VivoSeq
Other

Propionic Acidemia

11

Pipeline Programs↗

2

Active Trials↗

4

Companies Involved↗

Propionic Acidemia is an emerging landscape with 11 programs across 4 companies. The most active players are CoA Therapeutics, Inc., a BridgeBio company, Sanofi, Moderna. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

38/100

Pipeline Velocity

6/100

Mech. Concentration

N/A

Completion Rate

50/100

Attrition Rate

33/100

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.

C

CoA Therapeutics, Inc., a BridgeBio company

1 program

Phase 1Sanofi logo

Sanofi

1 program

Phase 1Moderna logo

Moderna

2 programs

Phase 2R

RECORDATI GROUP

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

11programs
Small molecule655%
Gene therapy327%
RNA therapy218%

Modality classified for 100% of 11 pipeline programs.

Analysis

Trial Status

6

clinical trials

Terminated2 · 33%
Completed2 · 33%
Recruiting2 · 33%

Clinical Trials

Trial Activity

6 trials

Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia
Recruiting·Moderna logoModerna·Phase 1/2·NCT04159103·77 enrolled
Phase 1 Study of Intravenous Administration of a Serotype rh.10 Replication Deficient Adeno-associated Virus Gene Transfer Vector Expressing the Human Propionyl-CoA Carboxylase cDNA (AAVrh10-PCCA) to Individuals With Propionic Acidemia
Recruiting·MMayo Clinic·Phase 1·NCT07643844·9 enrolled
Carglumic Acid in Methylmalonic Acidemia and Propionic Acidemia
Completed·KKing Abdullah International Medical Research Center·Phase 3·NCT02426775·33 enrolled
Anaplerotic Therapy in Propionic Acidemia
Completed·—·Phase 1·NCT00645879·3 enrolled
Long-term Outcome of N-Carbamylglutamate Treatment in Propionic Acidemia and Methylmalonic Acidemia
Terminated·—·Phase 2·NCT01597440·1 enrolled
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
Terminated·CCoA Therapeutics, Inc., a BridgeBio company·Phase 1·NCT04836494·79 enrolled

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