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

~ 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 ~

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

About ALS

Amyotrophic lateral sclerosis (ALS), a motor neuron disease (MND) also known as Lou Gehrig disease, is a rare neurodegenerative disease characterized by the rapid and progressive loss of motor neurons, leading to paralysis, and death due to the inability to breathe. The vast majority of cases of ALS (90-95%) are categorized as sporadic, meaning they occur without a known cause, while 5-10% are familial, linked to a genetic mutation passed down through families. ALS is a fatal disease, with most individuals dying from complications such as respiratory failure within 3 to 5 years of diagnosis, though some may survive longer.

About VectorY

VectorY is on a mission to provide people with neurodegenerative diseases a longer, better life by creating transformative vectorized antibody treatments. Our platform combines the promise of precise therapeutic antibodies with one-time AAV-based delivery to the CNS. Unique in-house expertise in antibodies, AAV vectors, protein degradation, manufacturing and neuroscience drives the rapid development of much needed disease-modifying therapies for neurodegenerative diseases such as ALS. For more information, see www.vectorytx.com.

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“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.