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Rocket Pharmaceu (RCKT) is a pioneering biotechnology company developing gene therapies for rare pediatric diseases through innovative lentiviral-based approaches. This page provides investors and industry observers with a comprehensive repository of official company announcements, clinical development updates, and strategic partnership news.
Access timely updates on RCKT's research milestones, regulatory progress, and financial performance. Our curated collection includes press releases related to clinical trial outcomes, manufacturing advancements, and collaborations with leading medical institutions. Users will find verified information on therapeutic developments targeting high-need genetic disorders.
Key areas covered include novel treatment pipelines, FDA designations, intellectual property updates, and scientific presentations. Bookmark this page to stay informed about Rocket Pharmaceu's contributions to gene therapy innovation and its evolving position in the biopharmaceutical sector.
Rocket Pharmaceuticals (NASDAQ: RCKT) recently announced promising clinical updates from its ongoing gene therapy trials. The Phase 1 trial for RP-A501 in Danon Disease showed the treatment was well-tolerated in pediatric patients with no reported toxicities. In the Phase 2 trial for Fanconi Anemia, five out of nine patients exhibited sustained increased resistance to mitomycin-C, achieving the trial's primary endpoint. Additionally, treatments for Pyruvate Kinase Deficiency demonstrated normalized hemoglobin levels in patients 18 months post-infusion.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is set to present at the UBS Global Healthcare Conference on May 24 at 7:45 a.m. ET in New York. CEO Gaurav Shah will deliver the presentation, with a live audio webcast available on the company's website. Rocket focuses on developing genetic therapies for rare childhood disorders, including Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency. The company employs a platform-agnostic approach to offer innovative treatment options, addressing significant unmet needs in pediatric healthcare.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) reported a net loss of $43M, or $0.67 per share, for Q1 2022. The company holds $346.6M in cash, extending its operational runway into H1 2024. Key updates include the advancement of four clinical programs targeting Danon Disease, Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency, with data readouts expected throughout 2022. Significant leadership appointments were made, including Fady Malik, M.D., to the Board and Carlos Martin as Chief Commercial Officer. The company also anticipates AAV cGMP manufacturing readiness in Q2 2022.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is set to present at the Bank of America Securities Healthcare Conference on May 11 at 2:20 p.m. ET. The company specializes in developing genetic therapies for rare childhood disorders, including Fanconi Anemia and Danon Disease. Rocket utilizes a platform-agnostic approach, focusing on tailored therapy solutions. Given the ongoing impact of COVID-19, the company emphasizes the safety and effectiveness of its product candidates, while also addressing potential delays in clinical trials.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announces key upcoming presentations: President Kinnari Patel will present at the Guggenheim 3rd Annual Genomic Medicines and Rare Disease Conference on April 1 at 8:00 a.m. ET, and CEO Gaurav Shah will speak at the 21st Annual Needham Virtual Healthcare Conference on April 11 at 10:15 a.m. ET. The live webcasts will be accessible on the company's website, with replays available post-event. Rocket focuses on genetic therapies for rare childhood disorders, including Fanconi Anemia and Danon Disease.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has appointed Fady Malik, M.D., Ph.D., to its Board of Directors. Dr. Malik, with nearly 25 years of experience as a cardiovascular physician-scientist and biopharmaceutical executive, will serve as an independent non-executive director. CEO Gaurav Shah emphasized Dr. Malik's expertise in R&D and leadership in bringing medicines from research to clinical application, particularly in cardiology. Rocket is advancing its clinical pipeline for rare childhood disorders, including Danon Disease, and aims for agency alignment on its Phase 2 clinical study.
Rocket Pharmaceuticals (NASDAQ: RCKT) has appointed Jessie Yeung as Vice President of Investor Relations and Corporate Finance. With over 15 years of experience, including a previous role at Legend Biotech (NASDAQ: LEGN), Yeung will lead investor relations and capital market strategies. CEO Gaurav Shah emphasized Yeung's expertise will enhance communication with investors as the company approaches significant milestones in gene therapy clinical programs.
Rocket Pharmaceuticals, focusing on genetic therapies for rare childhood disorders, announced positive Phase 1 results for Danon Disease, showing clinical benefits in four adults. The data indicated sustained improvement across clinical metrics. In pivotal Phase 2 trials for LAD-I, nine patients exhibited successful engraftment, with CD18 expression ranging from 26% to 87%. Six out of eight patients in the Fanconi Anemia trial demonstrated engraftment, with data expected soon. The company holds a strong cash position of $388.7 million, ensuring operational funding into the second half of 2023.
Rocket Pharmaceuticals (NASDAQ: RCKT), a clinical-stage company focused on genetic therapies for rare childhood disorders, has announced that CEO Gaurav Shah will participate in a virtual fireside chat at the 11th Annual SVB Leerink Global Healthcare Conference on February 16 at 10 a.m. ET. Investors can access a live audio webcast of the presentation on the company's website. Rocket's pipeline includes therapies for conditions such as Fanconi Anemia and Danon Disease, utilizing both lentiviral and adeno-associated viral vector approaches.
Rocket Pharmaceuticals (NASDAQ: RCKT) announced key milestones for 2022 during the J.P. Morgan Healthcare Conference. Pivotal data from the LAD-I and Fanconi Anemia trials will be reported in Q2 and Q3, respectively. AAV GMP manufacturing in Cranbury, NJ is expected to begin in Q2, and data from Danon Disease pediatric cohorts is anticipated in Q3. Additionally, the initiation of pivotal Phase 2 trials for Danon Disease and PKD is planned for Q4, reflecting significant progress in advancing their genetic therapies for rare childhood disorders.