Man Cured Of Sickle Cell Disease In New York Thanks To New Gene Therapy
NY resident Sebastien Beauzile was treated with a pioneering new gene therapy approach called Lyfgenia, curing his sickle ...
The Punch on MSN3 天
Gene therapy delivers alternative sickle cell cure in US
Sebastien Beauzile from Laurelton, Long Island, has become the first person in New York State to be cured of sickle cell ...
MedPage Today on MSN7 天
Approved Sickle Cell Drug Fails to Beat Placebo in Trial
Crizanlizumab (Adakveo) failed to outperform placebo in patients with sickle cell disease, the phase III STAND trial showed.
GEN - Genetic Engineering and Biotechnology News10 小时
From Tools to Trials: Editing Therapy Companies Pivot to Development
A pair of privately-held developers of editing-based therapies have some successes to show for their recent efforts to ...
MoneyWeek on MSN13 天
Vertex Pharmaceuticals is an uncommon opportunity in rare diseases
Vertex Pharmaceuticals operates in a profitable subsector and is poised for further success, says Dr Michael Tubbs ...
pharmaphorum2 天
UK first to approve Vertex, CRISPR drug for blood disorders
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.
Harvard Medical School29 天
Creating the World’s First CRISPR Medicine, for Sickle Cell Disease
In 2008, Orkin, Sankaran, and colleagues achieved their vision by identifying a new therapeutic target for sickle cell disease. In December 2023, through the development efforts of CRISPR Therapeutics ...
Newsday6 天
Sickle cell anemia patient reunites with Long Island doctors whose gene therapy treatments ...
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 ...
pharmaphorum4 天
FDA fast tracks type 1 diabetes cell therapy from Vertex
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 ...