Lonza Supporting Rocket Pharma with Clinical Manufacturing of RP-L201 for the Treatment of Leukocyte Adhesion Deficiency-I

Lonza announced that a clinical manufacturing agreement is in place with Rocket Pharmaceuticals, Inc., a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders. The agreement covers development and manufacturing of RP-L201, a phase 1/2 candidate for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).

The companies have been working together since 2018 and decided to make the collaboration public in parallel to the successful Preliminary Data from Phase 1/2 Trial announced by Rocket on December 9th, 2019.

The agreement covers the manufacturing of clinical product leveraging Lonza’s lentiviral vector platform. Manufacturing takes place in both the Houston (TX), USA and Geleen, Netherlands Lonza cGMP manufacturing sites. The agreement also includes analytical assays and development services.

Autologous hematopoietic stem and progenitor cells (HSPCs) are transduced ex vivo with a lentiviral vector (LV) containing the ITGB2 gene encoding for the human CD18 receptor (β2 integrin subunit). The therapy aims to treat Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare immune disorder characterized by low or absent neutrophil CD18 expression, predisposing affected individuals to recurrent and fatal infections in childhood. Initial results from the first patient treated with RP-L201 demonstrated early evidence of safety and potential efficacy.