VivoSeq
Other

Osteopetrosis

11

Pipeline Programs↗

2

Active Trials↗

7

Companies Involved↗

Osteopetrosis is an emerging landscape with 11 programs across 7 companies. The most active players are Rocket Pharmaceuticals, Akero Therapeutics, Alnylam Pharmaceuticals. 1 therapy is approved with 5 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

38/100

Pipeline Velocity

6/100

Mech. Concentration

33/100

Completion Rate

57/100

Attrition Rate

30/100

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 1Akero Therapeutics logo

Akero Therapeutics

1 program

Phase 2Alnylam Pharmaceuticals logo

Alnylam Pharmaceuticals

1 program

Phase 3Mario Negri Institute for Pharmacological Research logo

Mario Negri Institute for Pharmacological Research

1 program

Phase 3S

SPP Pharmaclon Ltd.

1 program

Phase 3G

Genzyme, a Sanofi Company

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

94%

Small molecule

16 of 17 classified programs — the dominant modality here.

6%ALL OTHERSCell therapy16%

Small molecule leads; the ring shows all 17 classified programs, each share of the total. Modality classified for 100% of 18 pipeline programs.

Analysis

Trial Status

10

clinical trials

Completed4 · 40%
Terminated3 · 30%
Unknown status1 · 10%
Active, not recruiting1 · 10%
Recruiting1 · 10%

Clinical Trials

Trial Activity

10 trials

A Phase I/II Open Label Study to Assess Safety, Feasibility and Efficacy of ex Vivo Expanded, Autologous Haematopoietic Stem and Progenitor Cell Populations That Contain CD34+ Cells Transduced With a Lentiviral Vector Encoding the TCIRG1 cDNA in Children With Autosomal Recessive Osteopetrosis Caused by Mutations in the TCIRG1 Gene.
Recruiting·FFondazione Telethon·Preclinical·NCT07665021·8 enrolled
MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Active, not recruiting·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT02171104·149 enrolled
ACTIMMUNE in Intermediate Osteopetrosis
Completed·LLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center·Phase 2·NCT02666768·5 enrolled
Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis
Completed·IInterMune·N/A·NCT00043329·6 enrolled
Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis
Completed·FFDA Office of Orphan Products Development·Phase 3·NCT00004402·30 enrolled
Allogeneic Transplantation For Severe Osteopetrosis
Completed·MMasonic Cancer Center, University of Minnesota·Phase 2/3·NCT00775931·7 enrolled
Stem Cell Transplantation for Children Affected With Osteopetrosis
Terminated·SSt. Jude Children's Research Hospital·N/A·NCT00145587·15 enrolled
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
Terminated·Rocket Pharmaceuticals logoRocket Pharmaceuticals·Phase 1·NCT04525352·1 enrolled
Reduced Intensity AlloTransplant For Osteopetrosis
Terminated·MMasonic Cancer Center, University of Minnesota·Phase 2·NCT00638820·3 enrolled
Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis
Unknown status·TTehran University of Medical Sciences·Phase 2/3·NCT01087398·10 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)