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RP-L102 Offers Hope for Children With Fanconi Anemia

Gene Therapy Granted Regenerative Medicine, Fast Track Designations

On November 27, RP-L102 (Rocket Pharmaceuticals), a lentiviral vector-based gene therapy for the treatment of Fanconi Anemia (FA), was granted regenerative medicine advanced therapy and fast track designations by the FDA.

FA is a rare pediatric disease characterized 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.

Allogeneic hematopoietic stem cell transplantation (HSCT) corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic. HSCT is frequently complicated by graft-versus-host disease and it also increases the risk of solid tumors, primarily squamous cell carcinomas.

RP-L102’s lentiviral vector carries the FANC-A gene as part of the PGK-FANCA-WPRE expression cassette, which includes a phosphoglycerate kinase (PKG) promoter and an optimized woodchuck hepatitis virus posttranscriptional regulatory element (WPRE). After the removal and isolation of hematopoietic stem cells, genetically modified CD34+ enriched hematopoietic cells are infused back into patients to restore function.

In addition to its orphan drug, fast track, and regenerative medicine advanced therapy (RMAT) designations for the treatment of FA type A in the U.S., RP-L102 has also received orphan drug designation in Europe.

Source: Rocket Pharma, November 27, 2018,

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