The California Institute for Regenerative Medicine (CIRM) congratulates Rocket Pharmaceuticals, Inc, a leading late-stage biotechnology company. The company received U.S. FDA acceptance of the Biologics License Application (BLA) and Priority Review for RP-L201 (marnetegragene autotemcel). CIRM helped support the development of this entiviral vector (LV)-based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).
What BLA Means
The FDA’s acceptance of the BLA marks a major step toward delivering a one‑time gene therapy for LAD‑I, a rare pediatric immune disorder. LAD-I causes recurrent and often fatal infections. Without treatment, most affected children do not survive beyond childhood. The FDA set the Prescription Drug User Fee Act (PDUFA) date for March 31, 2024.
“Right now, the only treatment is a bone marrow transplant, which carries significant risks,” said Kinnari Patel, PharmD, MBA, President and Chief Operating Officer of Rocket Pharma. “We are grateful to CIRM for funding this critical research. We remain committed to bringing RP‑L201 to patients as quickly as possible.”
CIRM Funding
CIRM provided $5.9 million to support the clinical trial of RP‑L201. The gene therapy modifies a patient’s own blood stem cells by inserting a corrected version of the mutated gene. Clinicians then reinfuse the engineered cells to restore functional immune cells that can fight infections.
LAD‑I causes recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations, and can be fatal. A mutation in the gene that produces the CD18 protein disrupts immune function and weakens the body’s ability to combat infections.
“Rocket’s dedication, along with CIRM’s support, has been instrumental in advancing this potential life‑saving therapy,” said Maria T. Millan, MD, President and CEO of CIRM. “This milestone represents a major step in our commitment to developing innovative treatments that can transform the lives of children and families affected by severe LAD‑I.”
About RP-L201 (marnetegragene autotemcel)
RP‑L201 is an investigational gene therapy made from a patient’s own hematopoietic stem cells. These cells are genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene, which encodes CD18 — a key beta‑2 integrin protein that enables leukocytes to adhere and move out of blood vessels to fight infection.
The program holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease, and Fast Track designations, as well as PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU and Orphan Drug status in both regions.
RP‑L201 was in‑licensed from CIEMAT, the Centro de Investigación Biomédica en Red de Enfermedades Raras, and the Instituto de Investigación Sanitaria Fundación Jiménez Díaz. The lentiviral vector was developed through a collaboration between University College London (UCL) and CIEMAT.
For more information about Rocket, please visit www.rocketpharma.com.