Gemma Biotherapeutics

2 results · Page 1 of 1

Swipe the table sideways for more columns.

Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, GM1 gangliosidosis, Gangliosidoses, Gangliosidosis, Generalized GM1, Type 1, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, beta-Galactosidase Deficiency, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis	Active, not recruiting	Phase 1 / Phase 2	2026-06-30		NCT04713475
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1	Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Spinal Muscular Atrophies of Childhood, Spinal Muscular Atrophy Type I, central nervous system disorder, hereditary disease, inherited neurodegenerative disorder, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, spinal cord disorder, spinal muscular atrophy	Recruiting	Phase 1 / Phase 2	2026-06-29		NCT07070999

Clinical trials