MEDICAL SCHOOL
 
185 S ORANGE AVE
NEWARK, NJ 07103-2757
United States

 Nonprofit College or University
Abstract

DESCRIPTION provided by applicant Parkinsonandapos s disease PD is a neurodegenerative disorder characterized by motor function and eventually cognitive decline GM depletion is validated as a therapeutic target for Parkinsonandapos s disease PD in clinical studies in which GM replacement stabilized motor function loss and may be disease modifying However GM has poor pharmacologic properties that include poor potency mg SC bid man and aqueous solubility mg in mL no oral bioavailability and limited blood brain barrier penetration crosses the BBB that makes continuous dosing by injection painful patient compliance difficult and chronic treatment undesirable for this devastating disease Our goal in this program
is to assess GM analogs for their pharmacodynamic properties in a PD mouse model validate their potential efficacy for PD using a genetic PD mouse model and ultimately proceed to nonclinical ADMET studies and an IND

PUBLIC HEALTH RELEVANCE GM depletion is validated as a therapeutic target for Parkinsonandapos s disease PD in preclinical and clinical studies in which GM replacement was capable of modifying the motor losses resulting from the disease state However GM has poor pharmacologic properties that make continuous dosing by injection painful patient compliance difficult and chronic treatment undesirable for this devastating disease We have developed novel potent orally bioavailable analogs of GM which can solve this road block and become the first disease modifying treatment for PD

* Information listed above is at the time of submission. *

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