European regulator ready to review curative sickle cell disease therapy

25 January 2023

The European Medicines Agency is to review a regulatory submission from Swiss gene editing specialist CRISPR Therapeutics (Nasdaq: CRSP) and partner Vertex Pharmaceutical (Nasdaq: VRTX).

The companies have submitted for approval of the gene therapy candidate exagamglogene autotemcel (exa-cel) formerly known as CTX001, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

The submission is backed by positive data from two global Phase III studies, which tested exa-cel)= as a potential one-time curative treatment for people with SCD or TDT.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK