Many drug companies are developing gene-based treatments for sickle cell and beta thalassemia, a related blood disorder. But Novartis and Precision say they still see a need for treatments that can work inside the body, rather than via stem cells edited in a laboratory, and can be deployed in countries where stem cell transplants can’t be easily carried out.
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The European Sickle Cell Federation officially launches its global campaign “Sickle Cell Has No Colour.. It’s in the Blood”, on World Sickle Cell Day june 19th 2022.
The campaign is to highlight the misconceptions relating to the racial structure of Sickle Cell Disease and its global prevalence.

Find more information on this campaign and the press release here.

4TH Global Sickle Cell Disease Congress in Paris, France.

ESCF participates in the 4th Global Sickle Cell Disease congress in Paris, France organised by the Global Sickle Cell Disease Network-GSCDN.
The federation successfully held a patient perspective session during the conference on the 18th of June and highlighted major expectations from patients in Europe and around the globe on treatments, management and policy for Sickle cell disease.

The gathering was an opportunity for the ESCF Board to hold its first in person board and patient meeting since the organisation’s official establishment in June 2020.

Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With exagamglogene autotemcel (exa-cel) at the 2022 European Hematology Association (EHA) Congress | Vertex Pharmaceuticals Newsroom (vrtx.com)

31 patients with severe Sickle Cell Disease characterized by recurrent vaso-occlusive crises (VOCs) (mean of 3.9 VOCs per year over the prior two years) were free of excruciating pain episodes after an exa-cel infusion through duration of follow-up, with follow-up ranging from 2.0 to 32.3 months.