Rocket Pharmaceuticals Announces Publication of Manuscript Evaluating Mosaicism in Fanconi Anemia

NEW YORK–(COMMERCIAL THREAD) –Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company developing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, announces recent publication from a peer-reviewed manuscript: the first comprehensive review of somatic mosaicism in Fanconi anemia (FA) in the journal Annals of hematology. The review, titled “Mosaicism in Fanconi Anemia: Concise Review and Assessment of Published Cases with an Emphasis on the Clinical Course of Blood Count Normalization”, summarizes the existing literature regarding mosaicism in FA. , a phenomenon previously described as “natural gene therapy”. Mosaicism occurs when a second spontaneous mutation occurs in an FA gene (already mutated), resulting in a functional FA protein and restored cellular DNA repair capacity. When this type of reversion or compensatory mutation occurs in hematopoietic stem cells (HSCs), these cells have sometimes been able to restore a more normalized blood and bone marrow environment, in some cases over decades. The review provides the FA community with an additional resource to better understand the phenomenon and further supports Rocket’s approach of treating patients with RP-L102, the Company’s gene therapy candidate for FA, without conditioning regime prior to treatment.

“Although mosaicism in AF has been discussed within the medical and research community for more than two decades, uncertainty as to the clinical significance of reversion mutations has remained,” said Jonathan Schwartz, MD , Medical Director and Senior Vice President of Rocket. “It is important to note that the results of this review support the advantage of selective growth of gene-corrected stem cells over diseased stem cells, potentially avoiding the need for conditioning in AF gene therapy. In addition to giving us a better understanding of how the RP-L102 can best help patients, we hope that bringing this data together into a cohesive review can better help the entire FA community as we work together to optimize treatment options.

The publication includes a literature-based assessment of AF mosaicism and the identification of a cohort of cases that most likely resulted from reversion in long-term repopulated HSC populations. In addition, the manuscript details diagnostic methods and results of published cases of mosaicism with an evaluation of cases in which patients with mosaic AF have been followed for long term in a clinical setting. These results provide further details on the mechanism and timeline by which gene-corrected cells can repopulate the blood and bone marrow of patients after treatment with Rocket’s gene therapy candidate, RP-L102, which is currently in development. ‘study in a global study allowing phase 2 registration.

“We are grateful for the opportunity to bring together decades of diligent investigative reports from many parts of the world, including our collaborators in Spain and the United States,” added Dr. Schwartz, “and we look forward to facilitate an additional global bench – bedside collaborations as the Phase 2 clinical program progresses.

The phase 2 trial (NCT04069533, NCT04248439) is designed to assess the therapeutic efficacy of RP-L102 in patients with FA. Patients are treated with RP-L102 “Process B” which incorporates a modified stem cell enrichment process, transduction activators, as well as a commercial grade vector and final drug product. Improvement of mitomycin-C resistance (MMC) in cells forming bone marrow colonies (progenitors) is the primary endpoint and may also serve as a surrogate endpoint for accelerated approval.

About Fanconi anemia

Fanconi anemia (FA) is a rare pediatric disease characterized by bone marrow failure, deformities and predisposition to cancer. The main cause of death in patients with FA is bone marrow failure, which usually occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft versus host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, primarily squamous cell carcinomas of the head and neck area. About 60 to 70% of patients with FA have FANC-A genetic mutation, which codes for a protein essential for DNA repair. Mutation in the FANC-A leads to chromosome disruption and increased sensitivity to oxidative and environmental stress. Chromosomal fragility induced by DNA alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard for the diagnosis of AF. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene which can lead to stabilization or correction of the blood count of an AF patient in the absence of any treatment administered. Somatic mosaicism, often referred to as ‘nature’s gene therapy’, provides a strong rationale for the development of AF gene therapy due to the selective growth advantage of corrected hematopoietic stem cells over FA cells.1.

1Soulier, J., et al. (2005) Detection of somatic mosaicism and classification of patients with Fanconi anemia by analysis of the FA / BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) advances an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare disorders in childhood. The company’s platform-independent approach enables it to design the best therapy for each indication, creating potentially transformative options for patients with rare genetic diseases. Rocket’s clinical programs using lentiviral vector gene therapy (LVV) are for the treatment of Fanconi anemia (FA), a difficult-to-treat genetic disease that results in bone marrow failure and potentially cancer, leukocyte-I adhesion deficiency (LAD-I), a serious pediatric genetic disorder that causes recurrent and life-threatening infections that are often fatal, and pyruvate kinase (PKD) deficiency, a rare, monogenic red blood cell disease resulting in increased destruction of red blood cells and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV) gene therapy is for Danon’s disease, a devastating pediatric heart failure. Rocket’s preclinical pipeline program is for infantile malignant osteopetrosis (IMO), a disease derived from the bone marrow. For more information on Rocket, please visit

Rocket Caution Regarding Forward-Looking Statements

Various statements in this release regarding Rocket’s future expectations, plans and prospects, including, without limitation, Rocket’s expectations regarding the safety, efficacy 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’s disease, as well as the safety, efficacy and timing of preclinical studies and related clinical trials, may constitute forward-looking statements for safety purposes contain provisions under the Private Securities Litigation Reform Act of 1995 and other federal laws on securities and are subject to substantial risks, uncertainties and assumptions. You should not rely on these forward-looking statements, which often include words such as “believe”, “expect”, “anticipate”, “intend”, “plan”, “will”, ” estimate “,” search, “” will “,” may “,” suggest “or similar terms, variations of these terms or the negative of these terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such results. Actual results may differ materially from those indicated by these forward-looking statements due to various important factors, including, without limitation, Rocket’s ability to successfully demonstrate the efficacy and safety of these products and studies. preclinical and clinical trials, its gene therapy programs, the preclinical and clinical results of its product candidates, which may not support further development and market approval, the potential benefits of Rocket’s product candidates, the actions of regulations, which may affect the initiation, timing and progress of preclinical studies and clinical trials of its product candidates, the ability of Rocket and its licensors to obtain, maintain and protect its intellectual property and their respective intellectual property, the timing, cost or other aspects of a possible commercial launch of the p Rocket’s candidate products, Rocket’s ability to manage operating expenses, Rocket’s ability to secure additional funding to support its operations and to establish and maintain strategic business alliances and new business initiatives, Rocket’s dependence vis-à-vis third parties for the development, manufacture, marketing, sale and distribution of product candidates, the outcome of litigation and unforeseen expenses, as well as those risks discussed in more detail in the section entitled “Factors” risk ”in Rocket’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed November 8, 2019 with the SEC. Therefore, you should not place undue reliance on these forward-looking statements. All of these statements speak only as of the date of their publication, and Rocket assumes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. .

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