Drug Profile
Mozafancogene autotemcel - Rocket Pharmaceuticals
Alternative Names: LV-RP-L102; RP L102; RP-L02Latest Information Update: 05 Apr 2024
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
- Preregistration Fanconi's anaemia
Most Recent Events
- 02 Apr 2024 Preregistration for Fanconi's anaemia (In children, In infants) in Europe (IV)
- 02 Apr 2024 Rocket Pharmaceuticals plans for regulatory filings in Europe in 1H of 2024
- 02 Apr 2024 Rocket Pharmaceuticals plans to file biologics license application (BLA) for FA to the US FDA in the first half of 2024