Actio Biosciences

Private

#Rare Disease #Epilepsy #Charcot-Marie-Tooth Disease #Genetics

Genetics-driven precision medicine company developing small molecule therapeutics for rare and common diseases. Pipeline includes KCNT1 inhibitor for epilepsy and TRPV4 inhibitor for Charcot-Marie-Tooth disease. Acquired by Jazz Pharmaceuticals for $820M.

Company Info

Sector:Rare Disease / Precision Medicine

Headquarters:San Diego, California, USA

Founded:2023

Website:actiobiosciences.com/

Upcoming Milestones & Key Dates

2027

Regulatory

IND filing for KCNT1 epilepsy expected

Clinical Development Pipeline

ACT-001

Target: KCNT1 potassium channel inhibitor

KCNT1-related epilepsy

Preclinical

Phase 1

Phase 2

Phase 3

Approved

ACT-002

Target: TRPV4 inhibitor

Charcot-Marie-Tooth disease type 2C

Preclinical

Phase 1

Phase 2

Phase 3

Approved

Recent News

No recent news updates available for this company.

Leadership Team

David Goldstein

CEO & Co-Founder

John McHutchison

Co-Founder, Board Member

Careers & Job Openings

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