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Drug Profile

Research programme: RNA-targeting antisense oligonucleotide therapeutics - CAMP4 Therapeutics/BioMarin Pharmaceutical

Alternative Names: RNA-Actuators™

Latest Information Update: 11 Feb 2025

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At a glance

Originator CAMP4 Therapeutics
Developer BioMarin Pharmaceutical; CAMP4 Therapeutics
Class Antisense oligonucleotides
Mechanism of Action Gene silencing

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Research Unspecified

Most Recent Events

10 Oct 2024 Early research in Unspecified in USA (Parenteral)

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