Page 17 - 2021 Spring CMTA Report - Special Research Edition

D E M Y E L I N AT I N G T Y P E S

DRUG DEVELOPMENT STAGE
STAR ALLIANCE PARTNERS THERAPY TYPE
Discovery Research Tools Preclinical Phase1 Phase2 Phase3
Pharnext Small Molecule P3
Acceleron* Biological P2
InFlectis BioScience Small Molecule P1/P2
Cyprus + University of Wisconsin + University of Texas, SW Gene Therapy Preclinical
Ionis Gene Therapy Preclinical
National Institutes of Health* Assays/Small Molecule Preclinical
Regenacy Pharmaceuticals* Small Molecule Preclinical Developing and Characterizing
Sanofi* Small Molecule/Biomarkers Preclinical
STAR Biotech Partner A* Small Molecule Preclinical New Mouse Models of CMT1X
1A STAR Biotech Partner B* Small Molecule Preclinical Mouse and rat models are invaluable in disease research
STAR Biotech Partner C Gene Therapy Preclinical because models can be developed to mimic the exact
STAR Partner D Biological Preclinical same genetic trends seen in patients. They can also
University of Wisconsin Small Molecule Preclinical be used for gene therapy testing, axonal degeneration
University of Wisconsin Target Validation Preclinical assessment and testing various therapeutics.
Jackson Laboratory SARM1/Target Validation Preclinical Over the years, the CMTA has funded the development
Jackson Laboratory HDAC6/Target Validation Preclinical and usage of multiple models for CMT1A and CMT1B.
University of Rochester, NY Biomarkers (Wearables) Preclinical Continued development of new genetically engineered
mouse models for CMT1X is ongoing in many labs and
University at Bu‰alo, NY Small Molecule Preclinical is critical because CMT1X is more heterogeneous (> 400
University of Wisconsin + Memorial Sloan Kettering Target Discovery Discovery variants identified) than other forms of CMT. It is therefore
essential that multiple mouse models are developed to
DRUG DEVELOPMENT STAGE model the genetic variability found in CMT1X patients.
STAR ALLIANCE PARTNERS THERAPY TYPE
Discovery Research Tools Preclinical Phase1 Phase2 Phase3 Additionally, further work is being done to characterize
Acceleron* Biological P2 new CMT1X mouse models as they are developed.
This means that each model (representing a specific
InFlectis BioScience Small Molecule P1/P2 mutation) is analyzed to characterize every aspect of the
National Institutes of Health* Assays/Small Molecule Preclinical progression and severity of CMT1X. Some of the
San Ra‰aele Scientific Institute Small Molecule Preclinical characterizations include age of onset, quantifying
University at Bu‰alo, NY Small Molecule Preclinical changes in axonal loss, nerve conduction, demyelination
1B Universities of Iowa and Wisconsin Biomarkers Preclinical patterns over time and performance of various motor skills.
University of Wisconsin Target Validation Preclinical Both development of new models and characterization of
each model are critical to support the development of new
University of Iowa Target Validation Preclinical drugs to treat CMT and, ultimately, to find a cure.
University at Bu‰alo, NY Animal Model/Target Validation Laboratory
University at Bu‰alo, NY Target Discovery Discovery

DRUG DEVELOPMENT STAGE
STAR ALLIANCE PARTNERS THERAPY TYPE
Discovery Research Tools Preclinical Phase1 Phase2 Phase3 STAR
Acceleron* Biological P2
Cyprus Institute Gene Therapy Preclinical
University of Pennsylvania Target Validation Preclinical ALLIANCE
1X Jackson Laboratory SARM1/Target Validation Preclinical
Universities of Iowa & Wisconsin Biomarkers Preclinical
University of Illinois (Chicago) Animal Model Laboratory
Jackson Laboratory Animal Model Laboratory PARTNERS

DRUG DEVELOPMENT STAGE
STAR ALLIANCE PARTNERS THERAPY TYPE
Discovery Research Tools Preclinical Phase1 Phase2 Phase3
4A – University of Texas South Western Gene Therapy Preclinical 32+ Research Partners
4B – San Ra‰aele Scientific Institute Small Molecule Preclinical 50+ Research Projects
4 4C – Cyprus Institute Gene Therapy Preclinical
4J – Neurogene Gene Therapy Preclinical $17M+ in Research Funding
4A – Envigo Preclinical Testing Partner Research Tools/Preclinical * Project concluded
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