Rocket Pharmaceuticals, Inc. RCKT

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) said September 15 that the FDA agreed to count three patients treated under the modified RP-A501 protocol toward the pivotal Phase 2 efficacy cohort of 12 male patients. Nine additional patients remain to be enrolled and treated, with dosing expected to finish by mid-2027. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is developing RP-A501 […]

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Needham said the FDA outcome was better than expected because the regulator could have demanded meaningful changes to Rocket’s Danon disease trial.

Rocket Pharmaceuticals (NASDAQ:RCKT) shares rose 8% on Tuesday after the company said it reached alignment with the US Food and Drug Administration on the pivotal Phase 2 trial of RP-A501 for Danon disease. The FDA confirmed a pivotal efficacy population of 12 patients, including the first three patients treated under the study’s modified protocol, according to Rocket.

The FDA confirmed continued enrollment and dosing under the modified trial protocol after reviewing safety data from the first three patients treated at the recalibrated dose.

CRANBURY, N.J., September 15, 2026--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and life-threatening diseases, focused on inherited cardiovascular disorders, today announced alignment with the U.S. Food and Drug Administration (FDA) on continued enrollment and dosing and key elements of the global pivotal Phase 2 trial of RP-A501 for the treatment of Danon disease, including the recalibrated dose, 12

CRANBURY, N.J., September 03, 2026--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and devastating diseases, focused on inherited cardiovascular disorders, today announced that the Company will participate in the 2026 Cantor Global Healthcare Conference and the Morgan Stanley 24th Annual Global Healthcare Conference in New York.

Rocket Pharmaceuticals' (RCKT) Q2 update confirmed its programs are on track, with management expect

CRANBURY, N.J., August 10, 2026--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and devastating diseases, focused on inherited cardiovascular disorders, today reported financial and recent operational results for the second quarter ended June 30, 2026.

CRANBURY, N.J., August 03, 2026--Rocket Pharmaceuticals, Inc. (Rocket or the Company) (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and devastating diseases, focused on inherited cardiovascular disorders, today announced a positive clinical safety update regarding the initial three patients treated under the modified protocol for its global, pivotal Phase 2 trial of RP-A501 in Danon disease. Early safety has been promising with th

We recently compiled a list of the 10 Most Promising Small-Cap Stocks to Buy According to Hedge Funds. Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) is one of the most promising stocks on our list. TheFly reported on June 3 that BofA Securities analyst Jason Zemansky increased the price target on RCKT to $10 from $9 while maintaining a Buy […]