Business
Rocket Pharmaceuticals Reports Full Year 2019 Financial Results and Operational Highlights
—Construction of R&D and cGMP Manufacturing Facility Underway in New Jersey— —Preliminary Clinical Data of RP-L102 in FA and RP-L201 in LAD-I Support
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Construction of R&D and cGMP Manufacturing Facility Underway in New Jersey—\n\n\n—Preliminary Clinical Data of RP-L102 in FA and RP-L201 in LAD-I Support Potential of Lentiviral Pipeline and “Process B” Manufacturing—\n\n\n—First Three Patients with Danon Disease in Low-Dose Phase 1 Cohort Treated with RP-A501—\n\n\n—Strong Balance Sheet with Cash Runway into 2022—\n\n NEW YORK--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial results for the year ended December 31, 2019, and provides an update on the Company's recent pipeline developments, as well as upcoming milestones.\n\n\n“2019 was a pivotal year for Rocket marked by multiple clinical and regulatory achievements across the pipeline,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “We now have four gene therapy programs in the clinic, have established proof of concept for our lenti pipeline utilizing our commercial-grade ‘Process B’ manufacturing in both FA and LAD-I and have treated the first three patients in our Phase 1 trial for Danon Disease.”\n\n\nDr. Shah continued, “We are excited to build upon the momentum by bringing our fifth program to the clinic, advancing FA and LAD-I with BLA/MAA filings commencing in the next two to three years and completing our R&D and manufacturing facility. In addition to reporting progress from our FA and LAD-I programs, we’re looking forward to presenting initial proof of concept data from PKD and Danon this year. With these anticipated milestones by year end, we are even closer to making our gene therapies available to patients and families with rare disease.”\n\n\nFull Year 2019 and Recent Pipeline Developments\n\n\n\nRocket expands footprint in Cranbury, New Jersey. Rocket’s Research & Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operations will be housed in a new 103,720 square foot facility, of which 50,000 square feet will be dedicated to adeno-associated virus (AAV) Current Good Manufacturing Practice (cGMP) manufacturing. The manufacturing facility will result in a one-time additional spend of $30 million in the first half of 2020. With construction underway, Rocket has secured adequate supply of cGMP AAV9 t...