Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating pediatric diseases. It has four clinical-stage ex vivo lentiviral vector programs for fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction; pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia; and infantile malignant osteopetrosis, a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. It has license agreements with Fred Hutchinson Cancer Research Center; Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; CIEMAT and UCL Business PLC; The Regents of the University of California; and REGENXBIO Inc. The company also has a research and collaboration agreement with Lund University; and strategic collaboration agreement with Stanford University School of Medicine. Rocket Pharmaceuticals, Inc. is headquartered in Cranbury, New Jersey.
IPO Year: 2015
Exchange: NASDAQ
Website: rocketpharma.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 10/16/2024 | Sector Outperform | Scotiabank | |
| 4/2/2024 | $39.00 | Neutral | Goldman |
| 10/24/2023 | $65.00 | Overweight | Cantor Fitzgerald |
| 2/1/2023 | $45.00 | Overweight | Morgan Stanley |
| 11/8/2022 | $53.00 | Buy | Canaccord Genuity |
| 11/1/2022 | $35.00 | Buy | BTIG Research |
| 7/8/2022 | $22.00 | Outperform | Raymond James |
| 2/24/2022 | $62.00 → $64.00 | Outperform | SVB Leerink |
| 12/15/2021 | $56.00 → $60.00 | Buy | Chardan Capital |
| 10/20/2021 | Outperform | Cowen |
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Canaccord Genuity analyst Whitney Ijem maintains Rocket Pharmaceuticals (NASDAQ:RCKT) with a Buy and lowers the price target from $49 to $40.
Chardan Capital analyst Geulah Livshits maintains Rocket Pharmaceuticals (NASDAQ:RCKT) with a Buy and maintains $62 price target.
Needham analyst Gil Blum maintains Rocket Pharmaceuticals (NASDAQ:RCKT) with a Buy and lowers the price target from $53 to $52.
Rocket Pharmaceuticals Inc (NASDAQ:RCKT) stock is trading down after the company announced a regulatory update for Kresladi (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I). The FDA has issued a Complete Response Letter (CRL) to Rocket’s Biologics License Application for Kresladi, in which the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review. Rocket met with FDA senior leaders from the Center for Biologics Evaluation and Research (CBER) to align on the limited scope of additional CMC information needed to support Kresladi’s approval.
U.S. stock futures were higher this morning, with the Nasdaq futures gaining around 0.2% on Friday. Shares of NIKE, Inc. (NYSE:NKE) fell sharply in today's pre-market trading following fourth-quarter earnings and soft FY25 guidance. Analysts at Stifel and Morgan Stanley downgraded the stock following the report. NIKE shares dipped 15.6% to $79.42 in pre-market trading. Here are some big stocks recording losses in today's pre-market trading session. Accolade, Inc. (NASDAQ:ACCD) shares tumbled 32.1% to $4.34 in pre-market trading after the company reported first-quarter financial results and issued worse-than-expected FY25 revenue guidance. Also, Needham maintained a Buy rating
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All six evaluable Phase 1 patients with Danon disease are alive and transplant-free up to age 25 years RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10% reduction in LV mass index at 12 months and sustained through most recent follow up (up to five years) First patient treated shows preliminary evidence of robust protein expression in heart on five-year biopsy All evaluable patients had reductions in NYHA heart failure (from Class II to Class I) and improvements in KCCQ (median 27-point increase) at 24-54 months Substantial improvements in troponin (median reduction 84%) and BNP (median reduction 57%) observed 24-54 mo
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the company will be presenting new data on the results of the RP-A501 Phase 1 trial to treat patients with Danon disease at the American Heart Association's 2024 Late-Breaking Science sessions being held from November 16-18, 2024 in Chicago, Illinois. Details for the presentation are as follows: Title: Danon Disease Phase 1 RP-A501 Results: The First Single-Dose Intravenous Gene Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a Monogenic Cardiomyopathy Session
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the UBS Global Healthcare Conference in Rancho Palos Verdes, CA. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, November 12, at 5:00 p.m. PT. A webcast of the fireside chat will be available here on the Investors section of the Company's website. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipe
Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association's 2024 Late-Breaking Scientific Sessions Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025 Rolling BLA submission initiated for RP-L102 for Fanconi Anemia Progress on FDA-approval of KRESLADI for severe LAD-I; Approval anticipated in 2025 Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors Cash, cash equivalents and investments of approximately $235.7M; expected operational runway into 2026
