VectorY to Present Poster on Huntington’s Preclinical Data at the Hereditary Disease Foundation, August 24-27 in Cambridge, MA
- Category: DNA RNA and Cells
- Published on Tuesday, 23 August 2022 14:19
- Hits: 755
AMSTERDAM, The Netherlands I August 23, 2022 I VectorY, a biotech company focusing on the development of innovative gene therapy approaches for the treatment of neurodegenerative and muscular disorders through vectorized antibodies, announces today that it will present a poster at the Hereditary Disease Foundation 2022 Meeting in Cambridge, Massachusetts, on August 24-27, 2022.
The poster shows that VectorY’s AAV vector, dose-dependently transduced AAV iPS derived human neurons and astrocytes ex vivo, and AAV vector-delivered transgenes were expressed long-term in the brains and spinal cords of mice and pigs. Several VecTab candidates were identified that effectively removed huntingtin aggregates from U2OS cells that expressed a CAG-repeat-expanded pathological form of huntingtin.
Huntington’s Disease is caused by aggregates of polyglutamine-rich HTT protein, which result from CAG repeat expansions of the Huntingtin gene. VecTabs, which are secreted- or intracellular-antibody fragments that are AAV-vectorized and delivered to neurons and/or astrocytes, have previously been shown to efficiently target such misfolded proteins (TDP-43) and toxic lipids (oxPL). The poster data shows that the platform is also able to target polyglutamine-rich proteins like HTT, demonstrating the clearance of toxic HTT aggregates.
The details of the poster are as follows:
Title: VecTabs®: Targeting misfolded and aggregated proteins with vectorized antibodies for the treatment of ALS and HD
Time and date: 25 August, 6:30 p.m. - 10:00 p.m. EDT
Location: Royal Sonesta Hotel Boston, 40 Edwin Land Boulevard, Cambridge, MA 02142
Pavlina Konstantinova, Chief Scientific Officer of VectorY, said: “The results that will be reported at the Hereditary Disease Foundation are encouraging signals that our VecTab technology has the potential to effectively target Huntington’s Disease. Our technology enables the specific targeting of misfolded and aggregated proteins, which have been shown to cause neurodegenerative diseases, whilst maintaining the function of the correctly folded native forms of those proteins.”
The new data demonstrates the effective removal of huntingtin (HTT) aggregates in a preclinical model. VectorY’s novel platform has demonstrated accelerated removal of toxic HTT proteins intracellularly, where they cause damage to the deep brain motor neurons. The research supports and highlights the therapeutic potential and versatility of VectorY’s VecTab platform technology, which was previously reported in preclinical models of amyotrophic lateral sclerosis.
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