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Rocket Pharmaceuticals (RCKT)
NASDAQ:RCKT
Rp-A701 Is A Recombinant Viral Vector Composed Of An Aav Serotype Rh.74 (Aavrh.74) Capsid Encapsulating The Transgene, Bcl2-Associated Athanogene 3 (Bag3)
Dilated Cardiomyopathy (Dcm)
Phase I
Not Yet Recruiting
A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy
Jul 25, 2025
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Rp-A501
Danon Disease
Phase II
Recruiting
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
Oct 11, 2023
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Rp-L301
Pyruvate Kinase Deficiency
Phase I
Completed
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
Sep 24, 2019
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FAQ
What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases:
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
a. Preclinical testing
b. Phase 1 – Safety and dosage
c. Phase 2 – Effectiveness and side effects
d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments
e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does Rocket Pharmaceuticals (RCKT) have in its pipeline
RCKT is currently developing the following drugs: Rp-A701 Is A Recombinant Viral Vector Composed Of An Aav Serotype Rh.74 (Aavrh.74) Capsid Encapsulating The Transgene, Bcl2-Associated Athanogene 3 (Bag3), Rp-A501, Rp-L301. These drug candidates are in various stages of clinical development as the company works toward FDA approval.