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that all patients have been enrolled in the global, pivotal Phase 2 clinical trial evaluating RP-A501 to treat male patients with Danon disease. After the two-patient safety run-in, followed by harmonized global site activations, the remaining 10 patients were enrolled across the United States (U.S.) and European Union within three months. Given the prevalence of Danon disease across regions, the Company plans to pursue regulatory filings concomitantly in the U.S. and ex-U.S. "From a
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of Mikael Dolsten, M.D., Ph.D., as an independent director to its Board of Directors. Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience. "As an industry-leading pharmaceutical executive, I am very pleased to welcome Mikael to our Board of Directors," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "His research and development experience will help advance our leading scientific capabilities and
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the Company will participate in the Morgan Stanley 22nd Annual Global Healthcare Conference and the 2024 Cantor Fitzgerald Global Healthcare Conference in New York. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat and management will host investor meetings at each conference. Participation details are as follows: Morgan Stanley 22nd Annual Global Healthcare Conference Date: September 5, 2024 Time: 2:35 PM ET 2024 Cantor Fitzgerald Global Healthcare
Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat PKP2 arrhythmogenic cardiomyopathy Working toward FDA-approval of KRESLADI for severe LAD-I; Commercial infrastructure and capabilities in place for launch Presented long-term KRESLADI™ follow-up data from the global Phase 1/2 study for severe LAD-I, results from the global Phase 1/2 study of RP-L102 for Fanconi Anemia, and data from the Phase 1 study of RP-L301 for PKD at ASGCT in May Cash, cash equivalents and investments of approximately $278.8M; expected operational runway into 2026 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet needs, today announced a regulatory update for KRESLADI™ (marnetegragene autotemcel; marne-cel), a lentiviral (LV) vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I). The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to Rocket's Biologics License Application for KRESLADI™ wherein the FDA requested limited additional Chemistry Manufacturing and Controls (CMC) information to complete its review. Rocket met with FDA senior leader
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the European Commission (EC), based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted orphan medicinal product designation for RP-A601, the Company's adeno-associated virus (AAV.rh74)-based gene therapy candidate for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). PKP2-ACM is a devastating, inherited heart disease associated with life-threatening arrhythmias, card
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Scotiabank initiated coverage of Rocket Pharmaceuticals with a rating of Sector Outperform
Goldman initiated coverage of Rocket Pharmaceuticals with a rating of Neutral and set a new price target of $39.00
Cantor Fitzgerald initiated coverage of Rocket Pharmaceuticals with a rating of Overweight and set a new price target of $65.00
Morgan Stanley initiated coverage of Rocket Pharmaceuticals with a rating of Overweight and set a new price target of $45.00
Canaccord Genuity initiated coverage of Rocket Pharmaceuticals with a rating of Buy and set a new price target of $53.00
BTIG Research initiated coverage of Rocket Pharmaceuticals with a rating of Buy and set a new price target of $35.00
Raymond James initiated coverage of Rocket Pharmaceuticals with a rating of Outperform and set a new price target of $22.00
SVB Leerink reiterated coverage of Rocket Pharmaceuticals with a rating of Outperform and set a new price target of $64.00 from $62.00 previously
Chardan Capital reiterated coverage of Rocket Pharmaceuticals with a rating of Buy and set a new price target of $60.00 from $56.00 previously
Cowen resumed coverage of Rocket Pharmaceuticals with a rating of Outperform
Enrollment completed in September and dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease Updated Phase 1 data from RP-A501 for Danon disease anticipated at American Heart Association's 2024 Late-Breaking Scientific Sessions Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Preliminary data expected in the first half of 2025 Rolling BLA submission initiated for RP-L102 for Fanconi Anemia Progress on FDA-approval of KRESLADI for severe LAD-I; Approval anticipated in 2025 Appointed Mikael Dolsten, M.D., Ph.D., to Board of Directors Cash, cash equivalents and investments of approximately $235.7M; expected operational runway into 2026
