Netherlands-based VectorY Therapeutics BV has raised €74.5 million to support development of new products for neurodegenerative diseases using a vector technology that enables the production of therapeutic antibodies in vivo. The financing includes new investors Angelini Ventures, Invest-NL of the Netherlands and the European Investment Bank. A group of returning investors including Forbion and Eli Lilly & Co also participated.
The financing comes as the company, founded in August 2020, prepares for an expansion of a Phase 1/2 study of its lead product which is being conducted in patients with amyotrophic lateral sclerosis (ALS), a disease with a global prevalence of about four to six cases per 100,000 people. The product, VTx-002, is a vectorised antibody where the capsids of AAV vectors have been engineered to incorporate the DNA of antibody fragments. When the vectors are delivered to the body, the antibodies are produced in vivo and secreted to perform their task.
VTx-002 targets a nuclear protein called transactive response DNA-binding protein 43 (TDP-43) which, in a disease setting, mislocates from the nucleus of a cell to the cytoplasm and forms aggregates that are toxic. The therapy is intended to target specific epitopes on the TDP-43 aggregated proteins in order to prevent proteinopathy in the cytoplasm, while protecting TDP-43 in the nucleus. The dysfunction of the TDP-43 protein is a feature of ALS.
In addition to ALS, VectorY has preclinical programmes targeting mutant huntingtin genes in Huntington’s disease, TDP-43 in frontotemporal dementia, and phosphorylated tau in Alzheimer’s disease.
“Neurodegenerative diseases represent some of the most complex challenges in medicine, and patients and families urgently need new treatment options,” Jim Scibetta, the company’s chief executive, said in a statement issued on 11 November.
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