Metabolic disorders

Breaking: First personalised mRNA CRISPR therapy...
CMN Interview

May. 15, 2025

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
CMN Briefs

Feb. 25, 2025

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

Type 1 Primary Hyperoxaluria, PH1, (NCT06892301)
Sponsors:
Guangzhou Women and Children's Medical Center
Indicator
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
Indicator
IND Enabling
Phase I
Phase II
Phase III
Primary Hyperoxaluria Type 1, (NCT06839235)
Sponsors:
Arbor Biotechnologies
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Glycogen Storage Disease Type Ia, GSDIa, (NCT06735755)
Sponsors:
Beam Therapeutics Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Type 1 Primary Hyperoxaluria, PH1, (NCT06511349)
Sponsors:
RenJi Hospital
Indicator
IND Enabling
Phase I
Phase II
Phase III
Ornithine Transcarbamylase Deficiency, OTC, (NCT06255782)
Sponsors:
iECURE, Inc.
IndicatorIndicator
IND Enabling
Phase I
Phase II
Phase III
Refractory Dyslipidemias, (ACTRN12623000809639)
Sponsors:
CRISPR Therapeutics AG
Indicator
IND Enabling
Phase I
Phase II
Phase III
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