Metabolic disorders

Base editing rescues liver disease in organoids

Chinese researchers have corrected a maple syrup urine disease mutation in patient-derived liver organoids using adenine base editing. The corrected...

Breaking: First personalised mRNA CRISPR therapy...

A team of academic and industry scientists have developed and delivered what may be the world’s first mRNA-based, patient-specific CRISPR therapy for...

RNA-based Therapies for Metabolic Liver Diseases

A new review in Gut explores how advances in RNA-based therapies may impact the treatment of liver metabolic diseases with unprecedented precision and...

Metabolic disorders

Familial Chylomicronemia Syndrome, FCS, (NCT07176923)
Sponsors:
CorrectSequence Therapeutics Co., Ltd

IND Enabling

Phase I

Phase II

Phase III

Type 1 Diabetes, T1D, (NCT06239636)
Sponsors:
Per-Ola Carlsson

IND Enabling

Phase I

Phase II

Phase III

Type 1 Primary Hyperoxaluria, PH1, (NCT06892301)
Sponsors:
Guangzhou Women and Children's Medical Center

IND Enabling

Phase I

Phase II

Phase III

Phenylketonuria, PKU, (NCT04768348)
Sponsors:
Homology Medicines, Inc

IND Enabling

Phase I

Phase II

Phase III

Mucopolysaccharidosis II, MPS II, (NCT05238324)
Sponsors:
Homology Medicines, Inc

IND Enabling

Phase I

Phase II

Phase III

Primary Hyperoxaluria Type 1, (NCT06839235)
Sponsors:
Arbor Biotechnologies

IND Enabling

Phase I

Phase II

Phase III

Glycogen Storage Disease Type Ia, GSDIa, (NCT06735755)
Sponsors:
Beam Therapeutics Inc.

IND Enabling

Phase I

Phase II

Phase III

Type 1 Primary Hyperoxaluria, PH1, (NCT06511349)
Sponsors:
RenJi Hospital

IND Enabling

Phase I

Phase II

Phase III

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