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Rocket Pharmaceuticals (9IP1) Drug Pipeline

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Drug pipeline
Drug NameConditionStageStatusStudy NameTrial Start DateArticle
Rp-A701 Is A Recombinant Viral Vector Composed Of An Aav Serotype Rh.74 Capsid Encapsulating The Transgene, Bcl2-Associated Athanogene 3Dilated Cardiomyopathy (Dcm)Phase INot Yet RecruitingA Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated CardiomyopathyJul 25, 2025
Rp-L301, Intervention/TreatmentPyruvate Kinase DeficiencyPhase IISuspendedClinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase DeficiencyMay 15, 2024―
Rp-A501Danon DiseasePhase IIRecruitingA Gene Therapy Study of RP-A501 in Male Patients With Danon DiseaseOct 11, 2023
Rp-A601Pkp2 Arrhythmogenic Cardiomyopathy (Pkp2-Acm)Phase IRecruitingA Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)May 22, 2023―
Rp-L301Pyruvate Kinase DeficiencyPhase ICompletedGene Therapy for Pyruvate Kinase Deficiency (PKD)Sep 24, 2019―

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
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 Pharma (DE:9IP1) have in its pipeline
Rocket Pharmaceuticals DE:9IP1 is currently developing the following drugs: Rp-A701 Is A Recombinant Viral Vector Composed Of An Aav Serotype Rh.74 Capsid Encapsulating The Transgene, Bcl2-Associated Athanogene 3, Rp-L301, Intervention/Treatment, Rp-A501, Rp-A601, Rp-L301. These drug candidates are in various stages of clinical development as the company works toward FDA approval.