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 ...

Investing.com -- Shares of Ocugen, Inc. (NASDAQ: OCGN) climbed 11.6% following the announcement of a licensing agreement with a leading Korean pharmaceutical company for the rights to OCU400, its gene ...

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

With FDA platform technology designation for its viral vector, SRPT can fast-track other therapies using the same ...

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 ...