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MY001

Development stage: Preclinical
Lead developer: Mabylon
Modality: scFv Fragments → Antibody Fragments → Engineered Antibody Formats → Antibody-Based Therapeutics

Overview

**MY001** is a preclinical Mabylon intrabody program for amyotrophic lateral sclerosis and frontotemporal dementia. It comprises human-derived intracellular antibodies directed against pathological TAR DNA-binding protein 43, with the intended disease-modifying effect of preventing toxic TDP-43 aggregation and restoring or preserving normal TDP-43 biology in TDP-43 proteinopathies. The delivery construct, route of administration, dosage form, and precise antibody sequence have not been publicly disclosed. ([mabylon.com](https://www.mabylon.com/))

Targets

TARDBP (TAR DNA-binding protein 43)

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