$RCKT

“the PRV was originally issued in connection with the FDAffds approval of the Companyffds biologics license application for KRESLADIfffd (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.”

“The PRV was awarded following the U.S. Food and Drug Administration (FDA) accelerated approval of KRESLADI™ (marnetegragene autotemcel).”

“The PRV was originally issued in connection with the FDA's approval of the Company's biologics license application for KRESLADI™ (marnetegragene autotemcel), an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.”

“The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to Rocket's Biologics License Application for KRESLADI™ wherein the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review.”

“On October 3, 2025, Rocket Pharmaceuticals, Inc. ("Rocket" or the "Company") reported that it has voluntarily withdrawn its Biologics License Application ("BLA") to the U.S. Food and Drug Administration ("FDA") for mozafancogene autotemcel (also known as "Fanskya," "Fanca-cel," or "RP-L102"), an investigational gene therapy for the treatment of Fanconi Anemia ("FA").”