Trial Profile
A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A
Status:
Active, no longer recruiting
Phase of Trial:
Phase I
Latest Information Update: 08 Nov 2023
Price :
$35
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At a glance
- Drugs Mozafancogene autotemcel (Primary)
- Indications Fanconi's anaemia
- Focus Adverse reactions; Registrational
- Sponsors Rocket Pharmaceuticals
- 06 Nov 2023 According to a Rocket Pharmaceuticals media release, Product filings for RP-L102 anticipated in the first half of 2024 in the U.S. and Europe. The company is finalizing the CMC package with the FDA.
- 09 Jan 2023 According to a Rocket Pharmaceuticals media release, BLA filling for FA is on track for Q4 2023.
- 13 Dec 2022 Updated results presented at the 64th American Society of Hematology Annual Meeting and Exposition.