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343,220 Results for "rocket pharma".
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Gene therapy
Rocket Aborts FDA Filing for Rare Blood Disease Gene Therapy
Rocket Pharmaceuticals’ strategic realignment initiative in July pulled funding from fanca-cel, which the biotech was developing for Fanconi anemia.
October 6, 2025
·
2 min read
·
Tristan Manalac
Gene therapy
Rocket Re-Ignites Phase II Trial After FDA Lifts Clinical Hold
In late May, a patient died after receiving Rocket Pharmaceuticals’ investigational gene therapy for Danon disease, spurring the hold. After lowering the dose and changing the regimen of immune modulators patients receive, the company has received FDA clearance for the trial to continue.
August 20, 2025
·
2 min read
·
Dan Samorodnitsky
Press Releases
Rocket Pharmaceuticals Reports Third Quarter 2025 Financial Results and Highlights Recent Progress
November 6, 2025
·
12 min read
Press Releases
Rocket Pharmaceuticals Announces FDA Acceptance of BLA Resubmission of KRESLADI™ for the Treatment of Severe Leukocyte Adhesion Deficiency-I (LAD-I)
October 15, 2025
·
8 min read
Press Releases
Rocket Pharmaceuticals Announces Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) - October 10, 2025
October 13, 2025
·
6 min read
Press Releases
Rocket Pharmaceuticals to Participate in Upcoming September 2025 Investor Conferences
August 29, 2025
·
5 min read
Podcast
COVID-19 Vaccine Overhaul, Rocket Grounded, ‘One Tough Hombre’ on MFN
HHS Secretary RFK Jr. removes the COVID-19 vaccine recommendation for healthy kids and pregnant women—the latest in a string of changes to vaccine policies; judge issues an order to halt HHS’ reorganization and mass layoff plans; Rocket Pharmaceuticals’ pivotal Danon disease trial is on hold after a patient death; and President Trump has named Mehmet Oz to spearhead his Most Favored Nation drug pricing policy.
May 28, 2025
·
2 min read
·
Heather McKenzie
Press Releases
MGI Tech Announces Exclusive Licensing Agreement with Swiss Rockets AG to Globalize Proprietary CoolMPSä Sequencing Technology
October 29, 2025
·
4 min read
Press Releases
Rocket Pharmaceuticals Reports Second Quarter 2025 Financial Results and Highlights Recent Progress
August 8, 2025
·
13 min read
Press Releases
Rocket Pharmaceuticals Announces Strategic Corporate Reorganization and Pipeline Prioritization of Cardiovascular Programs
July 25, 2025
·
8 min read
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