Rocket Pharmaceuticals Announces FDA Clearance of IND for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis

—Rocket’s Fifth Gene Therapy Program to Enter the Clinic in the Fourth Quarter of 2020—

NEW YORK, NY, USA I June 29, 2020 I Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-L401. RP-L401 is the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosis (IMO), a rare, severe monogenic bone resorption disorder characterized by skeletal deformities, neurologic abnormalities and bone marrow failure. RP-L401 was in-licensed from Lund University, under the research leadership of Dr. Johan Richter, M.D., Ph.D. and Dr. Ilana Moscatelli, Ph.D. The vector was in-licensed through a collaboration with Dr. Axel Schambach, M.D., Ph.D. of the Medizinische Hochschule Hannover.

“This FDA acceptance marks the fifth Rocket-Sponsored IND cleared for our gene therapy platform in less than two years and is an important milestone for Rocket,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “IMO represents a significant unmet medical need as patients often die in the first years of life, and the only current treatment option is a bone marrow transplant, which is associated with difficult complications. Promising preclinical data indicate that even a modest level of engraftment of 10-30% corrected hematopoietic progenitors can restore bone resorption and potentially prevent the devastating morbidity and childhood mortality associated with IMO. We are looking forward to opening the clinical trial as we work to provide a potentially curative treatment option for patients with IMO.”

Kinnari Patel, Pharm. D., MBA, Chief Operating Officer and Executive Vice President, Development at Rocket, added, “Our IMO program represents the essence of Rocket’s vision and mission – to find potentially curative gene therapies for rare, devastating childhood disorders. These patients need more treatment options, and the rapid advancement of this program is a testament to the hard work and expertise of the Rocket team and its academic collaborators.”

The non-randomized, open-label Phase 1 clinical trial will enroll two pediatric patients, one month of age or older. The trial is designed to assess safety and tolerability of RP-L401, as well as preliminary efficacy, including potential improvements in bone abnormalities/density, hematologic status and endocrine abnormalities. University of California, Los Angeles will serve as the lead trial site. The trial will be led by principal investigator Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los Angeles.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood 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 (LVV)-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, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit

SOURCE: Rocket Pharmaceuticals

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