Drug Profile
Research programme: gene therapies - Spark Therapeutics
Latest Information Update: 28 Mar 2023
Price :
$50
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At a glance
- Originator The Childrens Hospital of Philadelphia
- 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 Neurodegenerative disorders
- No development reported Haematological disorders
Most Recent Events
- 27 Mar 2023 Preclinical development in Neurodegenerative disorders is ongoing in USA (Parenteral) (Spark Therapeutics pipeline, March 2023)
- 28 Feb 2021 No recent reports of development identified for preclinical development in Haematological-disorders in USA (Parenteral)
- 28 Feb 2021 No recent reports of development identified for preclinical development in Neurodegenerative-disorders in USA (Parenteral)