Pharmafile Logo
  • - PMLiVE
  • Diversity, Equity and Inclusion
  • - PMLiVE
  • Communiqué 53 Cover Image

cell and genetic therapies

- PMLiVE

Diabeloop’s DBLG2 receives FDA and CE clearance for automated insulin delivery without meal input

While a lack of meal announcements can lead to lower glycaemic control, it reduces the mental burden for those with type 1 diabetes

- PMLiVE

NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

About 15,000 people in England are living with the inherited blood disorder

- PMLiVE

Study finds siblings with unique genetic defects could help progress type 1 diabetes drug search

The chronic, life-long disease affected around 8.4 million people worldwide in 2021

- PMLiVE

Pfizer’s sickle cell disease therapy Oxbryta recommended by NICE for NHS use

About 4,000 people in England with the genetic blood disorder are expected to benefit from the decision

- PMLiVE

NHS rolls out artificial pancreas for adults and children with type 1 diabetes in England

Type 1 diabetes accounts for approximately 10% of all diabetes cases in the UK

EU flag

EC approves Vertex/CRISPR’s Casgevy gene therapy for inherited blood disorders

There are now more than 8,000 patients who are potentially eligible for treatment with Casgevy

EU flag

CHMP recommends gene therapy for beta thalassemia and sickle cell disease

If approved, Casgevy will be the only genetic therapy in the EU to treat these conditions

- PMLiVE

Two KCL research projects awarded £4.8m to help find cure for type 1 diabetes

The Type 1 Diabetes Grand Challenge has awarded six research projects grants totalling £13m

- PMLiVE

FDA approves two cell-based gene therapies for sickle cell disease

Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

- PMLiVE

MHRA authorises world-first gene therapy for two inherited blood disorders

Casgevy is now the first licensed treatment that uses the gene-editing tool CRISPR

- PMLiVE

bluebird bio presents positive results for inherited blood disorder gene therapies

The company’s sickle cell disease therapy was accepted for FDA priority review earlier this year

- PMLiVE

FDA completes meeting for potential genetic therapy to treat sickle cell disease

If approved, exa-cel will be the first genetic therapy available to treat patients with severe sickle cell disease

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links