Scrip is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


Vertex Gears Up For Major Milestones, Starting With Sickle Cell Launch And Pain Drug Readout

Executive Summary

Having long attracted investors thanks to its promising pipeline, the moment of truth approaches for Vertex’s sickle cell gene therapy exo-cel, and VX-548, which has potential to transform acute and chronic pain treatment.

You may also be interested in...

Vertex Achieves Gene-Editing Milestone With First Ever CRISPR Therapy Approval

Great Britain is first to approve the groundbreaking CRISPR-based therapy for sickle cell and beta thalassemia. Vertex is yet to reveal its price, however, and wants to play down early expectations of its commercial potential.

Vertex’s Exa-Cel: Off-Target Gene Editing Analyses Sufficient Given Robust Clinical Efficacy, FDA Panel Says

Weighing Vertex’s evaluation of theoretical risks of off-target editing with the gene therapy’s strong clinical results in sickle cell disease, advisory committee says in silico and cellular analyses were sufficient but suggests closer monitoring of potential on- and off-target effects postapproval.

Roche Looks To Muscle Into Obesity Market With $2.7bn Carmot Buyout

The Swiss pharma sees the acquisition as the ‘backbone’ for its future cardiometabolic portfolio, which could include a combination with a muscle-building therapy.


Related Companies

Latest Headlines
See All



Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Thank you for submitting your question. We will respond to you within 2 business days. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts