Drug intelligence / Profile preview

scFv-PHF1

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

Overview

scFv-PHF1 is an engineered single-chain variable fragment (scFv) derived from the well-characterized monoclonal antibody PHF1. It specifically targets tau protein phosphorylated at serine residues 396 and 404 (Ser396/404), an epitope associated with late-stage pathological tau modifications and paired helical filaments (PHFs) in Alzheimer's disease and other tauopathies. The therapeutic strategy typically involves the use of a viral vector, such as adeno-associated virus 1 (AAV1), to deliver the genetic sequence encoding the scFv to peripheral tissues like skeletal muscle. Once expressed, the scFv-PHF1 fragment is intended to bind to and facilitate the clearance of cerebral tau species. While preclinical studies in transgenic mouse models (e.g., P301S mice) have demonstrated its ability to reduce brain tau levels, its efficacy in improving motor and behavioral functions has been noted as less robust compared to fragments targeting earlier tau modifications, such as scFv-MC1.

Other names
PHF1 single-chain variable fragmentPHF-1 single-chain variable fragmentPHF 1 single-chain variable fragmentanti-tau PHF1 scFvPHF1 scFvPHF-1 scFvPHF 1 scFv
02

Targets

p-tau396/404 (Microtubule-associated protein tau (phosphorylated at Ser396/Ser404))

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