Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Jefferies, BofA Securities, Piper Sandler and Barclays are acting as joint book-running managers for the proposed offering.

Qure divested for $25M, advanced AMT-130 with FDA approvals, and building infrastructure. A strong balance sheet backs ...

Wilson's new company, Philadelphia-based Gemma Biotherapeutics, plans to start testing gene therapies in Brazil this year.

Genetic defects are exceedingly common, which is not surprising considering just how many cells make up our bodies, including our reproductive cells. While most of these defects have no or only ...

Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and ...

A patient has died in a pivotal Phase II trial of Rocket Pharmaceuticals’ gene therapy for Danon disease after suffering a ...

Scientists engineered a way to deliver genes directly to lung and airway cells using a nasal spray * AAV.CCP.16 -// a viral ...

The virus-producing cells were incubated at 32°C in a 5% CO ... BaEV-enveloped viral vectors have garnered increasing attention in cell and gene therapy. However, in the majority of current studies on ...

For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body -- a job commonly given to adeno-associated viruses (AAV). To improve the AAV ...