£1.65m gene-editing therapy offers hope of cure for some blood disorder patients
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...
GoodNewsNetwork3d
New Base Editing Gene Therapy Cures its First Patient: A Sickle-Cell Sufferer Now in the ‘Gym Every Day’
Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.
Gene therapies have been uneven for DMD — but these companies hope to turn the tide
Despite mixed results using gene therapies to treat Duchenne muscular dystrophy, drug developers are pushing ahead with the ...
Groundbreaking £1.65m gene-editing therapy approved for NHS use offers hope to severe sickle cell patients
The UK has approximately 17,500 individuals living with sickle cell disease, a condition particularly prevalent amongst those ...
NHS England1d
Revolutionary gene-editing therapy for sickle cell ‘offers hope of a cure’ for NHS patients
NHS patients with sickle cell disease will be able to benefit from a groundbreaking gene-editing treatment that offers the prospect of a cure for the condition. The one-off gene therapy, known as ...
cirm.ca.gov12h
CIRM Awards Nearly $100 Million to Boost Discovery, Translational, and Clinical Disease Research
South San Francisco, CA, January 30, 2025 – The California Institute for Regenerative Medicine (CIRM), one of the world’s largest institutions dedicated to regenerative medicine, has awarded nearly ...
‘Sickle cell pain is the worst I have ever felt in my life - it’s hard to put into words'
Everything you need to know about agonising sickle cell disease and the ‘revolutionary’ new DNA therapy that's being rolled ...
Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation ...
Sickle cell patients share delight over approval of new treatment
NHS officials estimate that around 50 people a year will receive treatment now that it has been approved for use for certain ...
Pharmabiz8d
Solid Biosciences’ AAV-based gene therapy candidate, SGT-212 for Friedreich’s ataxia receives US FDA fast track designation
Solid Biosciences’ AAV-based gene therapy candidate, SGT-212 for Friedreich’s ataxia receives US FDA fast track designation: Charlestown, Massachusetts Thursday, January 23, 2 ...