Rocket Pharmaceuticals Receives Funding From The California Institute For Regenerative Medicine For Phase 1 Clinical Trial Of RP-L401 For Infantile Malignant Osteopetrosis

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 the California Institute for Regenerative Medicine...
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Rocket Pharmaceuticals, Inc. (RCKT) - Get Report ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the California Institute for Regenerative Medicine (CIRM) has awarded Rocket a $3.7 million CLIN2 grant award to support the clinical development of its lentiviral vector (LVV)-based gene therapy, RP-L401, 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. The CIRM was founded in 2004 following the passing of Proposition 71 or the California Stem Cell Research and Cures Initiative, which allowed $3 billion in state funding for stem cell research conducted in California. This will be Rocket's second CIRM grant after receiving one in 2019 for the development of the company's gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I).

"We're grateful the CIRM has recognized the promise of RP-L401 for IMO, a devastating pediatric rare disease for which the primary treatment option is allogeneic bone marrow transplant," said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. "RP-L401 could be a potentially curative treatment for this devastating disorder that affects children at a young age, and we are thankful to have this meaningful support from the CIRM to move our program forward for these families."

Rocket's Investigational New Drug Application (IND) for RP-L401 was accepted by the U.S. Food and Drug Administration (FDA) in June of 2020, and the gene therapy received Fast Track designation from the FDA in August 2020. Proceeds from the grant will help fund clinical trial costs, as well as provide manufactured drug product for Phase 1 patients enrolled at the U.S. clinical trial site, University of California, Los Angeles, 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. 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. Further information about the clinical program is available here.

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.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover. Rocket's IMO research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-12095). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (RCKT) - Get Report ("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 www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rocket's ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed November 6, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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