Drug intelligence / Profile preview

scFv-MC1

Development stage
Preclinical
Lead developer
New York University
Modality
Gene Therapies, Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics
Administration
Intramuscular
01

Overview

scFv-MC1 is a single-chain variable fragment (scFv) derived from the MC1 monoclonal antibody, which specifically targets early pathological conformations of the tau protein. It is primarily developed as a gene therapy candidate for Alzheimer's disease, utilizing an adeno-associated virus (AAV) vector—specifically AAV1—to deliver the genetic sequence to skeletal muscle cells. Once expressed and secreted into the periphery, the scFv-MC1 fragment is intended to cross the blood-brain barrier and facilitate the clearance of misfolded, soluble, and oligomeric tau species. Preclinical studies in P301S transgenic mice have demonstrated that this approach can improve motor and behavioral functions and enhance brain glucose metabolism.

Other names
MC1 single-chain variable fragmentMC-1 single-chain variable fragmentMC 1 single-chain variable fragmentanti-tau scFv-MC1
02

Targets

MAPT (Microtubule-associated protein tau)

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