2 April 2024 - Positive, previously disclosed results from the global Phase 1/2 trial demonstrated genetic and phenotypic correction combined with haematologic stabilisation extending out to 42 months after treatment with RP-L102.

Rocket Pharmaceuticals today announced that the EMA accepted the marketing authorisation application for RP-L102, its lentiviral vector-based investigational gene therapy for Fanconi anaemia, complementation group A, a rare genetic disorder caused by mutations in the FANCA gene affecting DNA repair and characterised by bone marrow failure, cancer predisposition, and congenital malformations.

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