Genetic Disorders

Pipeline Moves: Trial completion bumps advancement prospects for AstraZeneca’s oncology drug

This week on Pipeline Moves, we look at the recent completion of a Phase I/II trial investigating AstraZeneca’s Imfinzi in…

Horizon doses first participant in Phase I gout therapy trial

Horizon Therapeutics has dosed the first participant in a Phase I trial of HZN-457, previously called ARO-XDH, to treat gout,…

Regenxbio concludes subject dosing in Phase I/II severe MPS I trial

Regenxbio has concluded subject dosing in the expanded Cohort 2 of the Phase I/II clinical trial of investigational therapy RGX-111 to treat severe…

Pipeline Moves: positive topline results in pulmonary hypertension, genetic and metabolic disorders

This week on Pipeline Moves, we start with positive topline results from two Phase III trials in Alagille syndrome and pulmonary…

UK MHRA approves Avrobio’s Phase I/II Hunter syndrome therapy trial

The UK Medicines and Healthcare Products Regulatory Agency (MHRA) has granted approval for Avrobio’s clinical trial application (CTA) to begin…

Pipeline Moves: Trial completions in Duchenne muscular dystrophy and chronic urticaria

This week on Pipeline Moves, we look into the completion of a Phase I trial in Duchenne muscular dystrophy (DMD),…

FDA authorises Edgewise’s Phase II duchenne muscular dystrophy trial

The US Food and Drug Administration (FDA) has granted authorisation for Edgewise Therapeutics’ Phase II clinical trial of EDG-5506 for treating duchenne…

DCT Adoption Tracker reveals top therapy areas using a decentralisation approach

Telemedicine remains the leading decentralisation approach in clinical trials in Q2 of this year. The continued rise of telemedicine as…

CSL reports Phase III data for garadacimab to treat hereditary angioedema

CSL has reported positive top-line data from the Phase III VANGUARD clinical trial of garadacimab (CSL312) as a durable preventive…

Pipeline Moves: Lilly completes Phase II rheumatoid arthritis trial, next phase prospects rise

This week on Pipeline Moves, we bring positive news from two major big pharma players. Eli Lilly completed a Phase…