Leaders from the world of cell and gene therapy shared their personal stories and policy recommendations in an extraordinary roundtable conducted by the FDA in front of its recently-appointed top ...

Can a small fish help identify possible treatments for an ultra-rare inherited disease found in an Alabama boy? The genetic ...

By reprogramming brain cells, a new gene therapy approach for Alzheimer's developed by UC San Diego researchers could address ...

While heritable human genome editing is banned in the United States, across Europe and much of the world, the technology ...

Scientists have made a key breakthrough in treating respiratory diseases by developing a new drug delivery system that ...

Congress, convening virtually and in Milan, Italy, from June 12 to June 15, 2025, will feature a revamped program structure ...

The partners further aim to raise awareness and increase early diagnosis of BAG3-associated dilated cardiomyopathy.

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

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

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 ...

XMEA is a recessive, genetic disease that causes progressive muscle weakness. Can a small fish help identify possible ...