FierceBiotech8d
4DMT tosses 2 programs, halts funds for 3 others as it zeroes in on latest-stage clinical gene therapy
4D Molecular Therapeutics is axing two clinical programs and cutting off funds for three other assets, with resources to be ...
Medscape3d
Gene Therapy in Hemophilia: High Hopes and Costs, Low Uptake
Life-changing gene therapies for hemophilia have become available, yet many patients and providers have not yet rushed to try ...
First of its kind gene therapy model offers hope for X-linked sideroblastic anemia treatment
Castruccio Castracani and his team developed a lentiviral vector that activated the human ALAS2 gene in erythroid cells, or ...
Insmed’s gene therapy poised to challenge DMD landscape after IND clearance
Currently, the DMD treatment landscape includes Sarepta’s Elevidys (delandistrogene moxeparvovec), the first FDA-approved ...
Medical Xpress on MSN13d
Study highlights need for standardized measurement methods in gene therapy
In gene therapy, a person's faulty genes are either replaced or ... which surprised the researchers since SV-AUC is ...
The American Journal of Managed Care7d
Standardized Measurements Needed for AAVs in Gene Therapy, Study Finds
Common measurements for modified adeno-associated viruses (AAVs), a gene therapy delivery method, showed substantial ...
FierceBiotech5d
RegenXBio sells Hunter syndrome gene therapy's U.S., Asia commercial rights for $110M upfront
With an FDA approval submission for RegenXBio’s Hunter syndrome gene therapy already underway, the biopharma has now ...
The American Journal of Managed Care15d
Gene Therapy Demonstrates High Efficacy in Severe β-Thalassemia
A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...