Projects Results and Documents

PaVe-GT is committed to sharing lessons learned on our journey. As this project progresses over several years, this page will be populated with project results and documents, including communications with the U.S. Food and Drug Administration, all four Investigational New Drug packages with NIH-submitted data, product profiles, clinical study documents, and more.

  • 1
    Proof of Concept Design, create, and test the therapeutic in appropriate cell lines and mouse models.
  • 2
    Pre-clinical Development Produce cGMP grade materials. Conduct “IND-enabling” studies such as toxicology studies.
  • 3
    IND Filed Submit an Investigational New Drug (IND) application to FDA CBER, seeking permission to begin studies in humans.
  • 4
    Phase 1/2 Initiate and conduct human trials.
Acidemias Liver Targeting
Congenital Myasthenic Diseases Neuromuscular Junction (NMJ) Targeting
PCCA Deficiency (Early Proof of Concept Stage)
cblB (MMAB) Deficiency (Early Proof of Concept Stage)
COLQ Deficiency (Early Proof of Concept Stage)
DOK7 Deficiency (Early Proof of Concept Stage)
  • 1
    Proof of Concept
  • 2
    Pre-clinical Development
  • 3
    IND Filed
  • 4
    Phase 1/2
Acidemias Liver Targeting
Congenital (NMJ) Targeting
PCCA
cblB (MMAB)
COLQ
DOK7
1
2
3
4
PCCA
cblB (MMAB)
COLQ
DOK7
Acidemias Liver Targeting
Congenital (NMJ) Targeting
1
Proof of Concept
Design, create, and test the therapeutic in appropriate cell lines and mouse models.
2
Pre-clinical Development
Produce cGMP grade materials. Conduct “IND-enabling” studies such as toxicology studies.
3
IND Filed
Submit an Investigational New Drug (IND) application to FDA CBER, seeking permission to begin studies in humans.
4
Phase 1/2
Initiate and conduct human trials.

 

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