You Searched For "gene therapy" and got 20 results
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2 mins read
Scientists partially restored a blind man's vision using gene therapy and goggles
The first successful trial of a new optogenetics treatment let one man see blurry objects through s…
Andrea Michelson
2 mins read
IIT Kanpur licenses gene therapy to Reliance Life Sciences
The Indian Institute of Technology (IIT) in Kanpur has licensed a pioneering technology to Reliance…
2 mins read
The FDA just approved the first gene therapy for an inherited disease
"Today's approval marks another first in the field of gene therapy - both in how the therapy works …
Lydia Ramsey
4 mins read
The FDA's top official overseeing gene therapy had these 5 suggestions for startups flooding into the billion-dollar industry
Design research "very thoughtfully." The gold standard of science research is a type of trial that …
Emma Court
4 mins read
Swiss drug giant Roche reportedly readies to buy the biotech behind the first FDA-approved gene therapy, which just became the priciest medicine in the US
But developing the therapies and getting them to patients has proven a steep challenge. In addition…
Erin Brodwin
6 mins read
Pharma giants like Novartis, Pfizer, and Sanofi are getting serious about a cutting edge therapy that changes human genes
Recent M&A transactions show that big pharma is betting big on gene therapy. In April, Novartis…
Lydia Ramsey
3 mins read
Novartis just handed out a $8.7 billion endorsement for new therapies that are changing how we treat genetic diseases
Novartis isn't the only big pharma/biotech to be interested in gene and cell therapies. In January,…
Lydia Ramsey
4 mins read
The most expensive drug in the world has a $3.5 million price tag. 2 potential patients share their hopes and fears for the one-time treatment.
Patients with the blood disorder hemophilia told Insider they're excited about the new gene therapy…
Andrew Dunn
2 mins read
Kids with 'bubble boy' disease could finally get access to a life-saving gene therapy, as a study starts back up at UCLA
While families who have kids with the disease are desperate for the therapy, drug companies have be…
Andrew Dunn
4 mins read
The top scientist at AveXis told us about this surprising reason that it got bought for $9 billion by Novartis, and it could shape the future of gene therapy
Knudten's background was in pharmaceuticals manufacturing, though not specifically in the type of g…
Emma Court
2 mins read
A first-of-its-kind treatment for blindness just got one step closer to approval
In this Oct. 4, 2017 photo, Dr. Albert Maguire checks the eyes of Misa Kaabali, 8, at the Children'…
Lydia Ramsey
3 mins read
Many people with debilitating blood disorder hemophilia B may be cured within 3 years by new gene therapy, doctor said
The treatment is still in very early phases of development and a patient organization expressed cau…
Marianne Guenot
2 mins read
A deaf toddler can hear thanks to a world-first gene treatment that took just 16 minutes
After receiving treatment to correct a faulty gene, 18-month-old Opal Sandy can now hear.
Serafina Kenny
2 mins read
Gene-therapy biotech Bluebird Bio is set to lay off 30% of the company after warning it could struggle to survive through 2022
Insider identified Bluebird Bio last month as one of 13 biotechs in a dangerous financial position.
Andrew Dunn
1 min read
Sarepta Therapeutics spikes 57% after a key trial shows its experimental gene therapy worked in 3 patients
"I have been waiting my entire 49-year career to find a therapy that dramatically reduces CK levels…
Jonathan Garber,Lydia Ramsey
8 mins read
Million-dollar drugs are on the way to treat rare and devastating diseases. The US is scrambling to figure out how to pay for them.
Pharma giants are pouring billions into the promising space. In the last week alone, Swiss drug gia…
Emma Court
3 mins read
Top biotech executives say this is the 1 challenge that startups flooding into gene therapy could face
Gene therapies are incredibly complex medicines, and startups can't make assumptions about how thei…
Emma Court
4 mins read
Novartis just laid out the playbook for making its $2.1 million gene therapy treatment a blockbuster. Here are the next steps.
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Andrew Dunn
2 mins read
Pharma giant Roche just bought biotech firm Spark, the maker of an $850,000 eye drug
Spark announced earlier this month that although it had shipped 75 vials of its new drug and genera…
Callum Burroughs
1 min read
Here is the 10-slide deck a new biotech company addressing a critical gene-therapy bottleneck used to raise $55 million in just a few months.
Alexis Rovner had been looking into new ways of sequencing genes as part of her postdoctoral resear…