Fanconi Anemia Complementation Group A
Hematology
3
Pipeline Programs
1
Companies
4
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
2
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Rocket PharmaceuticalsNEW YORK, NY
4 programs1
2
RP-L102Phase 21 trial
RP-L102Phase 21 trial
RP-L102Phase 11 trial
Safety and efficacy assessmentsN/A1 trial
Active Trials
+1 more trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Rocket PharmaceuticalsRP-L102
Rocket PharmaceuticalsRP-L102
Rocket PharmaceuticalsRP-L102
Rocket PharmaceuticalsSafety and efficacy assessments
Clinical Trials (4)
Total enrollment: 23 patients across 4 trials
Gene Therapy for Fanconi Anemia, Complementation Group A
Start: Jul 2020Est. completion: May 20265 patients
Phase 2Active Not Recruiting
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
Start: Nov 2019Est. completion: Feb 20257 patients
Phase 2Active Not Recruiting
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
Start: Jan 2019Est. completion: Mar 20222 patients
Phase 1Unknown
Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy
Start: Jun 2020Est. completion: Jan 20359 patients
N/AEnrolling By Invitation
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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
1 companies competing in this space