ALS pipeline
Track ALS programs across genetic targeting, stress-response biology, cell therapy, and platform-trial bets.
Top programs in this hub
Starts with the highest-priority programs in the current dataset, then widens into the full bench below.
Gene-targeted ALS therapy with major strategic importance for precision neurodegeneration treatment.
- Mechanism
- SOD1 antisense reduction
- Modality
- ASO
- Next catalyst
- Longer-term follow-up and real-world use data.
- Last verified
- 20 Apr 2026
A UK-centered ALS platform trial that matters as infrastructure as much as any single drug inside it.
- Mechanism
- multi-arm adaptive platform trial
- Modality
- platform / multi-intervention trial
- Next catalyst
- Ongoing arm-level readouts and additions within the adaptive platform framework.
- Last verified
- 20 Apr 2026
Late-stage ALS program that stays relevant because the field still lacks enough credible shots on goal.
- Mechanism
- tyrosine kinase / mast cell modulation
- Modality
- small molecule
- Next catalyst
- Phase 3 progression and whether the long-running controversy resolves into a usable signal.
- Last verified
- 20 Apr 2026
What defines this bench
Quick pattern read on the mechanisms and modalities currently showing up most often.
A disease-specific view of the main programs, the common mechanisms, and the related program pages.
All tracked ALS programs
Every program in the current watchlist for this disease, in priority order.
Gene-targeted ALS therapy with major strategic importance for precision neurodegeneration treatment.
- Mechanism
- SOD1 antisense reduction
- Modality
- ASO
- Next catalyst
- Longer-term follow-up and real-world use data.
- Last verified
- 20 Apr 2026
A UK-centered ALS platform trial that matters as infrastructure as much as any single drug inside it.
- Mechanism
- multi-arm adaptive platform trial
- Modality
- platform / multi-intervention trial
- Next catalyst
- Ongoing arm-level readouts and additions within the adaptive platform framework.
- Last verified
- 20 Apr 2026
Late-stage ALS program that stays relevant because the field still lacks enough credible shots on goal.
- Mechanism
- tyrosine kinase / mast cell modulation
- Modality
- small molecule
- Next catalyst
- Phase 3 progression and whether the long-running controversy resolves into a usable signal.
- Last verified
- 20 Apr 2026
A controversial but undeniably field-relevant ALS cell-therapy program that should be represented in a serious pipeline monitor.
- Mechanism
- autologous MSC-NTF cell therapy
- Modality
- cell therapy
- Next catalyst
- Whether the renewed Phase 3 path can produce a cleaner efficacy narrative than prior attempts.
- Last verified
- 20 Apr 2026
ALS program aimed at stress-response biology rather than gene-specific targeting.
- Mechanism
- integrated stress response modulation
- Modality
- small molecule
- Next catalyst
- Readouts on clinical progression and tolerability.
- Last verified
- 20 Apr 2026
Important failed or withdrawn ALS program that still matters as pipeline context.
- Mechanism
- cell stress and mitochondrial support combination
- Modality
- small molecule combination
- Next catalyst
- No active development catalyst currently tracked here.
- Last verified
- 20 Apr 2026
Unusual bioenergetic approach in ALS that broadens the monitor beyond antisense and inflammatory narratives.
- Mechanism
- nanocatalytic bioenergetic support
- Modality
- nanomedicine / suspension
- Next catalyst
- Whether follow-up work supports a larger efficacy narrative in ALS.
- Last verified
- 20 Apr 2026
Related pages
Research, key targets, Discover, and ways to contribute.
Other disease hubs
Other disease areas in the pipeline.
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Disease hub Huntington's pipelineTrack Huntington's programs across gene therapy, antisense, allele-selective, and small-molecule huntingtin-lowering approaches.