2019: the year gene therapy came of age
In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.
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gene therapy
Blackstone to invest $400 million in gene therapy for bladder cancer
Ferring will invest $170 million in the joint venture with Blackstone, dubbed FerGene, bringing its total funding to $570 million
Second death in gene therapy trials under investigation
Novartis AG says investigation is underway into whether a second trial death could be related to the treatment
US approves first gene therapy for cancer
MIAMI: The United States approved the first gene therapy in the nation on Wednesday -- a treatment that uses a patient's own immune cells to fight leukemia -- opening a new era in the fight against cancer.
The treatment is made by Novartis and is called Kymriah, (tisagenlecleucel). This type of immunotherapy, known as a CAR-T cell therapy, was known by CTL019 until now.
It was approved by the US Food and Drug Administration for children and young adult patients up to age 25 with a form of acute lymphoblastic leukemia…