Gene therapy company Rocket Pharmaceuticals Inc (Nasdaq:RCKT) reported on Tuesday the receipt of the US Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations for RP-L102 based on the positive efficacy and safety results from the ongoing Phase 1/2 clinical trial in Europe for the treatment of Fanconi Anemia (FA).

A rare paediatric disease FA is reportedly characterised by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life.

The company added that RP-L102 is its lentiviral vector (LVV)-based gene therapy in development for patients with FA at Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain, CIBER-Rare Diseases and IIS-Fundación Jiménez Díaz.

Concurrently, the International Fanconi Anemia Gene Therapy Working Group helped the development of new generation of FA gene therapy programmes, which began with a HIV-1-derived, self-inactivating lentiviral vector. The ex vivo administration process begins with the removal and isolation of hematopoietic stem cells using a CD34+ selection process. Autologous genetically modified CD34+ enriched hematopoietic cells (fresh or cryopreserved) are infused back into patients to restore function.

In early 2019, the company intends to initiate a clinical trial of RP-L102 for FA utilising no conditioning and "Process B" which incorporates higher cell doses, transduction enhancers and commercial-grade vector manufacturing and cell processing. The global trial is expected to enroll approximately 12 FA patients at the Center for Definitive and Curative Medicine at Stanford University School of Medicine, Hospital Niño Jesús/CIEMAT and other leading centres in the US and in the EU.