A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...

Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

A gene therapy that patients breathe in has been found to shrink lung tumours by inserting immune-boosting genes into ...

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...

Recent therapies like hydroxyurea (Droxia, Bristol-Myers Squibb), the first FDA-approved drug for SCD in 1994, increase fetal hemoglobin (HbF), which, through unknown pathways, improves SCD symptoms ...

The Greater Philadelphia Business Coalition on Health webinar series provided an in-depth framework for the advantages of including cell and gene therapy coverage in employer health plans, as well as ...

After a series of deaths in patients taking Sarepta Therapeutics’ gene therapies, doubt has crept into investor sentiments ...

PDE6A-associated RP, a rare form of inherited retinal disease (IRD), is characterized by nyctalopia, visual field defects, and significant loss of visual acuity (VA). The disease primarily affects the ...

After facing a delayed decision deadline and a clinical hold, Regenxbio’s Hunter syndrome gene therapy has been rejected by ...