VectorY secures €74.5M to advance ALS gene therapy trial
Amsterdam-based VectorY Therapeutics has secured €74.5 million ($83.5 million) in new financing to advance its lead gene therapy candidate for amyotrophic lateral sclerosis (ALS) and expand its pipeline of treatments for neurodegenerative diseases. The funding includes a €30 million convertible loan from the European Investment Bank (EIB).
Why it matters: The financing will support VTx-002 through the final readout of its Phase 1/2 PIONEER-ALS trial, which began dosing patients in February 2026. The program is testing whether gene therapy can enable sustained production of therapeutic antibodies in the central nervous system following a single administration.
Zoom in: In September, VectorY tripled the planned enrollment in PIONEER-ALS from 12 to 36 patients, citing rapid recruitment and activation of clinical sites. The open-label study is evaluating two dose levels of VTx-002 across the U.S. and Europe. Its primary focus is safety and tolerability, but researchers are also tracking biomarkers, including neurofilament light chain (NfL), and clinical measures of disease progression, such as muscle strength and respiratory function.
How it works: VTx-002 uses an engineered adeno-associated virus (AAV) to deliver genetic instructions that allow cells in the central nervous system to produce therapeutic antibodies.
These antibodies are designed to target abnormal TDP-43, a protein that accumulates in motor neurons in up to 97% of ALS patients. The abnormal buildup, combined with the loss of TDP-43’s normal function inside the cell nucleus, is thought to contribute to neuronal damage. VectorY hopes that clearing these aggregates can preserve neuronal function and slow disease progression.
The financing: The latest package builds on the €129 million Series A raised in November 2023, bringing VectorY’s reported financing to €234.5 million since its €31 million seed round in 2021.
New backers include Angelini Ventures and Invest-NL, alongside the EIB, whose €30 million convertible loan is supported by the European Commission’s InvestEU program. Returning investors include Forbion, EQT Life Sciences, Eli Lilly, MRL Ventures, Insight Partners and BioGeneration Ventures.
Beyond ALS: The funding will also advance VTx-003, a preclinical program targeting mutant huntingtin protein in Huntington’s disease. Earlier-stage programs include VTx-004 for frontotemporal dementia and VTx-005 for Alzheimer’s disease.
What to watch: VectorY has not disclosed when it expects the final PIONEER-ALS readout. However, the September trial expansion was decided before any patient efficacy readout. Whether VTx-002 can safely reduce TDP-43 pathology and produce meaningful clinical benefits remains unproven.
