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RB 001

Drug Profile

RB 001

Alternative Names: RB-001

Latest Information Update: 04 Jul 2025

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At a glance

  • Originator Shenzhen Reborngene Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; 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 No

Highest Development Phases

  • Preclinical Telomeric 22q13 Monosomy Syndrome

Most Recent Events

  • 10 Jun 2025 Preclinical trials in Telomeric 22q13 Monosomy Syndrome in China (Intraventricular) prior to June 2025
  • 10 Jun 2025 Pharmacodynamics data from a preclinical study in Telomeric-22q13 monosomy syndrome released by Shenzhen Reborngene Therapeutics
  • 01 Jun 2025 Peking University First Hospital in collaboration with Shenzhen Reborngene Therapeutics plans a clinical trial for Telomeric-22q13 monosomy syndrome (In children, In adolescents) in China (Intraventricular, Injection) in June 2025 (NCT07014020)

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