Rocket Pharma Posts Interim Data From Immunodeficiency Disorder Gene Therapy Trial

Rocket Pharmaceuticals Inc RCKT has announced interim data updates from seven patients from the RP-L201 Phase 1/2 trial for Leukocyte Adhesion Deficiency-I (LAD-I).
Data were shared at the Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). RP-L201 is Rocket's ex-vivo lentiviral gene therapy candidate.
The interim data demonstrated preliminary efficacy in all seven patients with durable neutrophil CD18 expression that exceeded the 4-10% threshold.
Peripheral blood vector copy number (VCN) levels have been stable and in the 0.5 – 2.5 copy per genome range.
No patients have had LAD-I-related infections requiring hospitalization after hematopoietic reconstitution post-RP-L201.
The safety profile of RP-L201 appears favorable, with all infusions well tolerated and no drug product-related serious adverse events.
Severe LAD-I is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding the beta-2 integrin component CD18.
CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections.
Read Next: FDA Lifts Clinical Hold On Rocket Pharma's X-Linked Inherited Disorder Trial.
Price Action: RCKT shares traded higher 3.06% higher at $29 premarket on the last check Wednesday.