Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
By Deena Beasley (Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing ...
The News International8h
Investors shift focus from gene therapy to lucrative weight-loss drugs
Gene therapy, which promises a possible cure for rare diseases like sickle cell, is losing early investors to higher-reward ...
Gene-Therapy Maker Sarepta Tanks After Patient Death
Shares of Sarepta Therapeutics have struggled for the past year due to concerns about the commercial potential of its gene ...
STAT2d
Novartis finally delivers gene therapy data in older SMA patients. Will they still want it?
In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...
Breakthrough for baby with rare illness in world-first gene therapy trial
Fourteen-month-old Tomas was diagnosed with ornithine transcarbamylase (OTC) deficiency when he was a few weeks old ...
University of California1d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Clinical Trials Arena4h
iECURE gene therapy data suggests partial restoration of functional OTC
ECURE previously announced that the first patient dosed with ECUR-506 for OTC deficiency achieved a complete clinical ...
Upgrading Beam Therapeutics: Promising BEAM-302 Data Opens Door For Durable Gene Therapy
Beam Therapeutics reported promising Phase 1/2 data for BEAM-302, demonstrating durable gene correction for AATD. See why I ...
MedPage Today on MSN3d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
BioPharma Dive1d
Novartis builds case for new SMA gene therapy
To Novartis, fresh late-stage data support the idea that its intrathecal drug, which has the same active ingredient as ...
OXB honoured at 2025 CDMO Leadership Awards in 'Cell & Gene Therapy – Global' category
OXB honoured at 2025 CDMO Leadership Awards in 'Cell & Gene Therapy – Global' category Oxford, UK – 21 March 2025: OXB (LSE: ...