Osteogenesis imperfecta is a rare genetic disorder characterised by fragile bones and reduced bone mass. Credit: Darwin Laganzon from Pixabay.

Rocket Pharmaceuticals has started treatment with its investigational gene therapy, RP-L201, in a Phase I/II clinical trial to treat severe Leukocyte Adhesion Deficiency-I (LAD-I).

RP-L201 is a lentiviral vector (LVV)-based therapy that contains autologous genetically modified CD34+ hematopoietic cells. Rocket licensed the product from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain.

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LAD-I is a rare disorder caused by a mutation of the ITGB2 gene. The paediatric disease can lead to fatal infections as white blood cells lose the ability to leave the bloodstream to fight the infection.

Without a successful bone marrow transplant, severe LAD-I is associated with 60-75% mortality in the initial two years of life and survival beyond five years is rare.

The non-randomised, open-label Phase I/II study is designed to assess RP-L201 for the treatment of severe LAD-I in nine paediatric patients worldwide.

“Our hope is that patients would benefit from a one-time administration of autologous gene therapy.”

Phase I of the study will investigate the gene therapy’s safety and tolerability, while the Phase II portion will monitor the participants’ overall survival.

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The survival will be established by the proportion of patients alive at the age of two and a minimum of one-year after infusion without allogeneic hematopoietic stem cell transplant (HSCT).

In addition, the Phase II portion will characterise the safety and toxicity of the RP-L201 infusion.

Rocket Pharmaceuticals CEO and president Gaurav Shah said: “LAD-I represents a major area of unmet medical need as most severely-afflicted patients die before the age of two in the absence of a hematopoietic stem cell transplant, which is associated with graft-versus-host disease.

“Our hope is that patients would benefit from a one-time administration of autologous gene therapy facilitated with busulfan conditioning. This process does not require immunosuppression and eliminates the risk of graft-versus-host disease.”

The company expects to complete the Phase I/II trial in 2023.