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

Rhea-AI Summary

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announces clinical data presentations at the 24th American Society of Gene and Cell Therapy Annual Meeting, May 11-14, 2021. Key presentations include interim results from a Phase 1/2 study on gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I) and updates on Pyruvate Kinase Deficiency (PKD) and Fanconi Anemia (FA). With high unmet medical needs, Rocket aims to address severe pediatric diseases with innovative gene therapies, as detailed in oral and poster sessions throughout the event.

Positive

• Presenting interim results from a Phase 1/2 study on LAD-I gene therapy at a prominent event.
• Updating results from global Phase 1 study for PKD, showcasing international collaboration.
• Highlighting current strategies and ongoing clinical trials for FA gene therapy with expert presentations.

Negative

• None.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data presentations at the upcoming 24^thAmerican Society of Gene and Cell Therapy (ASGCT) Annual Meeting taking place May 11-14, 2021. Investigators 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 Deficiency-I (LAD-I): Interim Results
Session: Genetic Blood and Immune Disorders
Presenter: Donald 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
Date: Tuesday May 11, 2021
Time: 6:15-6:30 p.m. EDT
Location: Room 7
Abstract number: 39

Title: Lentiviral Mediated Gene Therapy for Pyruvate Kinase Deficiency: Updated Results of a Global Phase 1 Study for Adult and Pediatric Patients
Session: Gene Therapies for Hemoglobinopathies
Presenter: José Luis López Lorenzo, M.D., Hospital Universitario Fundación Jiménez Díaz, Madrid, Spain
Date: Wednesday May 12, 2021
Time: 6:45-7:00 p.m. 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: 10:00-10:45 a.m. EDT
Location: Room 7
Abstract number: 36

Select results from Dr. Bueren’s presentation 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: 10:00-11:45 a.m. EDT

Details for poster presentation are as follows:

Title: Gene Therapy for Fanconi Anemia [Group A]: Preliminary Results of Ongoing RP-L102 Clinical Trials
Session: Hematologic and Immunologic Diseases
Presenter: Agnieszka Czechowicz, M.D., Ph.D., Assistant Professor of Pediatrics, Division of Stem Cell Transplantation, Stanford University School of Medicine
Date: Tuesday, May 11, 2021
Time: 8:00-10:00 a.m. EDT
Location: Digital Gallery
Abstract number: 697

Abstracts for the presentations can be found online at: https://annualmeeting.asgct.org/

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rocket’s LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). 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 Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder 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 commonly 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 in-licensed from the Centro de Investigaciones Energeticas, Medioambientales y Tecnologicas (CIEMAT), Centro de Investigacion Biomedica en Red de Enfermedades Raras (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 cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically 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, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutation in the FANCA gene leads to chromosomal breakage and increased sensitivity 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 lead to stabilization or correction of a FA patient’s blood counts 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 because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) 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-threatenin

FAQ

What is the focus of Rocket Pharmaceuticals' data presentations at the ASGCT Annual Meeting?

Rocket Pharmaceuticals is presenting data on gene therapies for severe childhood disorders, specifically LAD-I, PKD, and FA.

When will the oral presentations regarding LAD-I take place?

The oral presentation on LAD-I is scheduled for May 11, 2021, from 6:15-6:30 p.m. EDT.

What are the implications of Rocket's gene therapy for Fanconi Anemia?

Rocket's research aims to address bone marrow failure and cancer risks in children with Fanconi Anemia through innovative gene therapy.

How many patients are estimated to have Pyruvate Kinase Deficiency in the U.S. and EU?

The estimated prevalence of Pyruvate Kinase Deficiency is between 3,000 to 8,000 patients.

What type of gene therapy is Rocket Pharmaceuticals developing for LAD-I?

Rocket is developing a lentiviral-mediated ex-vivo gene therapy for patients with severe LAD-I.