VivoSeq
Metabolic & Endocrine

Glycogen Storage Disease

0

Pipeline Programs↗

3

Active Trials↗

3

Companies Involved↗

Glycogen Storage Disease is an early-stage landscape with 0 programs across 3 companies. The most active players are Ultragenyx, Beam Therapeutics, Moderna.

Intelligence

Pipeline Metrics

Competitive Density

14/100

Pipeline Velocity

0/100

Mech. Concentration

N/A

Completion Rate

100/100

Attrition Rate

0/100

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.

Ultragenyx logo

Ultragenyx

0 programs

PreclinicalBeam Therapeutics logo

Beam Therapeutics

0 programs

PreclinicalModerna logo

Moderna

0 programs

Preclinical

Pipeline

Programs by Phase

No pipeline programs currently tracked for this disease.

Analysis

Trial Status

9

clinical trials

Completed5 · 56%
Recruiting2 · 22%
Unknown status1 · 11%
Active, not recruiting1 · 11%

Clinical Trials

Trial Activity

9 trials

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Recruiting·Beam Therapeutics logoBeam Therapeutics·Phase 1/2·NCT06735755·36 enrolled
GSDIa Disease Monitoring Program
Recruiting·Ultragenyx logoUltragenyx·N/A·NCT06636383·140 enrolled
A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Active, not recruiting·Moderna logoModerna·Phase 1/2·NCT05095727·15 enrolled
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Completed·Ultragenyx logoUltragenyx·Phase 3·NCT05139316·49 enrolled
Anaplerotic Therapy Using Triheptanoin for Patients With Glycogen Storage Disease Type I
Completed·—·Phase 1·NCT03665636·4 enrolled
The Effect of Triheptanoin in Adults With McArdle Disease (Glycogen Storage Disease Type V)
Completed·—·Phase 2·NCT02432768·22 enrolled
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Completed·Ultragenyx logoUltragenyx·Phase 1/2·NCT03517085·12 enrolled
Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia
Completed·UUniversity Medical Center Groningen·Phase 1/2·NCT04311307·20 enrolled
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14
Unknown status·OOrpha Labs·Phase 1/2·NCT03404856·5 enrolled

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