PYC Therapeutics Advances Polycystic Kidney Disease Therapy

PYC Therapeutics (ASX: PYC), a clinical-stage biotech firm, is pioneering RNA-based treatments for genetic disorders. Its lead candidate, PYC-003, targets Polycystic Kidney Disease (PKD)—a condition affecting over 10 million people globally with no current therapies. The company recently completed the first cohort of its Phase 1a Single Ascending Dose (SAD) trial in healthy volunteers and received approval to escalate dosing in the second cohort. Subject to safety reviews, this will enable further dosing and initiate trials in PKD patients by Q3 2025. The study will progress to include an Open-Label Extension and a Phase 1b Multiple Ascending Dose (MAD) trial. If successful, PYC-003 will enter a pivotal Phase 2/3 trial, potentially supporting a New Drug Application. Leveraging a proprietary drug delivery platform, PYC focuses on monogenic diseases—conditions with higher chances of successful clinical outcomes

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