VG801 is a dual AAV gene therapy that leverages mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the ...

Panelists discuss how splice modifiers work by enhancing protein production from the SMN2 gene, with risdiplam (Evrysdi) ...

Baby KJ Muldoon, the first patient to successfully receive personalized CRISPR gene editing therapy has returned home after over 300 days at the hospital.

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's disease ...

OCU400 is Ocugen’s novel modifier gene therapy for retinitis pigmentosa (RP). The OCU400 (AAV5-h NR2E3) gene therapy platform ...

Screening can now determine their risk for an ever-growing list of conditions — including ones we can’t do much about.

Researchers at the University of California San Diego School of Medicine have developed a gene therapy for Alzheimer's ...

Analysts reacted positively to the news that uniQure is in alignment with the FDA on an accelerated approval pathway and on ...

The former workers cited various reasons why they think Novo was cautious: its conservative culture and history of investing ...

The Platform Technology Designation, which precedes the current FDA leadership, is designed to streamline the drug ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...