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Corrector Program

Fabry disease

Hit to LeadActive

Key Facts

Indication
Fabry disease
Phase
Hit to Lead
Status
Active
Company

About Octant Biotech

Octant Biotech is a private, pre-revenue drug discovery company pioneering a platform-based approach to engineer small molecule drugs against complex cellular mechanisms. Its core technology, the Octant Navigator, integrates Generative Biology (high-throughput cellular sensor engineering), Generative Chemistry (nano-scale automated synthesis), and Generative AI in a rapid design-build-test-learn cycle. The company has built a diversified preclinical pipeline targeting genetic, oncology, metabolism, and immunology diseases, with its most advanced candidate, OCT-980 for retinitis pigmentosa, having initiated a Phase 1a study in healthy volunteers in Q1 2026.

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Other Fabry disease Drugs

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GMAC for Fabry DiseaseCellGenTechPreclinical
JR-171 (lepunafusp alfa)JCR PharmaceuticalsPhase 3
Undisclosed Gene TherapyuniQurePreclinical
LucerastatIdorsiaPhase 3
PRX–115 (pegunigalsidase alfa)Protalix BioTherapeuticsSubmitted
Isaralgagene civaparvovec (ST-920)Sangamo TherapeuticsPhase 1/2