Drug Profile
Research programme: rare liver disease gene therapies - Dimension Therapeutics/University of Pennsylvania
Alternative Names: DTX 501; DTX 601; DTX 701; DTX501 AAV PAH; DTX601 AAV ASS1; DTX701 AAV ATP7BLatest Information Update: 28 Jun 2020
Price :
$50
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At a glance
- Originator Dimension Therapeutics; University of Pennsylvania
- Developer University of Pennsylvania
- 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
- No development reported Liver disorders
Most Recent Events
- 28 Jun 2020 No recent reports of development identified for research development in Liver-disorders in USA
- 07 Nov 2017 Dimension Therapeutics has been acquired by Ultragenyx Pharmaceutical
- 05 May 2016 Early research in Liver disorders in USA (unspecified route)