Gene therapy has been available since 2022 for hemophilia B and since 2023 for hemophilia A, yet some major medical institutions have barely treated any patients so far. What’s the holdup?

In this article, we will discuss the 10 Best Gene Therapy Stocks to Buy Right Now. Gene therapy is referred to as a medical technique that involves modifying or manipulating genes to treat or ...

The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.

"Our study shows that we can deliver genes to renal tubules and podocytes [highly specialized cells] in the kidney, the two important target cell types for gene therapy, and while there is a ...

uniQure says it has reached an agreement with the FDA on a pathway to accelerated approval for its Huntington's disease gene therapy based on a phase 1/2 trial. The announcement led to a doubling ...

Gene therapy involves the introduction of new genes into cells, to restore or add gene expression, for the purpose of treating disease. Most commonly a mutated gene is replaced with DNA encoding a ...

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of ...

In a finding with implications for one of the most promising cutting-edge medical treatments, researchers evaluated several measurement techniques commonly used in gene therapy. The study ...

Pfizer gave Sangamo Therapeutics coal for Christmas, terminating a hemophilia A gene therapy pact to deprive the cash-strapped biotech of up to $220 million in milestones. Pfizer’s decision sent ...

Some gene therapy treatments use modified adeno-associated viruses (AAVs) like the one shown here to deliver therapeutic genetic material into a patient’s cells. In a finding with implications ...

*Refers to the latest 2 years of omaha.com stories. Cancel anytime. A preview of Nebraska Medicine's Innovation Design Unit in December 2024 After more than four decades of infusing himself with ...