VivoSeq
Rare Disease

Fanconi Anemia

34

Pipeline Programs↗

9

Active Trials↗

18

Companies Involved↗

Fanconi Anemia is a moderately active landscape with 34 programs across 18 companies. The most active players are Rocket Pharmaceuticals, Immunex, Par Pharmaceutical, Inc.. 2 therapies are approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

55/100

Pipeline Velocity

19/100

Mech. Concentration

14/100

Completion Rate

71/100

Attrition Rate

20/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

Rocket Pharmaceuticals logo

Rocket Pharmaceuticals

1 program

Phase 1I

Immunex

1 program

Phase 1P

Par Pharmaceutical, Inc.

1 program

Phase 1Mario Negri Institute for Pharmacological Research logo

Mario Negri Institute for Pharmacological Research

1 program

Phase 1Novartis logo

Novartis

3 programs

Phase 2Boehringer Ingelheim logo

Boehringer Ingelheim

2 programs

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

82%

Small molecule

28 of 34 classified programs — the dominant modality here.

18%ALL OTHERSProtein / enzyme26%Cell therapy26%Monoclonal antibody13%Gene therapy13%

Small molecule leads; the ring shows all 34 classified programs, each share of the total. Modality classified for 100% of 41 pipeline programs.

Analysis

Trial Status

35

clinical trials

Completed17 · 49%
Recruiting5 · 14%
Terminated5 · 14%
Active, not recruiting3 · 9%
Unknown status2 · 6%
Withdrawn2 · 6%
Other1 · 3%

Clinical Trials

Trial Activity

35 trials

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy
Recruiting·—·Phase 2·NCT02143830·70 enrolled
Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing Briquilimab
Recruiting·PPorteus, Matthew, MD·Phase 1/2·NCT04784052·18 enrolled
The Experience and Management of Cancer Screening-Related Anxiety in Fanconi Anemia: an Ethnographic Study
Recruiting·National Cancer Institute (NCI)·N/A·NCT06744283·20 enrolled
MRI as Noninvasive Innovative Approach in Detection and Monitoring of Malignant Oral Lesions in Fanconi Anemia Patients
Recruiting·UUniversity of Minnesota·N/A·NCT07649031·80 enrolled
Gene Transfer for Fanconi Anemia Using a Self-inactivating Lentiviral Vector
Recruiting·SShenzhen Geno-Immune Medical Institute·Preclinical·NCT03351868·10 enrolled
Eltrombopag for People With Fanconi Anemia
Active, not recruiting·National Heart, Lung, and Blood Institute (NHLBI)·Phase 2·NCT03206086·25 enrolled
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
Active, not recruiting·Rocket Pharmaceuticals logoRocket Pharmaceuticals·Phase 2·NCT04069533·7 enrolled
Gene Therapy for Fanconi Anemia, Complementation Group A
Active, not recruiting·Rocket Pharmaceuticals logoRocket Pharmaceuticals·Phase 2·NCT04248439·5 enrolled
Prenatal Transplantation for Fetuses With Fanconi Anemia
Not yet recruiting·—·Phase 1/2·NCT07408583·12 enrolled
TBI Dose De-escalation for Fanconi Anemia
Completed·MMasonic Cancer Center, University of Minnesota·Phase 2/3·NCT00352976·83 enrolled
Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1
Completed·HHospital Universitari Vall d'Hebron Research Institute·Phase 2·NCT02931071·13 enrolled
Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia
Completed·FFred Hutchinson Cancer Center·Phase 1·NCT00093743·2 enrolled
Quercetin in Children With Fanconi Anemia; a Pilot Study
Completed·—·Phase 1·NCT01720147·30 enrolled
Eltrombopag for Treatment of Fanconi Anemia
Completed·—·Phase 2·NCT06045052·8 enrolled
Hematopoietic Stem Cell Transplant for Fanconi Anemia
Completed·MMedical College of Wisconsin·Phase 2·NCT01071239·1 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)