Mucopolysaccharidosis Type IIIA
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Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Orchard TherapeuticsMA - Boston
1 programAutologous CD34+ cells transduced with a lentiviral vector containing the human SGSH genePHASE_1_21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
Denali TherapeuticsDNL126
Orchard TherapeuticsAutologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
Clinical Trials (2)
Total enrollment: 25 patients across 2 trials
Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)
Start: Dec 2023Est. completion: Aug 202820 patients
Phase 1/2Active Not Recruiting
NCT04201405Orchard TherapeuticsAutologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
Start: Jan 2020Est. completion: Oct 20265 patients
Phase 1/2Active Not Recruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 companies competing in this space
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