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1. Gene Therapy
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8. Cost of Gene Therapy
The cost of these treatments, though, ranges from
about $500,000 to $1.5m. And over a lifetime,
drugs like nusinersen can be even more
expensive: $750,000 in the first year followed
by $375,000 a year after that – for life. As these
prices suggest, it's expensive to get a gene therapy
drug to the market
Cell and gene therapies are
designed to halt a disease in
its tracks or reverse its
progress rather than simply
manage symptoms. These
are often one-time
treatments that may
alleviate the underlying
cause of a disease, and they
have the potential
to cure certain conditions.
Currently, the only way for you to receive gene therapy is to
participate in a clinical trial. Clinical trials are research
studies that help doctors determine whether a gene
therapy approach is safe for people.

9. Companies in the frontline
• Spark Therapeutics Inc. (NASDAQ: ONCE) is a commercial gene therapy company
that specializes in treatment for inherited retinal diseases, neurodegenerative diseases
and diseases that can be treated through the liver
• Dyadic International Inc. is a biotech company that focuses on finding applications for a fungus-
based technology…The technology is used in a wide range of treatments, including vaccines,
antibodies and enzyme therapies.
• TrovaGene Inc. is an oncology therapeutics company developing drugs that will affect cell
division to tackle the growth of cancers.
• Novartis AG is working on gene therapies using adeno-associated viruses (AAVs) to deliver the
treatment. The company uses innovative science and technology to address some of society's
most challenging healthcare issues

10. 5 most expensive gene therapy drugs that have been
approved today and their eligible patient populations

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