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.

Go deeper with GlobalData

Go deeper with GlobalData

The gold standard of business intelligence.

Find out more

Discover B2B Marketing That Performs

Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.

Find out more

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.

GlobalData Strategic Intelligence

US Tariffs are shifting - will you react or anticipate?

Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.

By GlobalData

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.

Clinical Trials Arena Excellence Awards - Nominations Closed

Nominations are now closed for the Clinical Trials Arena Excellence Awards. A big thanks to all the organisations that entered – your response has been outstanding, showcasing exceptional innovation, leadership, and impact.

Excellence in Action
YPrime won the Innovation award for AI in Clinical Trials and the Environmental award for Sustainable Trials, thanks to its eCOA, IRT and eConsent platforms. Explore how purpose-built AI, paperless workflows and circular hardware practices are reshaping timelines, data quality and ESG performance in clinical research.

Discover the Impact