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www.healthline.com/health-news/gene-therapy-drugs-in-2018

What to Expect from Gene Therapy Drugs in 2018 Written by Shawn Radcliffe — Updated on September 24, 2018 A new treatment for blindness has brought gene therapy into the real world, but it comes ...

www.msn.com/en-us/health/medical/gene-therapies-what-to-know/ar-BB1bm50q

The therapy Israel received, called lisocabtagene maraleucel, or liso-cel, is among a wave of new gene and cell therapies that are under development to treat cancer, blindness, and a range of ...

fee.org/articles/why-this-new-gene-therapy-drug-costs-21-million

Gene Therapy. A new gene therapy drug named Zoglensma became the most expensive drug in the world, costing patients over $2.1 million for one-time use. Zoglensma joins a small (and outlandishly expensive) group of treatments called gene therapy drugs. Gene therapy uses actual genes to treat or prevent diseases.

sicklecellanemianews.com/2020/01/23/fda-names-aru-1801-aruvant-gene-therapy...

A potential gene therapy for sickle cell disease (SCD) and beta-thalassemia, known as ARU-1801, has been named an orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development.. The FDA recently also designated ARU-1801 a potential rare pediatric disease treatment for these same two blood disorders, allowing for priority review.

www.zacks.com/stock/news/1107292/selectas-selb-gene-therapy-gets-orphan-drug...

The FDA bestows an orphan drug status on Selecta's (SELB) gene therapy candidate MMA-101 for the treatment of isolated methylmalonic acidemia. The candidate already has a Rare Pediatric Disease tag.

www.scientificamerican.com/article/gene-therapy-arrives

After decades of disputed results, treatment failures and some deaths in experimental trials, the first gene therapy drug, for a type of skin cancer, was approved in China in 2003. The rest of the ...

www.biopharmadive.com/news/fda-gene-therapy-manufacturing-delays-scrutiny/588382

One sign of increasing scrutiny is delays involving cell and gene therapy manufacturing. Over the past two months, Sarepta Therapeutics, Voyager Therapeutics, Iovance Biotherapeutics and, most recently, Bluebird bio, have been forced to reset development timelines as the FDA has requested more information about production processes. "It's in part due to the general evolution of the field ...

www.cellandgene.com/doc/how-are-cell-and-gene-therapies-changing-drug...

The biotechnology sector has seen important progress in the development of groundbreaking therapies in recent years, especially in the areas of immunotherapy and cell and gene therapy. In gene therapy alone, there are now clinical research programs targeting almost 50 different indications, up from only 10 a few years ago, 1 and the rate of ...

www.avexis.com

Novartis Gene Therapies is dedicated to developing and commercializing gene therapies for patients and families devastated by rare and life-threatening neurological genetic diseases. COOKIE NOTICE This site uses cookies to store information on your computer.

www.youtube.com/watch?v=GbJasFgJkLg

Gene therapy is the process of replacing defective genes with healthy ones, adding new genes to help the body fight or treat disease, or deactivating problem...