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SGT 501

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Drug Profile

SGT 501

Alternative Names: SGT-501

Latest Information Update: 27 Jan 2025

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At a glance

  • Originator Solid Biosciences
  • Class Antiarrhythmics; Gene therapies
  • Mechanism of Action CASQ2 protein replacements; Gene transference; Ryanodine receptor calcium release channel replacements
  • Orphan Drug Status

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

    Yes - Polymorphic catecholergic ventricular tachycardia
  • New Molecular Entity No

Highest Development Phases

  • Preclinical Polymorphic catecholergic ventricular tachycardia

Most Recent Events

  • 15 Jan 2025 Solid Biosicences plans to file an IND application for Polymorphic catecholergic ventricular tachycardia in first half of 2025
  • 31 Dec 2024 SGT 501 receives Rare Pediatric Disease designation from the FDA for Polymorphic catecholergic ventricular tachycardia before December 2024
  • 06 Nov 2024 Solid Biosciences completes a pre-IND meeting with the US FDA for SGT 501

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