Drug Profile

PYC 003

Alternative Names: PYC-003

Latest Information Update: 03 Apr 2025

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At a glance

Originator PYC Therapeutics
Class Antisense oligonucleotides; Drug conjugates; Morpholines; Peptide drug conjugates
Mechanism of Action RNA interference; SHANK3 protein replacements

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

Preclinical Polycystic kidney disease; Telomeric 22q13 Monosomy Syndrome

Most Recent Events

28 Feb 2025 PYC Therapeutics files an IND application with the US FDA in USA for Polycystic kidney disease, prior to February 2025
28 Feb 2025 US FDA approves IND application for PYC 003 in Polycystic kidney disease
03 Dec 2024 PYC Therapeutics plans a phase I trial for Polycystic kidney disease (IV, Infusion) in April 2025 (NCT06714006)

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