VivoSeq
Other

Methylmalonic Acidemia

0

Pipeline Programs↗

1

Active Trials↗

4

Companies Involved↗

Methylmalonic Acidemia is an early-stage landscape with 0 programs across 4 companies. The most active players are Moderna, HemoShear Therapeutics, PTC Therapeutics.

Intelligence

Pipeline Metrics

Competitive Density

6/100

Pipeline Velocity

0/100

Mech. Concentration

N/A

Completion Rate

0/100

Attrition Rate

80/100

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.

Moderna logo

Moderna

0 programs

PreclinicalH

HemoShear Therapeutics

0 programs

PreclinicalP

PTC Therapeutics

0 programs

PreclinicalL

LogicBio Therapeutics, Inc

0 programs

Preclinical

Pipeline

Programs by Phase

No pipeline programs currently tracked for this disease.

Analysis

Trial Status

5

clinical trials

Terminated3 · 60%
Active, not recruiting1 · 20%
Withdrawn1 · 20%

Clinical Trials

Trial Activity

Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
Terminated·HHemoShear Therapeutics·Phase 2·NCT04732429·26 enrolled
Ataluren for Nonsense Mutation Methylmalonic Acidemia
Terminated·PPTC Therapeutics·Phase 2·NCT01141075·11 enrolled
Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia
Terminated·LLogicBio Therapeutics, Inc·Phase 1/2·NCT04581785·4 enrolled
A Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia
Active, not recruiting·Moderna logoModerna·Phase 1/2·NCT04899310·74 enrolled
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia
Withdrawn·Moderna logoModerna·Phase 1/2·NCT03810690

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