The MHRA is the first Regulatory body in the world to authorise a Crispr-based gene therapy. Casgevy has been authorised for patients over twelve years old who suffer from beta-thalassaemia and sickle cell disease and for whom haematopoietic stem cell transplantation is an appropriate treatment, but a suitable donor is not available. The MHRA approval sets a president in the UK, and the NHS innovative medicine fund should pave the way for eligible patients to receive a treatment for their often painful and life-threatening disease.
Casgevy
by Jackie (née Heneghan) Peck | Jan 30, 2024 | Blog, MHRA, NHS
Recent Posts
Testimonials
“An ‘All-Inclusive’ Training” Pop Ohelga – Course Rating 10/10
GDP & RP Training
“Training was well laid out and followed a great structure. The trainer was very very good and very attentive”
Cogent Gold Standard Responsible Person Training
“Very good overview of GDP requirements which has given me a much better understanding of client requirements for the services and products we supply.” Bill Castle – Course Rating 9/10
GDP Training
We appointed PCL to assist us with our new CQC compliance registration. From start to finish, they acted with professionalism and expertise; taking what at first seemed to be a tedious and daunting process and making it clear and easy to understand. Even after our successful registration, the consultant remained on hand to answer any outstanding questions and to ensure that we felt adequately prepared for a potential visit from the CQC. Having had such a positive experience, we have no hesitatio… Read more
Pharmacy developing patient services
Very useful day with a good overview of MHRA GMP & GDP process, procedures and roles and responsibilities. Isatec on site training – Andrew King
GDP/RP Training
” Very interesting presentation which allowed me to gain a proper overview of the GDP process” Jack – Course Rating 9/10
GDP Training
“Brian is very thorough and very informative. Interactive too. A++++, Highly recommended” Anonymous – Course Rating 9/10
GDP & RP Training
Since 2012 we have been using PCL for regulatory support, staff resourcing, training and general advice. We have consistently found that the whole team has a high level of technical competence, are always willing to get stuck in to help us and are able to come up with robust ideas to improve our systems. I would have no hesitation in recommending their services in any of these areas.
Testimonial From Integro Pharmacy - David Hammond
“Very informative training day.” “Highly recommended”
Manus Mulligan
“A lot of valuable information to digest! Thank you.” Anna Drys – Course rating 9/10
GDP Training