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Jonathan Lasko, 31, holds the hand of his son Max, age 3.5, who was born with spinal muscular atrophy. A new one-time treatment was just approved for the disease, with a $2.1 million price tag.

The US just approved a $2.1 million treatment for a devastating disease. It’s the most expensive drug in the world.

The treatment, called Zolgensma, should last longer than a typical drug. It also has a much higher price tag.
Iora Health CEO Rushika Fernandopulle made our list of 10 people transforming healthcare.

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A new experimental gene therapy has cured babies born with a genetic disease dubbed "bubble boy" syndrome.

There’s now a cure for the deadly genetic disorder known as ‘bubble-boy’ disease

Doctors used a cutting-edge medical technique called gene therapy to treat 10 babies born with the disease. The biotech that helped saw its stock surge.
Human genetic material is stored at a laboratory in Munich.

Big pharma’s big bets, a hedge-fund rivalry, and Silicon Valley’s obsession with failing forward

Gene therapy, a decades-old technology that targets disease at its genetic root, is coming of age. New startups are pouring into the space, and it's become a hot area for M&A.

The top scientist at AveXis told us the ‘unique’ strength that powered its $9 billion sale to Novartis, and it could shape the future of g...

Manufacturing has emerged as a huge challenge for the booming gene therapy industry because these products are so new and complex.
Regulators at the FDA will hold a meeting in April on CBD.

Dispensed: Layoffs at Clover Health, a troubling marijuana-linked syndrome, and what’s ahead for the exploding gene therapy field

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Dispensed: Change at the FDA, insurance startup financials for 2018, and the first new depression drug in 35 years

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Carolyn Barrett, who was born with the rare disease spinal muscular atrophy, and her service dog Shadow head down to the Boston Common in February of 2017. A new gene therapy for spinal muscular atrophy could be approved this year, and may cost up to $5 million for a one-time treatment.

Drugs that cost as much as a house are on the way to treat rare and devastating diseases. The US is scrambling to figure out how to pay for them.

The cost of gene therapies will put a huge financial strain on the US health system as new products become available to treat more common diseases.

Swiss drug giant Roche reportedly readies to buy the biotech behind the first FDA-approved gene therapy and the priciest medicine in the US

Roche could pay $5 billion for biotech company Spark Therapeutics. The pharma giant likely wants to expand its presence in hemophilia, a lucrative market.

A revolutionary drug that could treat a rare and devastating disease is prohibitively expensive. But one state has a plan to pay for its potential $5 ...

A one-time treatment for a devastating rare disease could be paid for with an installment plan in Massachusetts. That could set a roadmap for the US.