Liver-directed AAV-based gene therapy offers promise for people living with hemophilia B or other genetic disorders. 6,7,11–18 However, the long-term safety and efficacy of this approach remain ...

Sarepta Therapeutics (NASDAQ:SRPT) stock draws an Overweight rating at Wells Fargo, based on commercial prospects for ...

Dr Tejinder Kataria, Chairperson, Radiation Oncology, Cancer Care, Medanta, Gurugram, highlights the benefits of gene therapy ...

Premium InsightsThe Hepatitis C drug market is entering a transformative phase as global health organizations, pharmaceutical ...

Despite the challenges, including what CSL CEO Paul McKenzie has described as a fragmented U.S. healthcare system, the blood ...

Ultragenyx's strong revenue growth, diverse streams, and late-stage pipeline make it a standout in rare disease and gene ...

Researchers at Children's Hospital of Philadelphia (CHOP) in collaboration with researchers at Royal Prince Alfred Hospital in Sydney, Australia announced the results of the longest follow-up study to ...

The two deaths occurred in children in Russia and Kazakhstan, with the liver failure starting five to six weeks after administration of the adeno-associated virus-based treatment. Both children ...

Belief BioMed (BBM) and Takeda China have announced the receipt of China’s NMPA approval for BBM-H901for treating haemophilia ...

This is the first hemophilia B gene therapy drug and the first recombinant adeno-associated virus (rAAV) vector gene therapy drug ... can deliver the optimized human coagulation FIX gene into the ...

The tragic death of the 16-year-old boy came as a result of acute liver failure ... labelling and is thought to be linked to the adeno-associated virus (AAV) used to deliver the genetic material.

Belief BioMed and Takeda China today jointly announced that BBM-H901, has been officially approved by the National Medical ...