Disease Domain | Count |
---|---|
Endocrinology and Metabolic Disease | 6 |
Nervous System Diseases | 6 |
Immune System Diseases | 1 |
Top 5 Drug Type | Count |
---|---|
AAV based gene therapy | 10 |
Gene therapy | 2 |
Target |
Mechanism VEGF inhibitors |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 3 |
First Approval Ctry. / Loc.- |
First Approval Date- |
Target |
Mechanism Iduronate sulfatase replacements [+1] |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 2/3 |
First Approval Ctry. / Loc.- |
First Approval Date- |
Target |
Mechanism TPP1 gene transference |
Active Org. |
Originator Org. |
Active Indication |
Inactive Indication- |
Drug Highest PhasePhase 2 |
First Approval Ctry. / Loc.- |
First Approval Date- |
Start Date08 May 2024 |
Sponsor / Collaborator |
Start Date24 Jul 2023 |
Sponsor / Collaborator |
Start Date17 May 2023 |
Sponsor / Collaborator |
Drug(Targets) | Indications | Global Highest Phase |
---|---|---|
RGX-121 ( GAG ) | Mucopolysaccharidosis II More | Phase 3 Clinical |
RGX-202 (Regenxbio) ( SLC6A8 ) | Muscular Dystrophy, Duchenne More | Phase 2 Clinical |
RGX-111 ( IDUA ) | Mucopolysaccharidosis I More | Phase 2 Clinical |
CLN2 gene therapy(REGENXBIO, Inc.) ( TPP1 ) | Neuronal Ceroid-Lipofuscinoses More | Phase 2 |
Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor(RegenxBio) ( LDLR ) | Hyperlipidemias More | Phase 2 |