inherited disease

News

After disappointing trial, Editas puts lead CRISPR drug on h...

Gene-testing specialist Editas Medicine has halted development of its lead clinical programme for congenital eye disorders after it generated lacklustre results in a phase 1/2 trial.

News

FDA turns down Amryt's epidermolysis bullosa drug

Amryt Pharma has been handed a major blow by the FDA, after the US regulator rejected its marketing application for Oleogel-S10, its drug for rare and debilitating skin disease epidermolysi

News

Applied Therapeutics slumps as FDA seeks more data on lead d...

Shares in Applied Therapeutics slumped today after it said it would have to delay a US filing for its lead drug programme AT-007, following an FDA request for more data.

Views & Analysis

Out of the darkness: new gene therapies for eye diseases

US FDA expert committee’s ‘yes’ could pave the way for a new generation of gene therapies for rare inherited eye disorders.

After disappointing trial, Editas puts lead CRISPR drug on h...

FDA turns down Amryt's epidermolysis bullosa drug

Applied Therapeutics slumps as FDA seeks more data on lead d...

Out of the darkness: new gene therapies for eye diseases

Rezzayo antifungal okayed as Candida auris rings alarm bells

An inside guide to unlocking clinical research in the UK

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