Rocket Pharmaceu Stock Price, News & Analysis

Rocket Pharmaceuticals (RCKT) announced positive updates from its Phase 1 Danon Disease Trial for RP-A501, showing initial improvements in pediatric patients and sustained clinical benefits in adults. The trial includes both safety and efficacy data, indicating that RP-A501 is generally well tolerated across cohorts. Notably, patients exhibited significant reductions in heart failure markers like BNP and improvements in NYHA class. The company anticipates support for advancing to a Phase 2 pivotal study with FDA feedback expected later this year.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical biotech firm focused on genetic therapies for rare childhood disorders, will have CEO Gaurav Shah participate in a fireside chat at the Chardan 6th Annual Genetic Medicines Conference on October 4 at 2:00 p.m. ET, in New York City. The event will be accessible via a live audio webcast on the company's website, with a replay available later. Rocket is advancing therapies for conditions like Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Danon Disease.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has announced the acquisition of Renovacor, Inc. (NYSE: RCOR) in an all-stock transaction valued at approximately $2.60 per share. This acquisition aims to enhance Rocket's leadership in AAV-based cardiac gene therapy, targeting BAG3-associated dilated cardiomyopathy, which presents a significant unmet medical need. The deal is expected to close by Q1 2023, pending shareholder and regulatory approvals. Rocket anticipates the acquisition will create strong synergies, adding approximately $38M in projected cash at closing and extending its cash runway into Q2 2024.

Rocket Pharmaceuticals is set to present updated data from its Phase 1 Danon Disease trial on September 30, 2022, at the Heart Failure Society of America Annual Scientific Meeting. The presentation will reveal efficacy and safety updates from both pediatric and adult cohorts. An investor event discussing this data will stream at 8:00 a.m. ET. The trial aims to address the serious pediatric heart condition, Danon Disease, utilizing gene therapy.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced that CEO Gaurav Shah will participate in a fireside chat at the Morgan Stanley 20th Annual Global Healthcare Conference on Monday, Sept. 13, at 3:30 p.m. ET. A live audio webcast will be available on the company’s Investors section of their website. Rocket focuses on developing genetic therapies for rare childhood disorders, including Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency. For more information, visit www.rocketpharma.com.

Rocket Pharmaceuticals (NASDAQ: RCKT) reported positive safety data from its Phase 1 Danon Disease study, indicating RP-A501 was well-tolerated in pediatric patients. They announced 100% overall survival in a pivotal Phase 2 trial for severe LAD-I, with regulatory filings expected in early 2023. The primary endpoint for the Fanconi Anemia trial was met, initiating FDA dialogue for BLA planning. The company reported a cash position of $321.4 million, ensuring operational runway into H1 2024. The appointment of Mayo Pujols as Chief Technical Officer bolsters its leadership in gene therapy.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has appointed Mayo Pujols as Chief Technical Officer and Executive Vice President. Pujols, with nearly 30 years of experience in technical operations and gene therapy, previously served as CEO of Andelyn Biosciences. He will oversee Rocket's cGMP-ready AAV manufacturing facility in Cranbury, N.J., preparing for a Phase 2 pivotal trial for Danon Disease. His leadership aims to enhance the company’s gene therapy capabilities and advance regulatory filings for its lentiviral platform.

Rocket Pharmaceuticals (NASDAQ: RCKT), a leader in genetic therapies for childhood disorders, announces CEO Gaurav Shah's participation in the William Blair Biotech Focus Conference on July 12-13, 2022, in New York City. A pre-recorded fireside chat will be accessible starting July 11 at 9:00 a.m. ET. The discussion will focus on Rocket's innovative gene therapy pipeline, including recent positive trial data for severe Leukocyte Adhesion Deficiency-I and updates on Fanconi Anemia and other conditions. More details can be found on Rocket's investor website.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) reported positive results from its Phase 2 trial for RP-L201, a gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I). The trial demonstrated 100% overall survival at one year post-infusion and significant clinical improvement in patients. All nine patients showed sustained CD18 expression with no serious adverse events. Rocket plans to engage health authorities for regulatory filings anticipated in the first half of 2023, focusing on the therapy's potential to transform treatment options for LAD-I.