Exciting news! We’ve added a special extra session to the HCC Online Education series. Vertex Pharmaceuticals Limited will present on CRISPR-Cas9 gene editing (Exa-cel) and its use in severe sickle cell disease and thalassaemia.
This timely presentation follows the recent NHS England approval of Exa-cel gene-editing therapy for sickle cell, and last year, thalassaemia. This one-time treatment targets the root cause of these haemoglobinopathies by editing a gene in bone marrow stem cells. Clinical trials have demonstrated a “functional cure” in nearly all participants, effectively eliminating painful sickle cell crises and hospitalisations.
This presentation is for haemoglobinopathy clinical staff only, rather than the public, and will be an exciting insight into gene editing. Please see below for the full details!
Presentation Details:
- Date: Friday 21st February 2025
- Time: 15:30 – 16:30
- Platform: Microsoft Teams Webinar
- Topic: An overview of the latest CRISPR-Cas9 gene editing (Exa-cel) clinical data in Severe Sickle Cell and Transfusion-Dependent Beta Thalassaemia
- Presenter: Tom Blair, Senior Medical Science Liaison (Haemoglobinopathies), Vertex
- Link To Attend: Click Here to Join – MS Teams
Introduction to Vertex & Tom Blair
Vertex Pharmaceuticals is a global biotechnology company focused on creating transformative medicines for people with serious diseases. For over a decade, Vertex has been committed to developing and delivering innovative therapies that address the root cause of diseases, improving patients’ lives. Their headquarters are in Boston (USA) and London (UK) and there is a strong focus on research and development.
Tom Blair is a Medical Science Liaison at Vertex with extensive experience in haematology and gene therapy. His research background includes a PhD from the University of Bristol focused on platelet function and a postdoctoral fellowship at Harvard Medical School/Boston Children’s Hospital, where he participated in clinical trials, including those for sickle cell. For the past several years, he has specialised in blood and coagulation disorders, with a dedicated focus on gene therapy and sickle cell/transfusion-dependent thalassaemia for the last 3.5 years.
Additional Educational Information:
If you are new to haemoglobinopathies, or even if you are not, here is a short video from Ted Ed that explains the basic overview of how CRISPR works. For more haemoglobinopathy videos check out our new-to-haemoglobinopathy page or for those who work in haemoglobinopathy please check out our clinical zone educational videos.
