Drug Profile
Sesiclenegene cosaparvovec - Amicus Therapeutics
Alternative Names: AT-GTX-501; CLN6 Batten Disease - Amicus Therapeutics; CLN6 gene therapy - Amicus Therapeutics; scAAV9.CB.CLN6Latest Information Update: 01 Feb 2022
Price :
$50
*
At a glance
- Originator Nationwide Children's Hospital; Ohio State University
- Developer Nationwide Children's Hospital
- Class Gene therapies
- Mechanism of Action CLN6 protein stimulants; Gene transference
-
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
- Discontinued Neuronal ceroid lipofuscinosis
Most Recent Events
- 24 Jan 2022 Amicus Therapeutics completes a phase I/II trial for Neuronal ceroid lipofuscinosis (In children, In adolescents, In adults) in USA (Intrathecal) (NCT02725580)
- 10 Jan 2022 Discontinued - Phase-I/II for Neuronal ceroid lipofuscinosis (In adolescents, In children, In adults) in USA (Intrathecal)
- 23 Mar 2021 scAAV9.CB.CLN6 is still in phase I/II trial in Neuronal ceroid lipofuscinosis in USA (NCT02725580)