WVE-004
Early / Mid-stage
terminated
terminated
CT.gov grounded
A genetically anchored ALS/FTD program whose termination is still important context for anyone taking the pipeline seriously.
Program overview
- Mechanism
- C9orf72-targeted oligonucleotide
- Modality
- oligonucleotide
- Phase
- Early / Mid-stage
- Status
- terminated
- Recruitment
- terminated
- Confidence
- medium
- Watch priority
- 3
- Last verified
- 20 Apr 2026
Sponsor and trial identifiers
- Sponsor
- Wave Life Sciences
- Trial IDs
- NCT04931862, NCT05683860
ClinicalTrials.gov exact matches: NCT04931862, NCT05683860
Regions and recruitment
Australia
Belgium
Canada
Ireland
Netherlands
New Zealand
Sweden
United Kingdom
Indication tags
FTD
C9orf72
ALS/FTD
Milestones and catalysts
- Last milestone
- A meaningful terminated C9 ALS/FTD program that still deserves visibility because the genetics-first strategy remains central to the field.
- Next expected catalyst
- No active catalyst, but it remains a useful reference point for C9-directed clinical translation.
Related pages
Disease hub
Frontotemporal dementia hub
Disease-specific views with programs, mechanisms, and related context.
Related programs
Each card explains why it is here so you can read the overlap at a glance.
Early / Mid-stage
active
recruiting
Gene-therapy FTD program that helps make the monitor feel like a real neurodegeneration pipeline rather than an Alzheimer's side project.
- Mechanism
- GRN gene replacement
- Modality
- AAV gene therapy
- Next catalyst
- Early safety and biological activity signals in GRN-FTD.
- Last verified
- 20 Apr 2026
Mid-stage
terminated
terminated
FTD program targeting the progranulin pathway through sortilin inhibition.
- Mechanism
- sortilin inhibition / progranulin elevation
- Modality
- monoclonal antibody
- Next catalyst
- Mid-stage readouts and biomarker alignment.
- Last verified
- 20 Apr 2026
Late-stage / Terminated
completed
completed
A major FTD progranulin program that now functions as an important field-level setback reference rather than an active growth story.
- Mechanism
- progranulin elevation
- Modality
- monoclonal antibody
- Next catalyst
- No near-term active catalyst, but it matters as context for the FTD field.
- Last verified
- 20 Apr 2026
Early / Mid-stage
completed
completed
An allele-selective Huntington's program that matters for the field's longer-run precision ambitions.
- Mechanism
- allele-selective HTT silencing
- Modality
- oligonucleotide
- Next catalyst
- How much of the allele-selective approach survives into future development strategy.
- Last verified
- 20 Apr 2026
Recent program changes
WVE-004
Added to the monitor: WVE-004 · Frontotemporal dementia · Early / Mid-stage.