VectorY Therapeutics announced $83.5 million in financing on November 11 to take its enlarged ALS trial through its final readout. The Amsterdam biotech tripled the study’s planned size in September, before receiving patient efficacy data.
The company is testing VTx-002, a gene therapy designed to give cells in the central nervous system instructions to make antibodies against abnormal TDP-43, a protein implicated in ALS. VectorY’s approach aims to sustain antibody production after a single treatment; the drug remains investigational.
PIONEER-ALS now plans to enroll 36 adults, up from 12, at two dose levels in an open-label Phase 1/2 study. VectorY said faster enrollment and site openings enabled the expansion. Researchers are assessing safety alongside exploratory signs of benefit, including muscle strength, breathing and survival.
New investors Angelini Ventures, Invest-NL and the European Investment Bank joined returning backers including Forbion, EQT Life Sciences and Eli Lilly. The EIB signed a convertible loan agreement representing about two-fifths of the package, supported by the European Commission’s InvestEU program.
VectorY closed a $138 million Series A in November 2023, when VTx-002 was still preclinical. EQT Life Sciences and Forbion co-led that round. Dosing in the ALS study began in February 2026.
The new financing also supports VTx-003, VectorY’s program targeting the mutant huntingtin protein in Huntington’s disease.