Drug Profile
Mozafancogene autotemcel - Rocket Pharmaceuticals
Alternative Names: LV-RP-L102; RP L102; RP-L02Latest Information Update: 08 Nov 2023
Price :
$50
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At a glance
- Originator CIEMAT
- Developer CIEMAT; Rocket Pharmaceuticals
- Class Antianaemics; Gene therapies; Stem cell therapies
- Mechanism of Action Fanconi anaemia complementation group A protein expression stimulants; 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
- Phase II Fanconi's anaemia
Most Recent Events
- 08 Nov 2023 Rocket Pharmaceuticals plans for regulatory filings in the US and Europe in 1H of 2024
- 19 May 2023 Efficacy and adverse events data from phase-II trial in Fanconi's anaemia presented presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT-2023)
- 27 Feb 2023 Rocket pharmaceuticals announces intention to submit BLA for Fanconi's anaemia in the fourth quarter of 2023