Business
Rocket Pharmaceuticals Announces Positive Clinical Data from RP-L201 Trial for the Treatment of LAD-I at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT)
—RP-L201 is well tolerated and leads to durable CD18 expression and consistent peripheral vector copy numbers in seven of the initial seven patients with
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—RP-L201 is well tolerated and leads to durable CD18 expression and consistent peripheral vector copy numbers in seven of the initial seven patients with follow-up ranging from 3 to 18 months —\n\n— Positive data updates show initial clinical benefit with no patients requiring hospitalization to date for LAD-I related infections following hematopoietic reconstitution —\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces interim data updates from the RP-L201 Phase 1/2 clinical trial for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I) at the 28th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT). Severe LAD-I is a rare pediatric disease that prevents patients from adequately combating infections. LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antimicrobials, require frequent hospitalizations and are ultimately fatal.\n\n“We are excited to share positive data updates from our Phase 1/2 trial for LAD-I and are pleased to report that in each of the initial seven RP-L201 treated patients, CD18 expression has significantly exceeded the 4-10% threshold associated with survival into adulthood, with consistent peripheral blood vector copy number levels,” said Jonathan Schwartz, M.D., Chief Medical Officer of Rocket Pharma. “These positive data continue to support the potential of RP-L201 to yield durable clinical benefit in patients with severe LAD-I and advances our potentially registration-enabling dataset in this fatal disorder and across Rocket’s lentiviral programs at large. We look forward to reporting additional clinical data later in the fourth quarter.”\n\nA Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results\n\nThe data presented at ESGCT are from the initial seven patients with severe LAD-I, as defined by CD18 expression of less than 2%, who were treated with RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate. The safety profile of RP-L201 appears favorable with all infusions well tolerated and no drug product-related serious adverse events (SAEs).\n\nPreliminary efficac...