Moreover, two pivotal trials have shown that combining Kalydeco ... feel he is leaving the company in fine shape. Vertex's gene-editing therapy for sickle cell disease, Casgevy, has been given ...

NHS England has agreed a deal with Vertex for its three-drug cystic fibrosis therapy Kaftrio ... 12 or over who have two copies of the F508del mutation gene, or one copy of F508del and a minimal ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

United Therapeutics said the FDA has cleared it to begin the first clinical trial testing whether organs from gene-edited ...

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first-of-its-kind genetic therapy for glioblastoma, a severe form of brain ...

Silver Spring’s United Therapeutics has scored a regulatory green light to start clinical trials for its genetically modified pig kidneys to be transplanted into humans. It's as significant for the ...

FDA 2025 pulmonology drug alerts, including drug approvals/warnings, vaccine approvals, and medical device recalls/updates.

Gene therapy using modified blood stem cells helped five adults with severe hemophilia A reach therapeutic levels of clotting ...

Thirty patients have received the gene therapy within a clinical trial and early-access program, and the foundation also hopes to seek approval in the US.

Copy number variants (CNVs) arise by homologous recombination (HR) between repeated sequences (recurrent CNVs) or by non-homologous recombination mechanisms that occur throughout the genome (non ...

In this manuscript, we give an overview of historical aspects, as well as the current state of clinical trials and approved gene therapy products for commercialization in Brazil. Focusing on gene ...