RCKT - Public offerings

All three initial patients treated with RP-A501 under the modified pivotal Phase 2 protocol completed the protocol-defined safety observation period; no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns observed Rocket is actively engaging with the FDA to align on the path to rapidly completing the pivotal Phase 2 trial in Danon disease Multiple clinical and regulatory milestones remain on track for the second half of 2026, including a broader Danon disease program update, a regulatory update for the planned RP-A601 pivotal study in PKP2-ACM, and initial patient dosing with RP-A701 in BAG3-DCM FDA approval of KRESLADI™ and subsequent monetizatio

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a fully integrated, commercial-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) for gross proceeds of $180 million. The Rare Pediatric Disease Priority Review Voucher was granted by the FDA in March 2026 in connection with the approval of KRESLADI™, Rocket's gene therapy for severe leukocyte adhesion deficiency-I (LAD-I), a rare and life-threatening primary immunodeficiency. As previously reported, before the PRV sale Rocket had cash, cash equivalents and investments of $1

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, 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

Agreement reached with FDA on Phase 2 pivotal study design of RP-L301 for PKD to support accelerated approval; Initiating single-arm, 10-patient pivotal study with primary endpoint of ≥1.5 point Hgb improvement at 12 months Initiated Phase 2 pivotal trial of RP-A501 for Danon Disease following FDA alignment Initiated Phase 1 study of RP-A601 for PKP2-ACM FDA accepted BLA with Priority Review for KRESLADITM (marnetegragene autotemcel) for severe LAD-I; PDUFA target action date is March 31, 2024 Product filings for RP-L102 for Fanconi Anemia anticipated in the first half of 2024 in the U.S. and Europe Cash, cash equivalents and investments of approximately $437.2M; expected operatio

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of (i) 7,812,500 shares of its common stock at a public offering price of $16.00 per share and (ii) to certain investors, pre-funded warrants to purchase 3,126,955 shares of common stock at a price of $15.99 per pre-funded warrant, which represents the per share public offering price for the common stock less the $0.01 per share exercise price for each such pre-funded warrant. The gross proceeds to Rocket fro

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today that it intends to offer and sell shares of its common stock, and to certain investors, pre-funded warrants to purchase shares of its common stock, in an underwritten public offering. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $150 million. The purchase price of each pre-funded warrant will equal the price per share at which shares of common stock are be

— Positive updates from Phase 1 clinical trial in Danon Disease showed RP-A501 was well tolerated with evidence of durable treatment effect and disease improvement for both pediatric patients and adult patients with up to nine and 36 months of follow-up, respectively— — Phase 2 pivotal study design and endpoint selection for Danon Disease on track with FDA feedback anticipated in Q4 2022; previously disclosed data to be presented at AHA 2022— — LAD-I and Fanconi Anemia (FA) gene therapy programs remain on track, regulatory filings for LAD-I and FA anticipated 1H 2023 and 2H 2023, respectively; clinical data across LAD-I, FA and PKD lentiviral (LV) programs to be presented at the 64th ASH

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today the pricing of an underwritten public offering of 6,800,000 shares of its common stock at a public offering price of $14.75 per share. The gross proceeds to Rocket from the offering, before deducting underwriting discounts and commissions and offering expenses, are expected to be $100.3 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 1,020,000 sh

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of investigational genetic therapies for rare childhood disorders with high unmet need, announced today that it has commenced an underwritten public offering of $100 million of shares of its common stock. Rocket also intends to grant the underwriters a 30-day option to purchase up to an additional $15 million of shares of its common stock. All of the shares in the proposed offering are to be sold by Rocket. Morgan Stanley, J.P. Morgan and SVB Securities are acting as joint book-running managers, and BTIG is acting as lead manager for the proposed offering.

CRANBURY, N.J.--(BUSINESS WIRE)--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 and operational results for the fourth quarter and year ended December 31, as well as guidance for anticipated 2021 milestones. “2021 is poised to be another exciting year for Rocket,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket. “We anticipate clinical data from all five of our gene therapy programs, initiation of our first in-house AAV cGMP production from our new manufacturing facility, and continued strengthening of our organization, manuf

CRANBURY, N.J.--(BUSINESS WIRE)--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 Company’s plans for the buildout of its new Research and Development (R&D) and Chemistry, Manufacturing and Controls (CMC) operation which will also serve as the Company’s new headquarters in Cranbury, New Jersey. This new 103,720 ft2 facility will support clinical development of Rocket’s growing pipeline of lentivirus (LV) and adeno-associated virus (AAV) gene therapies from discovery through pivotal trials, with space for potential future expansion and commercia

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 the closing of its previously announced upsized underwritten public offering of 5,339,286 shares of its common stock, including the exercise in full by the underwriters of their option to purchase an additional 696,428 shares, at the public offering price of $56.00 per share. The gross proceeds to Rocket from the offering are expected to be approximately $299 million, before deducting the underwriting discounts and commissions and other offering expenses. Rocket

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 the pricing of an upsized underwritten public offering of 4,642,858 shares of its common stock at a public offering price of $56.00 per share. The gross proceeds to Rocket from the offering are expected to be approximately $260 million, before deducting the underwriting discounts and commissions and other offering expenses. Rocket has granted the underwriters a 30-day option to purchase up to an additional 696,428 shares of its common stock. All the shares in th

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 has commenced an underwritten public offering of $175 million of shares of its common stock. Rocket also intends to grant the underwriters a 30-day option to purchase up to an additional 15% of the shares of its common stock sold in the public offering. All the shares in the offering are to be sold by Rocket. The offering is subject to market conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size