Fidest – Agenzia giornalistica/press agency

Quotidiano di informazione – Anno 32 n° 255

Rocket Pharmaceuticals Announces Presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

Posted by fidest press agency su giovedì, 3 maggio 2018

Chicago. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announced eight oral and poster presentations at the upcoming American Society of Gene & Cell Therapy (ASGCT) 2018 Annual Meeting being held May 16 -19, 2018 in Chicago, IL.“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,” 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. ”


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