Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an ...

Vertex Pharmaceuticals said on Saturday its gene therapy helped children aged between 5 and 11 years with sickle cell disease ...

The chief medical officer of Genetix Biotherapeutics, formerly known as Bluebird bio, discusses timelines and operational ...

Key Highlights Motixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease enabling accelerated access to gene therapies 90% (9 of 10) of ...

About 100,000 people in the U.S. have sickle cell disease, a condition that primarily affects people of color, the CDC says.

Exa-cel is the first FDA-approved therapy utilizing CRISPR/Cas9, a type of gene editing technology through which patients' ...

Motixafortide alone and in combination with natalizumab can support the collection of the large number of stem cells required ...

A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers ...

An illustration of sickled and normal red blood cells. This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter.

Genetix Biotherapeutics Inc. today announced real-world data from the commercial implementation of ZYNTEGLO™ and LYFGENIA™, first-in-class ex vivo gene addition therapies for the treatment of ...

CHICAGO (WLS) -- Sickle cell anemia disproportionately affects Black people, causing severe pain, potential organ damage and in some cases, death. Many times, only blood transfusions can provide ...

The launch of BIRSA 101 marks a major breakthrough in CRISPR gene-editing technology, positioning India to deliver affordable ...