Rocket Pharmaceuticals Announces Upcoming Clinical Data Presentations at the 24th Annual Meeting of the American Society of Gene and Cell Therapy

CRANBURY, NJ – (BUSINESS WIRE) – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for childhood rare diseases, today announces clinical data presentations at the upcoming 24thAmerican Annual Meeting of the Society of Gene and Cell Therapy (ASGCT) taking place May 11-14, 2021. Researchers will review new data from Rocket’s Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Fanconi Anemia (FA) gene therapy programs in oral and poster presentations.

Details for oral presentations are as follows:

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficit-I (LAD-I): Interim Results

Session: Genetic blood and immune disorders

Presenter: Donald Kohn, MD, 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

Date: Tuesday 11 May 2021

Time: 6:15 am – 6:30 pm EDT

Location: room 7

Abstract number: 39

Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Worldwide Phase 1 Trial for Adult and Pediatric Patients

Session: Gene Therapies for Hemoglobinopathies

Presenter: José Luis López Lorenzo, MD, Fundación Jiménez Díaz University Hospital, Madrid, Spain

Date: Wednesday May 12, 2021

Time: 6:45 PM – 7:00 PM EDT

Location: room 7

Abstract number: 83

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCs

Session: International focus on stem cell gene therapy

Presenter: Juan A. Bueren, Ph.D., Head of the Hematopoietic Innovative Therapies Division at the Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) in Spain / CIBER rare diseases / IIS-Fundación Jiménez Díaz

Date: Thursday, May 13, 2021

Time: 10:00 AM – 10:45 AM EDT

Location: room 7

Abstract number: 36

Certain results of Dr. Bueren will also be highlighted by Paula Rio, Ph.D. Details for this invited presentation are as follows:

Title: Gene Therapy in Fanconi Anemia: Current Strategies to Enable the Correction of HSCs

Session: International focus on stem cell gene therapy

Presenter: Paula Río, Ph.D., Senior Researcher, Hematopoietic Innovative Therapies Division at CIEMAT in Spain / CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz

Date: Thursday, May 13, 2021

Time: 10:00 AM-11:45 AM EDT

Details for poster presentation are as follows:

Title: Gene Therapy for Fanconi Anemia [Group A]: Preliminary results from ongoing RP-L102 clinical studies

Session: Hematological and Immunological Diseases

Presenter: Agnieszka Czechowicz, MD, Ph.D., Assistant Professor of Pediatrics, Stem Cell Transplantation Department, Stanford University School of Medicine

Date: Tuesday 11 May 2021

Time: 8:00 am-10:00pm EDT

Location: Digital Gallery

Abstract number: 697

Abstracts for the presentations can be found online at:

About leukocyte adhesion deficiency-I

Severe leucocyte adhesion deficit-I (LAD-I) is a rare autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to fight infection. As a result, children with severe LAD-I are often affected right after birth. During childhood, they suffer from recurring life-threatening bacterial and fungal infections that respond poorly to antibiotics and require regular hospitalization. Children who survive childhood experience recurring serious infections, including pneumonia, gum ulcers, necrotic skin ulcers and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% before the age of 2 years and survival after the age of 5 years is uncommon. There is a great unmet medical need for patients with severe LAD-I.

Rocket’s LAD-I study is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are the sole responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disease that results from a mutation in the PKLR gene encoding the pyruvate kinase enzyme, a key component of the glycolytic pathway of red blood cells. Mutations in the PKLR gene result in increased red blood cell destruction and the condition ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 3,000 to 8,000 patients in the United States and the European Union. Children are the most common and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was licensed by the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica and Red de Diseases Rare (CIBERER) and Instituto de Investigacion Sanitaria Fundacion Jimenez Diaz (IIS-FJD).

About Fanconi Anemia

Fanconi anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and predisposition to cancer. The leading cause of death in patients with FA is bone marrow failure, which usually occurs during the first decade of life. Allogeneic haematopoietic stem cell transplantation (HSCT), when available, corrects the haematological 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, mainly squamous cell carcinomas of the head and neck region. About 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased susceptibility to oxidative and environmental stress. Increased sensitivity to DNA alkylating agents such as mitomycin-C (MMC) or Diepoxybutane (DEB) is a ‘gold standard’ test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can stabilize or correct the blood picture of an FA patient in the absence of any administered therapy. Somatic mosaicism, often referred to as ‘natural gene therapy’, provides a strong rationale for the development of FA gene therapy due to the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is working on an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood diseases. The Company’s platform agnostic approach allows it to design the best therapy for each indication, creating potentially transformative options for patients suffering from 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 possibly cancer, Leukocyte Adhesion Deficiency-I (LAD-I) , a serious pediatric genetic condition causing recurrent and life-threatening infections that are often fatal, pyruvate kinase deficiency (PKD), a rare monogenic red blood cell disorder that leads to increased red blood cell destruction and mild to life-threatening anemia, and infantile malignant osteopetrosis (IMO), a bone marrow-derived disease. Rocket’s first clinical program to use adeno-associated virus (AAV) -based gene therapy is for Danon’s disease, a devastating condition in children with heart failure. For more information about Rocket, visit

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release regarding Rocket’s future expectations, plans and prospects, including but not limited to Rocket’s expectations regarding the 2021 guidelines in light of COVID-19, candidate’s safety, effectiveness and timing products that Rocket can develop to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and their safety, effectiveness and timing of related preclinical and clinical investigations 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 rely 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 on such terms or the negative of those terms. While Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot provide such results. Actual results could differ materially from those indicated in these forward-looking statements as a result of several important factors, including, but not limited to, Rocket’s ability to monitor the impact of COVID-19 on its business activities and measures. to ensure the safety of patients, families and employees, the interest of patients and families in participating in each of Rocket’s ongoing studies, 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 offering for our ongoing and anticipated trials, regulatory actions, those of May affect the initiation, timing and progress of preclinical and clinical studies of its product candidates, Rocket’s reliance on third parties for development, manufacturing, marketing, sales and distribution of product candidates, the outcome of litigation and unexpected expenses, as well as these risks discussed more fully in the “Risk Factors” section of Rocket’s Annual Report on Form 10-K for the year ended December 31, 2020, filed March 1, 2021 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date they are made and Rocket assumes no obligation to update or publicly revise forward-looking statements, whether as a result of new information, future events or otherwise.

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