Gene Therapy for LAD-I Posts Impressive Survival Data


In an interview with CGTLive, study primary investigator Donald Kohn, MD, discussed the safety and efficacy observed in the phase 2 trial.

Patients treated with an investigational gene therapy to address severe leukocyte adhesion deficiency type 1 (LAD-I) showed impressive survival data and clinically significant reductions in hospitalizations and infections, according to findings presented at the 2022 American Society of Gene and Cell Therapy Annual Meeting, May 16-19, 2022, in Washington, DC.

As of the data cutoff for the phase 1/2 trial (NCT03812263), 7 of 9 patients dosed with the lentiviral gene therapy from Rocket Pharmaceuticals with at least 12 months of follow-up available had a 100% overall survival rate without allogeneic hematopoietic stem cell transplantation based on the Kaplan-Meier estimate post-infusion. In addition, all 9 treated patients have demonstrated statistically significant reductions in all-cause hospitalizations and severe infections, as well as resolution or improvement of all LAD-I-related skin abnormalities, including rashes and wound healing.

In addition, no treatment-related serious adverse events were recorded, and all patients in the cohort have demonstrated sustained CD18 restoration and expression on more than 10% of neutrophils.

The gene therapy consists of autologous hematopoietic stem cells that are genetically modified with a lentiviral vector that contains a functional copy of the ITGB2 gene, mutations in which cause deficiencies in beta-2 integrin.

In an interview with CGTLive, primary investigator Donald B. Kohn, MD, distinguished professor of microbiology, immunology, molecular genetics, and pediatrics (hem/onc), and director, Human Gene Medicine Program at UCLA, detailed the exciting findings and his hope that the treatment will soon become available. Watch below:

1. Kohn DB, Sevilla J, Rao G, et al. 1188: Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I). Presented at: 2022 American Socienty of Gene and Cell Therapy Annual Meeting. May 16-19, 2022; Washington, DC.
2. Rocket Pharmaceuticals Presents Positive Top-line Data from Severe Leukocyte Adhesion Deficiency-I Program at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). News release. Rocket Pharmaceuticals. May 19, 2022.
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