Drug Profile
Research programme: neurodegenerative disorders therapeutics - Spark Therapeutics
Alternative Names: SPK miHTTLatest Information Update: 27 Dec 2023
Price :
$50
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At a glance
- Originator University of Iowa Research Foundation
- Developer Spark Therapeutics
- 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
- Preclinical Huntington's disease
- No development reported Neuronal ceroid lipofuscinosis
Most Recent Events
- 22 Dec 2023 Preclinical development is ongoing for Huntington's disease in USA (Spark Therapeutics pipeline, December 2023)
- 28 May 2023 No recent reports of development identified for preclinical development in Huntington's-disease in USA (Parenteral)
- 28 May 2021 Preclinical development is ongoing for Huntington's disease in USA (Spark Therapeutics pipeline, May 2021)