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of Mikael Dolsten, M.D., Ph.D., as an independent director to its Board of Directors. Dr. Dolsten is an accomplished industry executive with extensive global pharmaceutical management experience. "As an industry-leading pharmaceutical executive, I am very pleased to welcome Mikael to our Board of Directors," said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. "His research and development experience will help advance our leading scientific capabilities and
Aaron Ondrey joins as Chief Financial Officer bringing seasoned leadership experience in financial management, strategic planning, commercial finance, and capital allocation Additional key appointments within existing corporate leadership team expand capabilities and expertise spanning early discovery through commercialization Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of Aaron Ondrey, as Chief Financial Officer, and the further evolution of its corporate leadership team through key appointments with existin
Advancing leading pipeline of six disclosed programs across AAV cardiovascular and LV hematology portfolios; all milestones remain on track for 2024 Expanding commercial capabilities to support launch of LV portfolio beginning with KRESLADI™ (marnetegragene autotemcel) for severe LAD-I; PDUFA date of June 30, 2024 Cash, cash equivalents and investments of approximately $407.5M; expected operational runway into 2026 Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and operational results for the fourth quarter and year
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the appointment of R. Keith Woods as an independent, non-executive director to its Board of Directors. Mr. Woods is a seasoned executive with more than 30 years of experience spanning commercialization, global operations, business strategy and supply chain. "I'm thrilled to welcome Keith to our Board of Directors, who brings deep industry expertise and invaluable experience to the Rocket Board as we build upon our commercial and operational infrastructure to support our anticipa
— Mayo Pujols brings nearly three decades of technical operations and manufacturing experience, of which the past five years have been exclusively focused on cell and gene therapy, from leading biopharma companies including Andelyn Biosciences, Novartis and Celgene— — Mr. Pujols will lead technical operations and the Company's state-of-the-art, recently cGMP ready, Clinical and potential Commercial AAV manufacturing facility in Cranbury, N.J. — Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces the appointmen
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Fady Malik, M.D., Ph.D., to its Board of Directors. Dr. Malik brings nearly 25 years of experience as an internationally recognized cardiovascular physician-scientist and highly successful biopharmaceutical executive. He will serve as an independent non-executive director. "Fady is a tremendous addition to our Board of Directors, especially at this exciting time as we seek agency alignment on a Phase 2, potentially registration-enabling clinical study for Danon Disease, and continue to bui
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Jessie Yeung as Vice President of Investor Relations and Corporate Finance. Ms. Yeung brings more than 15 years of investor relations, corporate finance and capital market experience across industries including the biopharmaceutical and financial sectors. She will be responsible for leading the investor relations function and capital markets strategy. "I'm thrilled to welcome Jessie to Rocket as we continue to advance our world-class pipeline of gene therapy clinical programs and move towa
— Positive Phase 1 data from Danon Disease gene therapy trial demonstrated sustained benefit across clinical, functional and biomarker endpoints in all four adult patients with long-term follow up; pediatric cohort data expected Q3 2022 — — Reported engraftment in all nine severe LAD-I patients treated in pivotal Phase 2 trial and CD18 expression ranging from 26% to 87% of normal; top-line data expected Q2 2022— — Demonstrated evidence of engraftment in six Fanconi Anemia patients with at least 12-months of follow-up with MMC resistance between 16% and 63% at a minimum of one timepoint in pivotal Phase 2 trial; top-line data expected Q3 2022 — — Cash position of $388.7M; runway expected
Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces the appointment of Martin L. Wilson as General Counsel, Chief Compliance Officer and Senior Vice President. Mr. Wilson brings nearly 20 years of legal, compliance and executive experience and accomplishment within the life sciences industry. Mr. Wilson will be responsible for the Legal and Compliance functions and serve as a key member of the leadership team. "The Rocket family is thrilled to welcome Martin to our talented executive leadership team as we advance our world-class pipeline of gene therapies and
— 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