Drug Profile
Avalotcagene ontaparvovec - Dimension Therapeutics
Alternative Names: DTX 301; Ornithine-transcarbamylase-gene-therapy-Dimension-Therapeutics; OTC-gene-therapy-Dimension-Therapeutics; scAAV8OTCLatest Information Update: 05 Nov 2023
Price :
$50
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At a glance
- Originator Dimension Therapeutics
- Developer Dimension Therapeutics; Ultragenyx Pharmaceutical
- Class Gene therapies
- Mechanism of Action Gene transference; Ornithine carbamoyltransferase replacements
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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 III Inborn urea cycle disorders
Most Recent Events
- 15 Feb 2023 Phase-III clinical trials in Inborn urea cycle disorders (In adolescents, In adults, In the elderly) in Spain, Canada, USA (IV) (NCT05345171)
- 06 Jan 2023 Ultragenyx plans a phase III (eNH3ance study) for Inborn urea cycle disorders in the first quarter of 2023
- 31 Dec 2022 Ultragenyx Pharmaceutical has patent protection for two patent families covering elements of DTX 301