First Participant Randomized in AskBio European Phase 2 Gene Therapy Trial for Congestive Heart Failure
AB-1002 is an investigational gene therapy for the treatment of adults with non-ischemic cardiomyopathy and New York Heart ...
Zawya26d
Qatar: Sidra Medicine establishes gene therapy centre for rare paediatric diseases
The Gene Therapy Centre is designed to address the growing demand for innovative treatments for SMA and DMD. It plans to expand the service in the future for other rare and complex genetic diseases.
National Institute for Health and Care Excellence26d
Groundbreaking one-off gene therapy approved for severe sickle cell disease
Our independent appraisal committee has approved the use of gene editing therapy exagamglogene autotemcel (exa-cel) for use in the NHS in England, providing a potential cure for some people with ...
Managed Healthcare Executive26d
Gene Therapy’s Pipeline Has a Steady Flow of More Than 2,000 Candidates
according to report delineating the gene therapy development landscape. Just over 2,000 gene therapies are at some stage of development and 11 are in the preregistration phase when the clinicals ...
NHS England26d
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
The one-off gene therapy, known as exagamglogene autotemcel (or ‘exa-cel’), has been approved for use on the NHS in England from today (Friday 31 January) by the National Institute for Health and Care ...
GEN27d
Rentschler Withdraws from Cell and Gene Therapy Arena
Rentschler Biopharma is withdrawing from the field of cell and gene therapy, ceasing operations at its Stevenage, UK site, and increasing its focus on biologics (cell culture-based therapeutic ...
ahajournals.org27d
Abstract TP228: CYP2C19 GENETIC VARIANTS AND THROMBOTIC RISK IN CLOPIDOGREL THERAPY: A PATH TO PERSONALIZED ANTI-PLATELET THERAPY
This study explores genetic variants associated with clopidogrel resistance to improve personalized therapy for thrombotic conditions. Objective: To evaluate the relationship between CYP2C19 ...
Gulf Times28d
Sidra Medicine establishes gene therapy centre for rare paediatric diseases
Sidra Medicine, a member of Qatar Foundation, has established a gene therapy centre to treat rare genetic diseases such as Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD).
Medical Xpress28d
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple ...
GoodNewsNetwork29d
New Base Editing Gene Therapy Cures its First Patient: A Sickle-Cell Sufferer Now in the ‘Gym Every Day’
MORE GENETIC MODIFICATIONS: Gene Therapy at Duke Improves NC Dad’s Failing Vision Just in Time for His Baby’s Birth “In my opinion, I’m perfect. I never felt fine before—before ...