NY resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, curing his sickle ...

Sebastien Beauzile from Laurelton, Long Island, has become the first person in New York State to be cured of sickle cell ...

A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...

Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given the nod for use by the NHS in England after an earlier rejection.

Crizanlizumab (Adakveo) failed to outperform placebo in patients with sickle cell disease, the phase III STAND trial showed.

But in December, Beauzile, 21, became the first person in New York State to be made symptom-free of sickle cell disease after undergoing Lyfgenia, a new type of gene therapy that uses stem cell ...

Vertex Pharma has made its name with ... with CRISPR Therapeutics on CTX001, a gene-editing therapy for beta thalassemia and sickle cell disease. “We have never been in more disease areas ...

Those who already own VRTX stock may retain it for some time to see if its CF sales continue to rise and how the Journavx and ...

From STAT’s Andrew Joseph: AstraZeneca said this morning it was buying the Belgian cell therapy company EsoBiotec for $425 million upfront, building out another cancer-targeting approach.

The fall in CRSP stock can be attributed to a lack of pipeline updates. Despite securing approval for its first marketed product in early 2024, it is yet to record product sales.