RB 001
Alternative Names: RB-001Latest Information Update: 04 Jul 2025
At a glance
- Originator Shenzhen Reborngene Therapeutics
- Class Gene therapies
- Mechanism of Action Gene transference; SHANK3 protein replacements
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- 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)