Web Results

pubmed.ncbi.nlm.nih.gov/31887345

Consequently, this review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene therapy drugs, etc. Furthermore, the gene therapy drugs were classified and addressed in accordance with the employed ...

geneticliteracyproject.org/2020/02/12/900-gene-therapy-drugs-are-in-the...

To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced ...

pubmed.ncbi.nlm.nih.gov/32014447

Tisagenlecleucel was the first gene therapy to receive approval from the FDA for any indication. The initial approval was for relapsed or refractory (r/r) pediatric and young-adult B-cell acute lymphoblastic leukemia in August 2017 and in May 2018 for adult r/r diffuse large B-cell lymphoma.

www.genengnews.com/a-lists/25-up-and-coming-gene-therapies-of-2019

As for approvals of new gene therapies, two candidates considered close to approvals appear on this list. One is Zynteglo™ (formerly LentiGlobin™), a bluebird bio gene therapy for thalassemia ...

www.washingtonpost.com/business/economy/the-fda-approves-a-gene-therapy-that...

The Food and Drug Administration on Friday approved the most expensive drug in the world, a $2.1 million one-time gene therapy for treatment of a rare disease that afflicts infants.

www.cnbc.com/2019/05/24/fda-approves-novartis-2-million-spinal-muscular...

The Food and Drug Administration on Friday approved Novartis' $2.1 million gene therapy for spinal muscular atrophy — making it the world's most expensive drug. The therapy, Zolgensma, is a one ...

www.npr.org/sections/health-shots/2020/07/20/800556057/gene-therapy-shows...

The first gene therapy for hemophilia could be approved by the FDA within six months, according to the drugmaker, raising hopes among families. But the drug's price could be $3 million per patient.

www.inquirer.com/health/oxbryta-adakveo-approved-fda-sickle-cell-disease...

The new drugs come as dozens of experimental therapies are in development, including some genetically engineered treatments that may offer a cure by repairing hemoglobin’s ability to carry oxygen. Early this year, for example, the FDA gave “fast track designation” to Vertex Pharmaceuticals’ gene-edited stem cell therapy for the disease.

www.drugs.com/history/roctavian.html

Roctavian FDA Approval Status. FDA Approved: No Brand name: Roctavian Generic name: valoctocogene roxaparvovec Company: BioMarin Pharmaceutical Inc. Treatment for: Hemophilia A Roctavian (valoctocogene roxaparvovec) is an investigational gene therapy in development for the treatment of hemophilia A.

www.cnn.com/2017/12/20/health/fda-gene-therapy-blindness-bn

The FDA has approved Luxtruna for patients with a rare inherited eye disease. It's the third gene therapy to win US approval, the first for a genetic disease.