Osteopetrosis, Autosomal Recessive 1

Nov. 15, 2021 12:00 UTC Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease —Results demonstrate sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up— — NYHA class improvement (from II to I) in all three patients (two low-dose, one high-dose) with closely monitored immunosuppressive regimen— —Decreased cardiac wall thickness with improved or stabilized ejection fraction on echocardiogram in all three patients with closely monitored immunosuppressive regimen— — Sustained improvement or stabilization in BNP and 6-minute walk test across four patients— —Sustained cardiac LAMP2B expression greater than 50% by immunohistochemistry and improved cardiac tissue architecture in all three patients with closely monitored immunosuppressive regimen— —RP-A501 generally well tolerated at low-dose with manageable safety profile— —Webcast to be held at 8:30 a.m. ET today— CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces updated data from the Phase 1 clinical trial evaluating a single intravenous infusion of RP-A501, the Company’s investigational gene therapy, for the treatment of Danon Disease. This update, which includes interim safety and efficacy data from patients in the low-dose (6.7e13 vg/kg; n=3) and high-dose (1.1e14 vg/kg; n=2) adult and adolescent cohorts, demonstrates RP-A501 was generally well tolerated at the low-dose and conferred sustained clinical benefit. “We are excited to announce positive data from our RP-A501 trial for Danon Disease showing clinical, functional and biomarker improvements at one year or beyond and potential early separation from the natural course of disease in adult and adolescent patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years. In this setting, even stabilization is highly beneficial, and clinical improvements as seen in our trial could be transformative and represent a step forward for the treatment of monogenic heart failure and gene therapy for cardiac diseases. We have initiated treatment in the pediatric cohort at the low-dose and anticipate moving as rapidly as possible toward Phase 2.” Dr. Barry Greenberg, Director of the Advanced Heart Failure Treatment Program at UC San Diego Health, Professor of Medicine at UC San Diego School of Medicine, and the principal investigator of the RP-A501 clinical study, is presenting a subset of the data at the American Heart Association (AHA) Scientific Sessions 2021. Dr. Greenberg added, “I am very encouraged by the ability to safely administer RP-A501 and show robust gene expression in the hearts of these young male patients with this devasting monogenic disorder. The clinical data to date also demonstrate encouraging results including clinical stabilization of this rapidly progressive disease.” Safety Results RP-A501 was generally well tolerated at the 6.7e13 vg/kg dose level. All observed adverse effects were reversible with no lasting sequelae. Early transaminase and creatinine kinase elevations returned to baseline or decreased. As previously disclosed, RP-A501 r-AAV dose-dependent toxicity was seen in one of the two patients treated at the 1.1e14 vg/kg dose level. The affected patient, who received the largest total dose, developed thrombotic microangiopathy (TMA) that fully resolved with supportive treatment including transient hemodialysis. Across both dose levels, the most common serious adverse event (SAE) observed was steroid-induced myopathy in three patients (two low-dose and one high-dose) which resolved subsequent to corticosteroid discontinuation. Based on the observed safety and efficacy to-date, the Company will focus on the low-dose cohort moving forward and will no longer administer the high-dose to study patients. An updated protocol developed in collaboration with the FDA has been implemented to mitigate development of TMA and other treatment-related adverse events. Clinical and Biomarker Results for Patients With at Least 12 Months Follow-Up NYHA class: An improvement in NYHA class (from II to I) was observed in three patients (two low-dose and one high-dose) who had closely monitored immunosuppression with follow-up greater than one year and stabilization was observed in one low-dose patient without a closely monitored immunosuppressive regimen. BNP: A substantial improvement in B-type natriuretic peptide (BNP), a key marker of heart failure, was observed in all three low-dose patients and one high-dose patient. Among the three low-dose patients, BNP decreased from a pretreatment baseline by 57% at 24 months, 79% at 18 months, and 75% at 15 months, respectively. In the high-dose patient, BNP decreased from a pretreatment baseline by 67% at 12 months. LV wall thickness and ejection fraction: In patients with closely monitored immunosuppression (two low-dose and one high-dose) left ventricular (LV) posterior wall thickness improved (average 23% decrease compared to pretreatment baseline) and ejection fraction improved or stabilized (average 20% increase compared to pretreatment baseline) at 12 to 18 months on echocardiography. Severe and progressive wall thickening is a hallmark of the hypertrophic cardiomyopathy of Danon Disease and is a major contributor to early mortality in male patients. Cardiac output and diastolic dysfunction: Cardiac output remained normal for all patients with improved or stable left heart filling pressures as measured by cardiac catheterization. 6MWT: Three low-dose patients and one high-dose patient demonstrated improvements in the 6-minute walk test (6MWT). One low-dose patient improved from a pretreatment baseline of 443 meters (m) to 467 m at 24 months. The second low-dose patient improved from a pretreatment baseline of 405 m to 410 m at 18 months. The third low-dose patient improved from a pretreatment baseline of 427 m to 435 m at 15 months. The high-dose patient improved from a pretreatment baseline of 436 m to 492 m at 12 months. Gene expression: Evidence of sustained cardiac LAMP2B gene expression by immunohistochemistry and Western blot with qualitative improvement of vacuoles and cardiac tissue architecture on electron microscopy was observed at both dose levels. Sustained cardiac LAMP2B gene expression by immunohistochemistry was observed in all three patients with a closely monitored immunosuppressive regimen. Specifically, LAMP2B gene expression by immunohistochemistry in the low-dose (6.7e13 vg/kg) was 68% in one patient at Month 12 and 92% in another patient at Month 9. In one patient who received the high-dose (1.1e14 vg/kg), LAMP2B gene expression by immunohistochemistry was 100% at Month 12. Explanted Heart As previously disclosed, one patient in the high-dose cohort underwent a heart transplant at Month 5. This patient had advanced disease including diminished LV ejection fraction (35%) on echocardiogram and markedly elevated LV filling pressure prior to treatment. His clinical course was characteristic of Danon Disease progression. Analysis of the explanted heart revealed significant fibrosis consistent with advanced Danon Disease. Myocardial tissue from the explanted heart at 5 months post-treatment displayed 100% LAMP2B protein expression by immunohistochemistry throughout non-fibrotic cardiac regions including the ventricles and other essential targeted areas. Webcast Details Rocket will host a conference call and webcast today at 8:30 a.m. ET to discuss the RP-A501 data in Danon Disease. Investors may access the conference call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550 from outside the United States. Please refer to conference ID number 50255994. A live webcast of the call can be accessed under “Events” in the Investors section of the Company’s website at . The webcast replay will be available on the Rocket website following the completion of the call. About RP-A501 RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B). RP-A501 is currently being evaluated in a Phase 1 clinical trial, from which preliminary data of the low-dose cohort showed it was generally well tolerated and provided evidence of improved cardiac function in patients. About Danon Disease Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and Europe. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon Disease. 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-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 . 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 the potential for RP-A501 to treat Danon Disease, Rocket’s expectations regarding its guidance for 2021 in light of COVID-19, the safety and effectiveness of RP-A501, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, Rocket’s plans for the advancement of its Danon Disease program following the lifting of the FDA’s clinical hold and additional data announcement 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 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 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.

