Express treatment of fatal childhood immunodeficiency, initial clinical trial results of innovative gene therapy are positive

▎WuXi AppTec content team editor A few days ago, Rocket Pharmaceuticals announced that its research gene therapy RP-L201 has obtained positive interim data in a phase 1/2 clinical trial for the treatment of leukocyte adhesion deficiency type I (LAD-I)
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Severe LAD-I is a rare autosomal recessive pediatric disease that can cause recurrent life-threatening bacterial and fungal infections, leading to frequent hospitalizations and ultimately fatalities
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The clinical manifestations of type I leukocyte adhesion deficiency are delayed umbilical cord shedding, repeated soft tissue infections and chronic periodontitis
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The disease is caused by mutations in the ITGB2 gene, which encodes the β-2 integrin component CD18
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This gene mutation leads to a decrease in the expression of CD18, and white blood cells cannot migrate from the blood vessels to the infected tissue to resist pathogens
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The current main treatment method is bone marrow transplantation.
If bone marrow transplantation is unsuccessful, the mortality rate of patients with severe LAD-I before the age of 2 is 60-75%, and the survival rate of patients over 5 years of age is extremely low
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Therefore, severe pediatric patients have highly unmet medical needs
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RP-L201 is an in vitro autologous hematopoietic stem cell gene therapy based on lentiviral vector
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The therapy uses lentiviral vectors to genetically modify cells to express the ITGB2 gene with normal functions
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Image source: Rocket Pharmaceuticals official website This interim data comes from 7 patients with severe LAD-I, whose CD18 expression is less than 2%
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The results of the test showed that all patients showed significant initial efficacy, and two of them were followed up for at least 12 months
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All 7 patients showed persistent neutrophil CD18 expression, which significantly exceeded the 4-10% threshold associated with survival to adulthood
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After RP-L201 treatment, no patients had LAD-I related infections requiring hospitalization
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In terms of safety, RP-L201 has good safety characteristics, all infusions are well tolerated, and there are no serious drug-related adverse events (SAE)
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Dr.
Jonathan Schwartz, Chief Medical Officer of Rocket Pharmaceuticals, said: "We are pleased to share the latest positive data from the Phase 1/2 trial for LAD-I
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They continue to support RP-L201 in producing lasting clinical benefits in patients with severe LAD-I.
We look forward to reporting additional clinical data later in the fourth quarter
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"Reference: [1] Rocket Pharmaceuticals Announces Positive Clinical Data from RP-L201 Trial for the Treatment of LAD-I at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT).
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