Rocket Pharma Posts Interim Data From Immunodeficiency Disorder Gene Therapy Trial
- Rocket Pharmaceuticals Inc (NASDAQ: 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.
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