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Community Conversations

Making an Informed Decision As a Family

4 mins WATCH NOW

Jewel is a teenager living with sickle cell disease. She and her mother, Paula make informed decision about participating in clinical studies.

My Contribution to Clinical Studies

3 mins WATCH NOW

Ada Gonzalez lives with sickle cell disease and enrolled in a clinical study decades ago that she benefits from today.

Research for Adults Aging with Sickle Cell

3 mins WATCH NOW

Andre, an adult living with sickle cell disease, talks about the need for treatment options for the aging and future generations with sickle cell disease.

Research Updates

World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia

17 Nov 2023

Health authorities in the U.K. on Thursday approved a new kind of medicine that uses CRISPR gene editing to treat the blood diseases sickle cell and beta thalassemia, marking it the first time a drug built with the Nobel Prize-winning technology has won regulatory clearance anywhere in the world.

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New treatment for sickle cell disease could soon be approved by the U.S. FDA: Dr. Peter Lin

17 Nov 2023

Doctor Peter Lin will tell us about a groundbreaking new treatment that uses gene-editing…with the potential to cure a serious blood disorder. And it could soon get the greenlight for use in the U-S.  

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Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

16 Nov 2023

The United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)

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