WVE-004

WVE-004

Wave Life Sciences · Frontotemporal dementia
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

Active in 8 regions
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.
Research Research agenda Research notes and background around Frontotemporal dementia.
Key Targets Key targets The mechanisms and intervention levers behind these programs.
Discover Discover Explainers and visual introductions to the science behind neurodegeneration.
Join Get involved Contribute research, writing, design, engineering, or review.

Related programs

Each card explains why it is here so you can read the overlap at a glance.

Priority 1
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
Priority 2
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
Priority 3
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

WVE-003

Wave Life Sciences · Huntington's disease
Priority 3
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

Source links

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