Rocket Pharmaceuticals, Inc. - RCKT STOCK NEWS

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has appointed Isabel Carmona as Chief Human Resources Officer and Senior Vice President. With over 25 years of experience, particularly in life sciences, Carmona will oversee all HR functions and contribute to Rocket's growth strategy. CEO Gaurav Shah emphasized her expertise in talent management as crucial for the company's expansion. Carmona previously served as CHRO at Ichnos Sciences, enhancing her capabilities in developing organizational frameworks. The leadership team, noted for extensive drug development experience, aims to advance Rocket's innovative gene therapies for rare diseases.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced a securities purchase agreement with RTW Investments, LP for 812,516 shares of common stock at $32.48 per share, raising approximately $26.4 million. The private placement is set to close on or about August 31, 2021. Proceeds will support ongoing research and development efforts. The shares will not be registered under the Securities Act, with plans to file a resale registration statement with the SEC. Rocket aims to advance its pipeline of genetic therapies for rare childhood disorders.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has announced that the FDA has lifted the clinical hold on its Phase 1 trial for RP-A501, aimed at treating Danon Disease. This decision follows the company’s compliance with the FDA's revised protocol for patient selection and safety management. Patient enrollment will resume with dosing expected to commence in Q3 2023. The trial is designed to assess the safety and efficacy of RP-A501, which is a potential gene therapy for this rare condition affecting cardiac function and is currently without specific treatment options.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) reports financial results for Q2 2021, highlighting key updates on its genetic therapies for rare childhood disorders. As of June 30, 2021, cash reserves stand at $426.8 million, projected to fund operations until H2 2023. R&D expenses rose to $24.8 million from $16.7 million year-over-year, contributing to a net loss of $34.5 million, or $0.55 per share. Significant clinical progress includes favorable results in Danon Disease and promising data from Fanconi Anemia trials, while enrollment in the IMO trial is temporarily paused following a patient's passing.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) will host a conference call and live audio webcast on Aug. 9, 2021, at 4:30 p.m. ET to discuss its second quarter 2021 financial results and recent business highlights. Investors can join by dialing (866) 939-3921 in the U.S. or +1 (678) 302-3550 internationally, using conference ID 50210581. A replay will be accessible on Rocket's website afterwards. Rocket is focused on developing genetic therapies for rare childhood disorders, including Fanconi Anemia and Danon disease. For more information, visit rocketpharma.com.

Rocket Pharmaceuticals (NASDAQ: RCKT) announced positive clinical data from its gene therapy programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), and Pyruvate Kinase Deficiency (PKD) at the 24th ASGCT Annual Meeting. The results indicate successful engraftment in FA patients, with 6 out of 9 showing clinical improvement. In the LAD-I trial, CD18 expression levels improved significantly, equating to better survival rates. Additionally, PKD patients demonstrated normalized hemoglobin levels post-treatment. The company aims to advance these therapies toward regulatory submissions.

Rocket Pharmaceuticals, Inc. (RCKT) reported financial results for Q1 2021, revealing a net loss of $40.2 million, or $0.65 per share, compared to $24.7 million, or $0.45 per share, year-over-year. The company has $466.4 million in cash, expected to fund operations into the second half of 2023. Key updates include successful Phase 1 trial results for RP-A501 in Danon Disease, and positive findings in RP-L201 for LAD-I. However, the FDA has requested a pause in patient dosing for RP-A501 to address risk mitigation, delaying enrollment by one quarter.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announces clinical data presentations at the 24th American Society of Gene and Cell Therapy Annual Meeting, May 11-14, 2021. Key presentations include interim results from a Phase 1/2 study on gene therapy for Leukocyte Adhesion Deficiency-I (LAD-I) and updates on Pyruvate Kinase Deficiency (PKD) and Fanconi Anemia (FA). With high unmet medical needs, Rocket aims to address severe pediatric diseases with innovative gene therapies, as detailed in oral and poster sessions throughout the event.

Rocket Pharmaceuticals (NASDAQ: RCKT) has announced positive interim data from its Phase 1/2 trial of RP-L201, a gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I), a life-threatening pediatric disorder. The data shows that all treated patients exhibited substantial CD18 expression, with one patient nearing survival at one year post-treatment. Significant improvements were noted in skin lesions and infection rates. The findings point towards potential BLA/MAA filings in the US and Europe. The study involved four patients with severe LAD-I, highlighting the therapy's safety and effectiveness.