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RegenXBio (RGNX) Drug Pipeline

1,133 Followers
Drug pipeline
Drug NameConditionStageStatusStudy NameTrial Start DateArticle
Rgx-121-3102Mps Ii, Hunter Syndrome (Mps Ii)Phase IIIActive Not RecruitingRGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)Nov 05, 2025
Rgx-202Duchenne Muscular Dystrophyphase2AndPhase3RecruitingAFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)Jan 04, 2023
Rgx-121Mucopolysaccharidosis Type Ii (Mps Ii)phase1AndPhase2CompletedRGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)Sep 23, 2020―
Rgx-111Mucopolysaccharidosis Type I (Mps I), Hurler Syndrome, Hurler-Scheie Syndromephase1AndPhase2SuspendedRGX-111 Gene Therapy in Patients With MPS IJun 18, 2018―
Rgx-121Mucopolysaccharidosis Type Ii (Mps Ii)phase2AndPhase3Active Not RecruitingCAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)May 01, 2018―

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 RegenXBio Inc. (RGNX) have in its pipeline
RegenXBio RGNX is currently developing the following drugs: Rgx-121-3102, Rgx-202, Rgx-121, Rgx-111. These drug candidates are in various stages of clinical development as the company works toward FDA approval.