Drug Profile
scAAV1.tMCK.NTF3 - Nationwide Children's Hospital/Sarepta Therapeutics
Alternative Names: AAV1.NT-3 gene therapy - Nationwide Children's Hospital; Neurotrophin-3 gene therapy - Nationwide Children's Hospital; rAAV1.NT-3 gene therapy - Nationwide Children's HospitalLatest Information Update: 02 Oct 2021
Price :
$50
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At a glance
- Originator Nationwide Children's Hospital
- Class Gene therapies
- Mechanism of Action Gene transference
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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
- Phase I/II Charcot-Marie-Tooth disease
Most Recent Events
- 23 Mar 2021 scAAV1.tMCK.NTF3 is still in phase I/II trial in Charcot-Marie-Tooth disease in USA (NCT03520751)
- 27 May 2019 Sarepta Therapeutics plans a clinical trial for Charcot-Marie-Tooth disease in 2019
- 08 Oct 2018 Nationwide Children’s Hospital and Sarepta Therapeutics enter an option agreement for neurotrophin-3 gene therapy for the treatment of Charcot-Marie-Tooth disease