CIRM-Funded Program Targeting Rare Pediatric Disease Announces FDA Acceptance of Biologics License Application with Priority Review

South San Francisco, CA – The California Institute for Regenerative Medicine (CIRM), the world’s largest institute dedicated to regenerative medicine, would like to congratulate Rocket Pharmaceuticals, Inc, a leading late-stage biotechnology company, on receiving U.S. FDA acceptance of the Biologics License Application (BLA) and Priority Review for RP-L201 (marnetegragene autotemcel), a lentiviral vector (LV)-based gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I) that received funding and support from CIRM.

The BLA acceptance by the FDA marks a significant milestone towards the goal of delivering a one-time gene therapy to patients facing the devastating effects of LAD-I, a rare pediatric genetic immune disorder that predisposes patients to recurrent and fatal infections, for whom survival beyond childhood is unlikely. The Prescription Drug User Fee Act (PDUFA) date set by the FDA is March 31, 2024.

“Currently, the only treatment option is a bone marrow transplant, which has substantial morbidity and mortality, and may not be accessible in time for these children,” said Kinnari Patel, PharmD, MBA, President and Chief Operating Officer, Rocket Pharma. “We are grateful to CIRM for providing funding to support this critical research, as well as the patients, caregivers and researchers who have shared this journey with us. We look forward to continuing to work towards bringing RP-L201 to patients as quickly as possible.”

CIRM provided $5.9 million funding to support clinical trial testing of RP-L201. The gene therapy uses a patient’s own blood stem cells and inserts a corrected version of the mutated gene.  These modified stem cells are then reintroduced back into the patient. The goal is to establish functional immune cells, enabling the body to combat infections.

LAD-I leads to recurring bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and can be fatal. It is caused by a mutation in a specific gene that causes low levels of a protein called CD18. The low levels of CD18 reduce the immune system’s ability to work efficiently and combat infections.

“Rocket’s dedication, along with CIRM’s support, has been instrumental in bringing this potential life-saving gene therapy closer to reality,” said Maria T. Millan, MD, President and CEO of CIRM. “This milestone represents a significant step in our commitment to advancing innovative therapies 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 that contains autologous (patient-derived) hematopoietic stem cells that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection. Rocket holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations in the U.S., PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU, and Orphan Drug designation in both regions for the program. RP-L201 was in-licensed from the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigación Biomédica en Red de Enfermedades Raras and Instituto de Investigación Sanitaria Fundación Jiménez Díaz. The lentiviral vector was developed in a collaboration between UCL and CIEMAT.

About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today’s most promising stem cell technologies. With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world’s largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

For more information go to www.cirm.ca.gov

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket’s clinical programs using lentiviral vector (LV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also is developing AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM). For more information about Rocket, please visit www.rocketpharma.com.