Tag: gene therapy

FDA Approves World’s Most Expensive Drug at $3.5 Million Per Dose to Treat Hemophilia

The U.S. Food and Drug Administration (FDA) has approved CSL Behring’s hemophilia B gene therapy, a one-off infusion treatment with a list price of $3.5 million, making it the world’s most expensive medicine. The approval of CSL Behring’s Hemgenix “provides a new treatment option for patients with Hemophilia B and represents important progress in the…


World’s-First Gene Therapy Developed for Type 1 Diabetes

The Australian government has given a funding boost to an Australian biomedical research organisation, the Garvan Institute of Medical Research, to conduct clinical trials for the world’s first gene therapy for type 1 diabetes. The therapy was also developed thanks to the government’s ten-year $600 million (US$385 million) research accelerator program, targeted at addressing chronic and complex diseases….


Salivary Gland Gene Therapy Shows Promise

An experimental trial showed that gene therapy can be performed safely in the human salivary gland. The accomplishment may lead to treatments to help head and neck cancer survivors who battle chronic dry mouth. People with head and neck cancer often receive radiation therapy to shrink their tumors. The radiation can damage salivary glands, which…


FDA Approves Bluebird Bio’s Gene Therapy for a Rare Neurological Disorder

The U.S. Food and Drug Administration (FDA) has approved Bluebird bio’s gene therapy for the treatment of a rare neurological disorder, the company said late on Friday. “SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys” with early, active Cerebral Adrenoleukodystrophy (CALD), the company said in a…


Bluebird’s $2.8 Million Gene Therapy Becomes Most Expensive Drug After US Approval

The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million. The approval sent the company’s shares 8 percent higher and is for the treatment of beta-thalassemia, which causes an oxygen shortage in…


COVID-19 ‘Vaccines’ Are Gene Therapy

Not a vaccine in the medical definition, the COVID-19 ‘vaccine’ is really an experimental gene therapy that does not render immunity or prevent infection or transmission of the disease. STORY AT-A-GLANCE mRNA “vaccines” created by Moderna and Pfizer are gene therapies. They fulfill all the definitions of gene therapy and none of the definitions for…


CRISPR Gene Editing Finds Possible Therapy to Sickle Cell Anaemia

Researchers from the University of New South Wales (UNSW) have found the mechanism behind sickle cell anaemia and beta thalassemia—common genetic anaemia—through CRISPR (DNA editing) techniques, with the discovery promising for future therapies. “We can use this understanding of the mechanism to help us look for new therapeutic approaches–it’s a key piece of the puzzle,”…


COVID-19 Vaccines — A Case of False Advertising

If the US government would call the COVID-19 ‘vaccines’ by what they really are, far fewer people would be lining up for their two shots. Did you know that mRNA COVID-19 vaccines aren’t vaccines in the medical and legal definition of a vaccine? They do not prevent you from getting the infection, nor do they…


Bayer Executive Says mRNA Vaccines are Gene Therapy

A speech made last year by a Bayer executive mentioning that messenger RNA (mRNA) vaccines are gene therapy is gaining traction on social media for the second time since it was initially posted online in October 2021. Stefan Oelrich, a member of the Board of Management of Bayer and head of the Pharmaceuticals Division, gave…