VectorY Announces Preclinical Data on TDP-43 and Oxidized Phospholipids at American Society of Gene and Cell Therapy (ASGCT) Annual Meeting

AMSTERDAM--(BUSINESS WIRE)-- VectorY Therapeutics, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of neurodegenerative and muscular disorders through vectorized antibodies, today announced further detail on the two presentations to be delivered at the American Society of Gene and Cell Therapy (ASGCT) Congress 2022 Annual Meeting being held May 16-19 in Washington, D.C.

The presented data establish the therapeutic potential and versatility of VectorY Therapeutics’ VecTab platform technology, enabling development of secreted or intracellular antibody fragments that are AAV5-vectorized and delivered to neurons and/or astrocytes, in vitro and in vivo. VecTabs were shown to efficiently target misfolded proteins (TDP-43) and toxic lipids (oxPL) that are involved in the pathogenesis of neurodegenerative diseases.

Details of the two posters are as follows:

• VTx-002: VecTabs reduce TDP-43 Pathological Aggregates for Treating Amyotrophic Lateral Sclerosis (Pos, W et al)

  TAR-DNA binding protein-43 (TDP-43) is necessary for correct processing and transport of multiple mRNA’s that are essential for neuronal survival, but cytoplasmic mis-localized, misfolded or aggregated TDP-43 has been implicated in the pathogenesis of >97% of ALS patients.

  VectorY has confirmed TDP-43 pathology in iPS-derived ALS motor neurons and developed a library of single-chain variable fragments (scFv), named VecTabs that exclusively bind to the misfolded toxic TDP-43 species, leaving the native TDP-43 functional. TDP-43-targeting intracellular VecTabs effectively cleared (large) TDP-43 aggregates from U2OS cells, and iPS-derived ALS motor neurons were efficiently transduced to express TDP-43 VecTabs. In mice and pigs biodistribution and expression of AAV5 VecTabs was demonstrated in the spinal cord and motor cortex.

  These data are the basis of further development of VTx-002 in ALS.

The poster is available: 17:30-18:30 EDT Tuesday 17 May 2022 – Hall D, Tu-154

• VTx-001: VecTabs target Oxidized Phosphatidylcholine for Treating Amyotrophic Lateral Sclerosis (Pasteuning, S et al)

  Oxidized phospholipids (oxPL) are potent neurotoxic lipid species that are involved in the pathogenesis of multiple sclerosis, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). Recent in vivo and ex vivo data indicate a pivotal role for oxPL in motor neuron death in ALS.

  VectoryY has developed AAV5-expressed secreted VecTabs that specifically bind and neutralize oxPL. These VecTabs protected iPS-derived motor neurons from oxPL-mediated cell death. The biodistribution and expression of OxPL VecTabs were investigated in mice and pigs, demonstrating transduction of the target spinal cord and cortical motor neurons, and expression of the therapeutic scFv.

  These data are the basis for further development of VTx-001 in ALS.

The poster is available 17:30-18:30 EDT Wednesday 18 May 2022 - Hall D Poster section W-169

To meet the VectorY team at ASGCT, please contact info@vectorytx.com.

Pavlina Konstantinova, Chief Scientific Officer of VectorY said: “The results reported in these two posters underline the potential impact of our VecTab technology on neuronal pathology in ALS. We also see that the ability of our VecTabs to specifically target misfolded proteins demonstrates their potential application across a wider range of pathologies in the CNS and other disease areas”.

ENDS

Notes to Editors

About VectorY

VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for neurodegenerative and -muscular diseases with high unmet medical need. Founded in October 2020 and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform, antibody-based targeted protein degradation technologies and proprietary manufacturing technology. While focusing initially on neurodegenerative and -muscular diseases, VectorY’s synergistic technologies may be applied across a wide range of indications. VectorY’s manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands.

For more information, see www.vectorytx.com.

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