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 |
|---|---|---|---|
| 3/12/2025 | $50.00 | Outperform | BMO Capital Markets |
| 12/30/2024 | $32.00 | Outperform | Wedbush |
| 12/18/2024 | $29.00 | Buy | Jefferies |
| 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 |
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 data for the first time from the Phase 1 clinical trial of RP-A601 to treat patients with plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held from May 13-17 in New Orleans, LA. Details for the oral presentation of the abstract at ASGCT are as follows: Title: Preliminary Data from a Phase I Gene Therapy Trial of RP-A601 (AAVrh.74-PKP2a) for Adult Pati
Phase 2 pivotal study of RP-A501 for Danon disease is ongoing; Program update expected mid-year 2025 Initial data from the Phase 1 study of RP-A601 for PKP2-ACM anticipated May 2025 IND submission for BAG3-DCM program expected mid-year 2025 Sarbani Chaudhuri appointed Chief Commercial & Medical Affairs Officer Cash, cash equivalents and investments of approximately $318.2M; expected operational runway into the fourth quarter of 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 recent operational results for the f
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 management will participate in the 24th Annual Needham Virtual Healthcare Conference taking place on April 8, 2025. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat at 8:45 a.m. ET on Tuesday, April 8. A webcast of the fireside chat will be available here and on the Investors section of the Company's website. An archived replay of the webcast will be available for approximately 30 days following the event. About Rocket Pharmaceuticals, Inc. Rocket Pha
Dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease; Program update expected in the first half of 2025 New England Journal of Medicine publication of Phase 1 study of RP-A501 to treat patients with Danon disease Long-term data from the Phase 1 trial of RP-A501 for Danon disease presented at AHA on safety and meaningful efficacy Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Initial data expected in the first half of 2025 Regulatory reviews ongoing for KRESLADI (severe LAD-I) and RP-L102 (Fanconi Anemia) Cash, cash equivalents and investments of approximately $372.3M; expected operational runway into the third quarter of 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 the Company will participate in the 43rd Annual J.P. Morgan Healthcare Conference, in San Francisco, California. Gaurav Shah, M.D., Chief Executive Officer, is scheduled to present on Monday, January 13, at 3:45 p.m. PT and host investor meetings throughout the conference. A webcast of the presentation will be available here and will be posted on the Investors section of the Company's website. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is 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, announced today the pricing of an upsized underwritten public offering of 13,200,000 shares of its common stock at a public offering price of $12.50 per share. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $165 million. All shares in the offering are to be sold by Rocket. In addition, Rocket has granted the underwriters a 30-day option to purchase up to an additional 1,980,000 shares of its common stock
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, announced today that it intends to offer and sell $150 million of shares of its common stock in an underwritten public offering. In addition, Rocket intends to grant the underwriters a 30-day option to purchase up to an additional 15 percent of shares of its common stock offered in the public offering. The offering is subject to market conditions and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. Morgan Stanley, Leerin
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 7th Annual Evercore HealthCONx Conference in Miami, Florida. Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat on Tuesday, December 3, at 1:45 p.m. ET and host investor meetings throughout the day. A webcast of the fireside chat will be available here beginning at 1:45 p.m. ET and will be posted on the Investors section of the Company's website. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NAS
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
— 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
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
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BMO Capital Markets initiated coverage of Rocket Pharmaceuticals with a rating of Outperform and set a new price target of $50.00
Wedbush initiated coverage of Rocket Pharmaceuticals with a rating of Outperform and set a new price target of $32.00
Jefferies initiated coverage of Rocket Pharmaceuticals with a rating of Buy and set a new price target of $29.00
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