RCKT earnings

First FDA-approved gene therapy for children with severe LAD-I due to biallelic variants in ITGB2 Severe LAD-I is an ultra-rare, life-threatening pediatric genetic immunodeficiency characterized by recurrent infections and high early-childhood mortality without treatment FDA grants Rare Pediatric Disease Priority Review Voucher Company to host conference call today, March 27 at 8:30 AM ET Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for KRESLADI™ (marnet

Conference call to be held later today at 8:30 a.m. ET Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing an integrated pipeline of genetic therapies for rare diseases, today announced an update related to RP-A501, its investigational gene therapy for Danon disease. A patient participating in the Phase 2 pivotal trial of RP-A501 experienced an unexpected Serious Adverse Event (SAE). The SAE involved clinical complications related to a capillary leak syndrome. Rocket is conducting a comprehensive root cause analysis and remains in active dialogue with the U.S. Food and Drug Administration (FDA) and other key stakeholders, with the c

— Reported positive safety data at ASGCT from pediatric cohort of Phase 1 Danon Disease study that demonstrated RP-A501 was well-tolerated; efficacy update across pediatric as well as adult cohorts on track for late Q3 2022— — Announced top-line data at ASGCT from pivotal Phase 2 trial for severe LAD-I that showed RP-L201 was well-tolerated with 100% overall survival at one year; regulatory filings anticipated in first half of 2023 — — Primary endpoint met for Fanconi Anemia pivotal Phase 2 trial, FDA dialogue initiated for BLA planning; continued data readouts for Fanconi Anemia and Pyruvate Kinase Deficiency programs expected in Q4 2022 — — Achieved Current Good Manufacturing Practice

— Continued to advance clinical gene therapy programs for the treatment of Danon Disease, Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency (PKD) — — Updates on all four programs to be presented at the 2022 Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) this month; trials on track for 2022 data readouts — — Appointed internationally recognized cardiovascular physician-scientist and biopharmaceutical executive Fady Malik, M.D., Ph.D., to Board of Directors – — Named experienced commercial leader Carlos Martin to Chief Commercial Officer and proven biotech executive Jessie Yeung to Vice President of Investor Relations and C

—Results demonstrate sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up— — NYHA class improvement (from II to I) in all three patients (two low-dose, one high-dose) with closely monitored immunosuppressive regimen— —Decreased cardiac wall thickness with improved or stabilized ejection fraction on echocardiogram in all three patients with closely monitored immunosuppressive regimen— — Sustained improvement or stabilization in BNP and 6-minute walk test across four patients— —Sustained cardiac LAMP2B expression greater than 50% by immunohistochemistry and improved cardiac tissue architecture in all three patients with clos

—Company to provide comprehensive update on the ongoing Phase 1 clinical trial of RP-A501, including data to be presented at the American Heart Association (AHA) Scientific Sessions 2021— Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host a conference call and live webcast on Monday, Nov. 15, 2021, at 8:30 a.m. ET. Rocket will present updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease, which will include data being presented in an ePoster at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually Nov. 13-1

— Treatment initiated in pediatric patient cohort in Danon Disease trial of RP-A501— — Treatment completed in nine of nine patients in LAD-1 Phase 1/2 trial; favorable safety profile and initial clinical benefit reported in all seven patients with at least 3 months of follow up — — Clinical updates anticipated in Danon Disease at AHA 2021 Scientific Sessions and in FA, LAD-I and PKD at 63rd ASH Annual Meeting — — Ending Balance Sheet with $421.5 Million in Cash; Cash Runway Expected into 2H'23 — Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today reports financial r

— Working with FDA on Changes to Clinical Trial Protocol in Danon Disease Trial; Rocket Anticipates Trial Will Resume in 3Q — — Increasing and Durable Benefit Observed in Low Dose Cohort (6.7e13 dose) in Danon Disease; Removing Higher Dose Cohort (1.1e14 dose) From Future Dosing Plans; Full Clinical Update Expected in 4Q – — Positive Results from FA, LAD-I, PKD Trials Presented at 24th ASGCT Annual Meeting Show Preliminary Evidence of Activity and Favorable Tolerability — — Clinical Updates in FA, LAD-I, PKD and IMO Also Expected in 4Q — — Ending Balance Sheet with $426.8 Million in Cash; Cash Runway Expected into 2H'23 — — Conference Call to Be Held at 4:30 p.m. ET Today — Rocket Pha

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host a conference call and live audio webcast on Monday, Aug. 9, 2021, at 4:30 p.m. ET to report second quarter 2021 financial results and recent business highlights. Investors may access the conference call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550 from outside the United States. Please refer to conference ID number 50210581. A live webcast of the call can be accessed under "Events" in the Investors section of the Company's website at https://ir.rocketpharma.com/. The webcast

—RP-A501 Phase 1 Trial for the Treatment of Danon Disease Placed on Clinical Hold To Address FDA Request For Additional Risk Mitigation Methods in Protocol; No New Safety Events Observed; Anticipate Delay in Enrollment by One Quarter — —Updated Low Dose Results in RP-A501 Treatment for Danon Disease Demonstrate Stabilization or Improvements in Key Clinical Outcomes Including 6MWT and NYHA Class; Full Data to be presented in 4Q — — Reported Hemoglobin Increases of Nearly 2x from Baseline Sustained Up to 6-Months Post RP-L301 Treatment in PKD — — Announced Sustained Efficacy, Durability and Safety Up to 18-Months Post RP-L201 Treatment in LAD-I — — Advanced Fifth Gene Therapy Program in

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that it will host a webcast on Monday, December 7 at 6:00PM EST to discuss presentations at the 62nd American Society of Hematology (ASH) Annual Meeting being held virtually December 5-8, 2020. The ASH presentations will highlight data from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I and the Pyruvate Kinase Deficiency programs. Following the discussion, Rocket management and key opinion leaders will be available for a brief Q&A session. To access the li