CRANBURY, N.J.--(BUSINESS WIRE)-- 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 24thAmerican 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: 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-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 . 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 2021 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 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 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.

April 14, 2021 11:00 UTC CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces positive interim data from the Company’s Phase 1/2 clinical trial studying RP-L201, its lentiviral-based gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I). Severe LAD-I is a rare pediatric disease that prevents patients from adequately combating infections. LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and are ultimately fatal. These results were presented in a virtual poster session at the Clinical Immunology Society (CIS) 2021 Annual Meeting. “Today’s positive updates on our LAD-I program add to the growing body of encouraging evidence that RP-L201 may provide durable clinical benefit for patients with severe LAD-I who face recurrent, life-threatening infections from birth,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “We are very pleased to report that a second patient is nearing survival at one-year post-treatment, the primary outcome measure for the Phase 2 portion of the study. In all patients treated, CD18 expression has substantially exceeded the 4-10% threshold associated with survival into adulthood, with consistent peripheral blood vector copy number levels. Improved disease-related skin lesions, absence of new infections post-treatment, and no further requirements for prophylactic anti-infectives were also observed in both Phase 1 patients with prolonged follow-up. Initial evidence of engraftment and phenotypic correction was observed in two additional patients with shorter follow-up. These updates move us one step closer towards BLA/MAA filings in the US and Europe and eventual commercialization of a potentially curative option for the children facing this truly devastating disease. We look forward to providing more comprehensive Phase 2 results in the second half of 2021.” The data reported in the poster presentation are from four pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. The patients were treated with RP-L201, Rocket’s ex-vivo lentiviral gene therapy candidate. Data were reported as of the cutoff date of February 2021. Patient 1001 was 9 years-of-age at enrollment and had been followed for 18-months after RP-L201 therapy. Patient 1004 was 3 years-of-age at enrollment and had been followed for 9-months. Patients 2006 and 2005 were 7 months- and 2 years-of-age at enrollment and had been followed for 3-months. Key highlights from the poster presentation include: RP-L201 was well tolerated, no safety issues reported with infusion or treatment All patients achieved hematopoietic reconstitution within 5-weeks Neutrophil CD18-expression and peripheral blood vector copy numbers (VCN) were assessed post-treatment to evaluate engraftment and phenotypic correction: 18-months post-treatment, Patient 1001 demonstrated durable CD18 expression of ~40% and resolution of skin lesions with no new lesions reported; 12-months post-treatment, peripheral blood VCN levels were 1.2 9-months post-treatment, Patient 1004 demonstrated CD18 expression of ~28%; 6-months post-treatment, peripheral blood VCN levels were 0.75 with kinetics consistent with those of the first patient 3-months post-treatment, Patient 2006 demonstrated CD18 expression of ~70%; 1.5-months post-treatment, peripheral blood VCN kinetics were consistent with those of the first two patients 3-months post-treatment, Patient 2005 demonstrated CD18 expression of ~51%; 1.5-months post-treatment, peripheral blood VCN kinetics were consistent with those of the first two patients To access the poster, please visit: 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 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. 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-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 . 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 2021 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 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 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. View source version on businesswire.com: