Drug Profile

PM 359

Alternative Names: PM-359

Latest Information Update: 30 May 2025

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At a glance

Originator Prime Medicine
Class Gene therapies; Immunotherapies
Mechanism of Action Gene transference; Neutrophil cytosolic factor 1 replacements

Orphan Drug Status

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

Yes - Chronic granulomatous disease
New Molecular Entity No

Highest Development Phases

Phase I/II Chronic granulomatous disease

Most Recent Events

19 May 2025 Prime Medicine announces intention to submit IND/CTA to regulatory body for Alpha-1-Antitrypsin-Deficiency in mid of 2026
19 May 2025 Prime Medicine announces intention to submit IND/CTA to regulatory body for Wilson’s Disease in 1H 2026
19 May 2025 Efficacy and adverse event data from a phase I/II trials in Chronic granulomatous disease released by

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