Pipelines

Milestone

At the forefront of therapeutic research, Rare Power is pioneering novel pharmacological treatments specifically targeting Huntington's Disease, Spinocerebellar Ataxias and Amyotrophic Lateral Sclerosis. Our drug development pipeline is progressing steadily towards Investigational New Drug (IND)-enabling studies and clinical trials.

RareCure Milestone Plan

RareCure Pipelines

Candidate

Indication

Target

*Discovery & Validation

IND-enabling

Phase I/IIA

RP001v63

Peptide biologic
HD/SCAs
RNA
2026
2024

RP002v9

Peptide biologic
C9ALS/FTD
RNA
2027
2024

RP004001

Repurposed small molecule
HD/SCAs/AD
Protein
2028
2024
*Discovery & Validation
  • Target validation, lead discovery and optimization
  • Lab animal and/or patient-derived iPSC efficacy tests
Current progress
Expected timeline
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