VectorY Therapeutics to Present Preclinical Data on Novel ALS Therapeutic Strategy at Target ALS Conference in Boston

Yahoo

07-05-2025

~ New data show AAV-delivered antibody fragments targeting oxidized phosphatidylcholines (PC-OxPL), a species of oxidized phospholipids, thereby neutralizing TDP-43 pathology and motor neuron toxicity in ALS models ~
~ Underscores potential of VTx-001 to act on upstream drivers of ALS ~
AMSTERDAM & BOSTON, May 07, 2025--(BUSINESS WIRE)--VectorY Therapeutics, a biotech company developing disease-modifying vectorized antibody therapies for the treatment of neurodegenerative diseases, today announced the presentation of new preclinical data on one of its two therapeutic vectorized antibodies for ALS, VTx-001, at the Target ALS Annual Meeting in Boston, Massachusetts taking place between May 6-8, 2025.
The data underscore the potential of VTx-001 to neutralize upstream pathological drivers in amyotrophic lateral sclerosis (ALS), including oxidized phosphatidylcholines (PC-OxPL), which have been implicated in triggering ALS hallmark TDP-43 proteinopathy and motor neuron degeneration. This research lays the foundation for a potential new therapeutic modality targeting oxidative phospholipid toxicity in ALS and possibly other neurodegenerative indications.
VectorY's research team identified a distinct PC-OxPL profile in the brains of sporadic ALS (sALS) patients, with apolipoprotein E (APOE) particles emerging as the primary carriers of PC-OxPLs in the central nervous system. PC-OxPL exposure of iPSC-derived motor neurons induced an ALS-like transcriptome, significant TDP-43 pathology, and motor neuron death.
In VectorY's preclinical data presented at the conference, VTx-001, an AAV-delivered, single-chain antibody fragment was shown to:
Neutralize PC-OxPL-induced TDP-43 pathology
Normalize disease-associated transcriptomic profiles
Prevent motor neuron death and functional decline in vivo
Achieve robust biodistribution and expression at therapeutic levels in a minipig model following intrathecal administration
"These findings support our hypothesis that PC-OxPL molecules are a key mediator of ALS pathology, acting upstream of TDP-43 proteinopathy and motor neuron toxicity," said Sander van Deventer, M.D., Ph.D., president of research and development at VectorY Therapeutics. "VTx-001 offers a novel and complementary mechanism of action - to our lead candidate, VTx-002, which directly targets TDP-43 and for which we anticipate filing an IND before the end of this year."
These data featured at the Target ALS Annual Meeting were recently published in the on-line publication bioRxiv. The manuscript, AAV-Delivered Anti-PC-OxPL Antibody Fragments: A Novel Therapeutic Approach to Target ALS, is available through the following link: https://doi.org/10.1101/2025.01.22.634350