VivoSeq
Hematology

Hemophilia B

37

Pipeline Programs↗

12

Active Trials↗

16

Companies Involved↗

Hemophilia B is a moderately active landscape with 37 programs across 16 companies. The most active players are Sangamo Therapeutics, Spark Therapeutics, Inc., Biogen. 2 therapies are approved with 12 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

57/100

Pipeline Velocity

0/100

Mech. Concentration

67/100

Completion Rate

85/100

Attrition Rate

10/100

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.

S

Sangamo Therapeutics

1 program

Phase 1S

Spark Therapeutics, Inc.

1 program

Phase 1Biogen logo

Biogen

1 program

Phase 1Novo Nordisk logo

Novo Nordisk

1 program

Phase 1Regeneron logo

Regeneron

2 programs

Phase 2C

Catalyst Biosciences

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

79%

Small molecule

19 of 24 classified programs — the dominant modality here.

21%ALL OTHERSGene therapy521%

Small molecule leads; the ring shows all 24 classified programs, each share of the total. Modality classified for 91% of 43 pipeline programs.

Analysis

Trial Status

49

clinical trials

Completed29 · 59%
Active, not recruiting8 · 16%
Recruiting4 · 8%
Terminated4 · 8%
Unknown status3 · 6%
Withdrawn1 · 2%

Clinical Trials

Trial Activity

49 trials

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B
Recruiting·BBe Biopharma·Phase 1/2·NCT06611436·24 enrolled
An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
Recruiting·CSL Behring logoCSL Behring·N/A·NCT06008938·500 enrolled
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Recruiting·IInstitute of Hematology & Blood Diseases Hospital, China·Phase 1·NCT05709288·9 enrolled
A Two-Part Open-Label Study of REGV131-LNP1265, A CRISPR/Cas9 Based Coagulation Factor IX Gene Insertion Therapy in Participants With Hemophilia B
Recruiting·Regeneron logoRegeneron·Preclinical·NCT06379789·130 enrolled
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
Active, not recruiting·BBaxalta now part of Shire·Phase 1/2·NCT01687608·30 enrolled
Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B
Active, not recruiting·Pfizer logoPfizer·Phase 2·NCT03307980·21 enrolled
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Active, not recruiting·Pfizer logoPfizer·Phase 3·NCT03861273·51 enrolled
Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Chinese Subjects With Hemophilia B Previously Treated With FIX Therapy
Active, not recruiting·CSL Behring logoCSL Behring·Phase 3·NCT06399289·23 enrolled
Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
Active, not recruiting·SSt. Jude Children's Research Hospital·Phase 1·NCT00979238·14 enrolled
HOPE-B Extension: Etranacogene Dezaparvovec 5-Year
Active, not recruiting·CSL Behring logoCSL Behring·Phase 3·NCT05900004·54 enrolled
ETERNITY: Etranacogene Dezaparvovec in Hemophilia B
Active, not recruiting·CSL Behring logoCSL Behring·Phase 3·NCT04677803·54 enrolled
BENEGENE-2: Fidanacogene Elaparvovec in Hemophilia B
Active, not recruiting·Pfizer logoPfizer·Phase 3·NCT04394572·45 enrolled
Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
Completed·CSL Behring logoCSL Behring·Phase 1·NCT01233440·25 enrolled
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
Completed·CSL Behring logoCSL Behring·Phase 1/2·NCT01361126·17 enrolled
Safety and Efficacy of Benefix in Patients With Hemophilia B in Usual Care Settings in China
Completed·Pfizer logoPfizer·Phase 4·NCT02336178·70 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)