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 ...

SEATTLE — Some of our most challenging health conditions result from genetic disorders like sickle cell disease and certain cancers. Gene therapy is revolutionizing how they're treated, and the Fred ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their earliest stages. Today, we can intervene in the first days or weeks of life.

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 ...

In the spring of 2024, the US Food and Drug Administration (FDA) approved fidanacogene elaparvovec (Beqvez), only the second gene therapy product for hemophilia B. Just a few months later, the Pfizer ...

Onasemnogene abeparvovec gene therapy post-nusinersen or risdiplam showed meaningful motor improvements in children with SMA, with a manageable safety profile. The study cohort included older, heavier ...

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

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