Investigational Gene Therapy for Fanconi Anemia Approved in the European Union | Docwire News

Not classified

The MAA for RP-L102, an investigational gene therapy for Fanconi anemia, has been approved by the EMA.

RP-L102 is a gene therapy containing autologous hematopoietic stem cells that have been modified to contain a functional copy of the FANCA gene. RP-L102 has the Priority Medicines and Advanced Therapy Medicinal Product designations in the European Union and the Regenerative Medicine Advanced Therapy, Rare Pediatric Disease, and Fast Track designations in the United States.

More infos

Back to news