ASGCT 2023: Rocket's hematology programs blast off toward regulators

As the American Society of Gene & Cell Therapy annual meeting draws to a close, Rocket Pharmaceuticals spotlighted three ongoing clinical programs for patients with blood disorders, two of which are nearing regulators’ desk. 

The company teased new data Friday from all three of its lentiviral gene therapy programs targeting blood disorders, including updated follow-up figures from an open-label phase 1/2 trial of its severe leukocyte adhesion deficiency-I (LAD-1) program. Rocket reported 100% overall survival at 12 months among nine LAD-1 patients with 12 to 24 months of follow-up. The company also reports that treated patients were seeing improvements in disease-related skin rash and “restoration of wound repair capabilities.” The company plans to file an approval application with the FDA before the end of June. 

Just a few months behind is Rocket’s lentiviral gene therapy for patients with Fanconi anemia, which the company expects to submit to U.S. regulators for approval in the fourth quarter of the year. Fueling the regulatory liftoff are new phase 2 data showing that RP-L102 spurred “sustained genetic correction” in eight of 12 evaluable patients and “comprehensive phenotypic correction” in seven of 12 patients with at least a year of follow-up. 

No serious safety signals have been observed so far, Rocket says, including no evidence of “insertional mutagenesis” in treated patients. The pivotal trial is now fully enrolled, and all patients have been dosed. 

The company also came equipped with data from the first pediatric patient with pyruvate kinase deficiency dosed with ex vivo lentiviral gene therapy RP-L301. Rocket says initial signs “suggest similar efficacy” as the long-term efficacy data in the adult cohort. Engraftment was achieved at Day 15, and hemoglobin levels stabilized six weeks post-infusion. 

New data from two adults show sustained hemoglobin levels and improvement in quality of life up to 30 days post-infusion. Rocket expects to launch a phase 2 trial of the treatment in the fourth quarter of 2023, with phase 1 enrollment complete for both adults and children.