Investigator Initiated Trial (IIT) Toolkit – A Guide to Effective Shipping Contracts to Advance a Study, onCore is a central repository of study information for clinical trials (including budgets, patient follow-up, case reports) The IIT tool is intended to provide instructions on the information needed to launch the IIT to facilitate effective contracting and move the study forward as quickly as possible. On each page below, information on the elements of the study, the questions that the contractors` office must answer, or the checklist items for the IP are displayed. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is an emerging clinical biotechnology company focused on developing first-rate treatments for gene therapy for rare and devastating diseases. Rockets` multiplatform development approach uses lentiviral vector vector vectors (LVVs) and adeno-associated viral vectors (AAV) to gene therapy platforms. Rocket`s flagship clinical program is a gene therapy based on LVV for the treatment of Fanconi anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure and potentially carcinogenic. Rockets` additional pipeline programs for bone marrow-derived diseases are for pyruvate kinase deficiency (PKD), leukodist I adherence deficiency (LAD-I) and malignant infant osteoopopetosis (IMO). Rocket is also developing a gene therapy program based on AAV for an indication of devastating pediatric heart failure, Danon`s disease. For more information about Rocket, visit www.rocketpharma.com.
Some of the statements contained in this press release may be forward-looking statements that involve a number of risks and uncertainties. For these statements, we seek the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Among the factors that could lead to significant differences between our actual results is the impact of the rupture on the relationships related to the AIM2CERV study; The success and timing of our clinical trials, including consideration of subjects; our ability to avoid clinical catches and resolve them quickly; our ability to obtain and maintain administrative authorization and/or reimbursement of our products for marketing; Our ability to obtain proper labelling of our products under each administrative authorization; Our product development and marketing projects; Successful development and implementation of our sales and marketing campaigns; The size and growth of potential markets for our products and our ability to serve those markets; our ability to compete successfully in potential markets for our product candidates when they are marketed; regulatory developments in the United States and other countries; The level and degree of acceptance of one of our products new products, candidates or uses for existing products or technologies introduced or announced by our competitors, and the date of these introductions or announcements; market conditions in the pharmaceutical and biotechnology sectors; our money available, including to support current and planned clinical activities; Accurate estimates of spending, future revenues, capital requirements and additional financing requirements; Our ability to secure additional funding our ability to preserve and maintain intellectual property protection for our product candidates; the success and timing of our preclinical studies, including ind studies; The date of our ind submissions; our ability to obtain FDA approval for study changes; Timing data surveys The ability of our candidates to work successfully in clinical trials; our ability to initiate, register and conduct pilot projects and clinical studies; our ability to maintain our existing cooperation; our manufacturing capacity and the performance of third-party producers; the results of our clinical research organizations, clinical trial sponsors and the researcher for clinical trials; our ability to implement our strategy