Business
Rocket Pharmaceuticals Reports First Quarter 2023 Financial Results and Highlights Recent Progress
Continued to advance leading pipeline of AAV cardiology and LV hematology assets; all 2023 milestones remain on track Updates on all four clinical programs
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nContinued to advance leading pipeline of AAV cardiology and LV hematology assets; all 2023 milestones remain on track\n\n\nUpdates on all four clinical programs and PKP2-ACM preclinical program to be presented at the 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)\n\n\nMark White, MB.ChB, named Chief Medical Officer following more than 25 years at AstraZeneca; Jonathan Schwartz, M.D., appointed Chief Gene Therapy Officer\n\n\nBecame founding consortium member of Rady Children’s Institute for Genetic Medicine’s BeginNGSTM newborn screening program to increase speed of diagnosis for genetic diseases\n\n\nCash, cash equivalents and investments of approximately $360M; expected operational runway into the first half of 2025\n\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending March 31, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.\n\n\n“I am very pleased with our first quarter progress, as we continued to advance our industry leading pipeline of now six programs across both AAV and LV platforms addressing life threatening cardiac and hematologic rare diseases, including two LV programs nearing fillings and four programs either demonstrating robust clinical results or strong preclinical proof of concept, “said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “Corporate and program milestones remain on track, including the planned initiation of the Phase 2 pivotal trial in Danon Disease this second quarter and regulatory filings for LAD-I and Fanconi Anemia this second quarter and fourth quarter, respectively.”\n\n\nDr. Shah continued, “In addition, we look forward to presenting data across all four of our clinical programs at the upcoming ASGCT meeting this month, as well as our recently announced preclinical program for PKP2-ACM, one of the most prevalent and devastating forms of heart disease impacting nearly 50,000 adults and children in the U.S. and EU. Further, I am proud to announce that we became a Founding Member of BeginNGSTM, a national program that advocates for and facilitates newbo...