'Will it really work?': Young sickle cell patient among the first to start new gene therapy
Since two gene therapies were approved for sickle cell disease in late 2023, only a handful of patients have started the ...
Medindia1h
YolTech Therapeutics to Initiate a Clinical Trial for YOLT-204, a First-in-Class Bone Marrow-Targeted In Vivo Gene Editing Therapy for ß-Thalassemia
YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204 , an investigational ...
New Texas Children's, Baylor program will combat sickle cell disease in sub-Saharan Africa
Dr. Joseph Lubega calls sickle cell disease “probably the saddest story of a health problem that you could imagine.” He and ...
HHS Challenges Vertex Over Fertility Services Program Tied To Casgevy Gene Therapy, Says Company Is 'Grasping at Straws'
HHS challenges Vertex Pharmaceuticals' fertility services program, alleging violations of anti-kickback laws linked to its ...
FierceBiotech13d
Vertex pays Orna $65M to work on next-gen gene therapies for blood disorders
Vertex is paying Orna $65 million upfront, in part via convertible note, according to a Jan. 7 release. Orna—which acquired ...
BioPharma Dive13d
Vertex, startup Orna to partner on gene editing research
The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle ...
Correcting Genetic Spelling Errors With Next-Generation Crispr
Treatments for rare diseases are hard to create and expensive to deliver, but there is new hope for editing the software of ...
Medical Xpress on MSN8d
Study highlights need for standardized measurement methods in gene therapy
National Institute of Standards and Technology scientists looked at multiple techniques used to measure the modified viruses ...
NBC News2d
'Will it really work?': Young sickle cell patient among the first to start new gene therapy
“You are one of the few in the country and even in the world, you know, taking this big step — gene therapy,” said Campbell, director of the hospital’s Comprehensive Sickle Cell Disease ...
YolTech Therapeutics to Initiate a Clinical Trial for YOLT-204, a First-in-Class Bone Marrow-Targeted In Vivo Gene Editing Therapy for β-Thalassemia
YolTech Therapeutics, a clinical-stage gene editing company dedicated to delivering lifelong cures, announced the initiation of a clinical trial for YOLT-204, an investigational therapy for the ...
Yahoo News2d
'Will it really work?': Young sickle cell patient among the first to start new gene therapy
But for a brief moment on a recent December morning, Wedam, 19, who has sickle cell disease, allowed himself to believe ... the first major step of the revolutionary sickle cell gene therapy process. ...