121 0 Kommentare Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) - Seite 2

The oral presentation includes positive, updated data (cut-off September 11, 2023) from the global Phase 1/2 pivotal studies of RP-L102, Rocket’s ex vivo LV gene therapy candidate for FA.

Consistent with results observed from the clinical program, RP-L102 is a potentially curative therapy to prevent FA-related bone marrow failure (BMF), which can be administered without a suitable allogeneic donor or transplant-related toxicities.
RP-L102 demonstrates sustained and progressively increasing genetic correction in eight of 12 patients with greater than 12 months of follow-up. Observed genetic correction is associated with phenotypic correction and hematologic stability.
RP-L102 remains well-tolerated with no significant safety signals.
For the first time, data demonstrate that RP-L102 confers polyclonal insertion patterns indicative of long-term hematopoietic stem cell repopulation of the bone marrow and peripheral blood and clonal diversity in the absence of conditioning.
Based on the positive efficacy and safety data from the global pivotal studies of RP-L102 for FA, the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for review. Rocket expects to submit the BLA to the FDA in the first half of 2024.

Gene Therapy for Adult and Pediatric Patients with Severe Pyruvate Kinase Deficiency: Results from a Global Study of RP-L301

The oral presentation includes positive, updated data (cut-off February 5, 2024) from the Phase 1 study from two adult patients with PKD treated with RP-L301 followed up to 36 months and two pediatric patients followed up to 12 months.

Sustained and clinically meaningful hemoglobin improvement observed in all patients including hemoglobin normalization in three of four patients. No patients have required red blood cell transfusion following neutrophil engraftment. Improvements in hemoglobin supported by improved markers of hemolysis and quality of life have been observed.
RP-L301 remains well-tolerated, with no drug-related serious adverse events.
Insertion site analyses in the peripheral blood and bone marrow for both adult patients through 36 months post-RP-L301 demonstrated highly polyclonal patterns with no clonal dominance or insertional mutagenesis. Testing is ongoing for pediatric patients who were more recently treated.
Based on the positive safety and efficacy data from the global Phase 1 study of RP-L301 for PKD, Rocket is working towards initiation of the Phase 2 pivotal study.