B/BE/25/BVW9 - Information sheet for the public (prepared by the notifier)Public information according to the provisions of Directive 2001/18/EC
Long title
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002
Date receipt dossier
EU record number
B/BE/25/BVW9
EudraCT number
2025-522697-37-00
Company / Sponsor
VectorY Therapeutics B.V.
Phase
I/II
Treated organism
Humans
Indication category
Neurodegenerative disease
Disease
Amyotrophic Lateral Sclerosis
Therapeutic approach
Gene therapy
Genetic modification
Non-replicating recombinant AAV vector with a AAV5.2 capsid and carrying the transgene corresponding to the single-chain variable fragment that binds to human TDP-43
Method of transfer of nucleic acid of interest
Non-replicating recombinant vector derived from adeno-associated virus serotype 5
Administered biological material
Genetically modified virus
Route of administration
Intracisternal
Locations in Belgium
UZ Leuven
Nr of subjects
up to 12 patients overall and 3 patients in Belgium
Foreseen duration
From December 2025 till October 2031
Type of procedure
Contained use and Deliberate release
Current status
Authorized