€74.5M boost for VectorY’s gene therapy against ALS

Amsterdam-based VectorY Therapeutics has raised €74.5 million to push its experimental treatment for amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease, through a key clinical trial.

At the centre of the raise is VTx-002, a potential first-of-its-kind therapy for ALS. The disease affects the brain cells and the spinal cord and slowly destroys the nerve cells that control the muscles, robbing people of the ability to move, speak and eventually breathe.

Instead of giving patients antibody drugs through repeated drips, VectorY uses a harmless virus to deliver the genetic instructions for an antibody straight to nerve cells. The cells then make the antibody themselves, potentially for years after a single treatment.

The antibody targets TDP-43, a protein that forms toxic clumps in the nerve cells of up to 97% of ALS patients. It is designed to latch on to the harmful clumps while leaving the healthy protein alone.

Expanding the trial

VTx-002 is being tested in PIONEER-ALS, an international early-stage study looking at safety and the first signs that the treatment works. In September, VectorY tripled the trial from 12 to 36 participants. The new cash is meant to carry the study through to its final results.

The money will also fund VTx-003, a treatment for Huntington's disease, another neurodegenerative disease, and help adapt the technology for other brain disorders. VectorY has further programmes in frontotemporal dementia and Alzheimer's disease.

Better ALS treatment

The round brings in three new investors: Italy's Angelini Ventures, the European Investment Bank (EIB) and Dutch state investor Invest-NL, which is contributing €7 million. Existing shareholders, including Forbion, EQT Life Sciences, Insight Partners, Eli Lilly and the ALS Investment Fund, also put in fresh money.

"People living with ALS need better options," said Regina Hodits, Managing Director of Angelini Ventures. She pointed to the company's clear biological target, its use of measurable markers in the trial and its in-house manufacturing. Hodits will join VectorY's board as an observer.

VectorY builds its own production system for the viruses it uses, which it says can be scaled up at lower cost than the industry standard. VTx-002 has not yet been approved by any regulator.