On April 30, 2018 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) ("Rocket"), a leading U.S.-based multi-platform gene therapy company, reported eight oral and poster presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2018 Annual Meeting being held May 16 -19, 2018 in Chicago, IL (Press release, Rocket Pharmaceuticals, APR 30, 2018, View Source;p=RssLanding&cat=news&id=2345662 [SID1234526550]).

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"Rocket has a robust pipeline of five innovative gene therapy programs across lentiviral vector (LVV) and adeno-associated viral vector (AAV) platforms, and we are pleased that our programs to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) are featured at ASGCT (Free ASGCT Whitepaper)," said Gaurav Shah, Chief Executive Officer and President of Rocket. "Our most advanced program to treat FA is in a Phase 1/2 trial for which updated data will be presented during the Presidential Symposium. In children with FA, FANCA gene mutations enable chromosomal abnormalities that frequently lead to bone marrow failure, acute myeloid leukemia and death, with relatively toxic bone marrow transplant regimens as a principal therapy. We are hopeful that our FANCA-focused LVV gene therapy has the potential to enable broadly-applicable prevention of bone marrow failure, leading to safer and transformative outcomes."

Jonathan Schwartz, M.D., Chief Medical Officer of Rocket, added, "Our second most advanced program is our LVV gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I), which is expected to enter the clinic early next year. We are focused on the most severe form of LAD-I in which approximately 61-75% of children do not survive past the age of 2 due to life threatening infections. Based on our research, a modest correction of the defective gene encoding for the CD18 receptor can enable survival to adulthood. Both FA and LAD-I are examples of Rocket’s focus on exploring definitive therapies for patients with clearly defined monogenic diseases by targeting the underlying genetic deficit; these are disorders where a modest number of gene-corrected stem cells can make a meaningful difference for patients otherwise facing very limited treatment options. "

Oral Presentations:

Title: Engraftment and Phenotypic Correction of Hematopoietic Stem Cells in Non-Conditioned Fanconi Anemia Patients Treated with Ex Vivo Gene Therapy
Session: 330 Presidential Symposium & Presentation of the Top Abstracts
Presenter: Juan Bueren, Ph.D. Head of the Hematopoietic Innovative Therapies Division, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT) / CIBER-Rare Diseases / IIS-Fundación Jiménez Díaz, and principal investigator of the RP-L102 trial.
Date: Friday, May 18, 2018
Session Time: 1:15 p.m. – 3:15 p.m. Central Time
Presentation Time: 2:45 p.m. – 3:00 p.m. Central Time
Location: International Ballroom North & South

Title: Immunotoxin-Based Conditioning Facilitates Autologous Hematopoietic Stem Cell Engraftment and Multi-Lineage Development in a Fanconi Anemia Mouse Model
Session: 115 Hematopoietic Cell Therapies
Presenter: Meera Srikanthan, M.D., Seattle Children’s Hospital; Fred Hutchinson Cancer Research Center
Date: Wednesday, May 16, 2018
Session Time: 10:30 a.m. – 12:00 p.m. Central Time
Presentation Time: 10:45 a.m. – 11:00 a.m. Central Time
Location: Salon A-5

Title: Gene Editing in Fanconi Anemia Hematopoietic Stem and Progenitor Cells
Session: 302 Advances in Genome Editing in HSCs – Organized by the Hematologic and Immunologic Gene and Cell Therapy Committee
Presenter: Paula Rio, Ph.D., Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT)
Date: Friday, May 18, 2018
Session Time: 8:00 a.m. -10:00 a.m. Central Time
Presentation Time: 9:00 a.m. – 9:30 a.m. Central Time
Location: International Ballroom North

Poster Presentations:

Title: Improvements in the Transduction Conditions of Human Hematopoietic Progenitors with the CPcoRPKW-17 Therapeutic Lentiviral Vector to be Used in a Pyruvate Kinase Deficiency Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency I: A Closer Step to a Gene Therapy Clinical Trial
Session: Hematologic & Immunologic Diseases I
Date: Wednesday, May 16, 2018
Time: 5:30 p.m. – 7:30 p.m. Central Time
Location: Stevens Salon C, D

Title: Pairs of Guide RNAs Mediate Precise Deletions on the PKLR Gene via Non Homologous End Joining Generating a Human Hematopoietic Progenitor Model of Pyruvate Kinase Deficiency
Session: Hematologic & Immunologic Diseases II
Date: Thursday, May 17, 2018
Time: 5:15 p.m. – 7:15 p.m. Central Time
Location: Stevens Salon C, D

Title: Leukocyte Adhesion Deficiency-I: A Comprehensive Review of Published Cases
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D

Title: Towards the Gene Therapy Clinical Trial for Pyrivate Kinase Deficiency
Session: Hematologic & Immunologic Diseases III
Date: Friday, May 18, 2018
Time: 5:45 p.m. – 7:45 p.m. Central Time
Location: Stevens Salon C, D