Drug Profile

PYC 001

Alternative Names: PYC-001

Latest Information Update: 20 Oct 2025

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At a glance

Originator PYC Therapeutics
Class Antisense oligonucleotides; Eye disorder therapies; Morpholines; Peptide drug conjugates
Mechanism of Action OPA1 protein expression stimulants; RNA interference

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

30 Sep 2025 Phase-I/II clinical trials in Optic atrophy (Treatment-naive) in Australia (Intravitreous) (NCT06970106)
15 May 2025 PYC Therapeutics plans a phase I/II trial for Optic atrophy (Treatment-naive) in Australia (Intravitreous), in June 2025 (NCT06970106)
31 Oct 2024 Phase-I clinical trials in Optic atrophy in Australia (Intravitreous) (NCT06461286)

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