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CheckRare Jun 10
Mark Pykett, PhD, Chief Scientific Officer at PTC Therapeutics, provides an overview of his company, including its focus on rare diseases.
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Ahdab AlSaieedi, PhD Jun 10
, as a new guide molecule, might become part of the toolbox that can allow scientists to control where the DNA inserts itself in the genome.
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Nature Medicine Jun 7
Replying to @NatureMedicine
Read our second part in this series: The history of antisense oligonucleotides goes back more than four decades... but the regulatory challenges of customizing this drug therapy might just be beginning.
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24x7esubmission Jun 15
Likely to be the Next Non-Invasive Treatment | Market Research Blog
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Volker Schulz Jun 14
. Why gene therapies are already changing our species .
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Michael Hughes 11h
Pleasure to attend the 6th INPDA Face to Face meeting in Lyon 🇫🇷. Amazing to meet Niemann-Pick organisations from all around the world and present an update on our NP-C gene therapy work.
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Concilium Search 1h
Gene therapies seize the top of the list of the most expensive drugs on the planet.  
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Frank Vinluan Jun 14
Replying to @frankvinluan
The price of works out to about $357,567 a year for five years, and bluebird says in addition to a multi-year payment plan, it will also work out "outcomes-based agreements" that tie payment to the 's performance. If it doesn't work, you don't pay.
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Cryoport, Inc. Jun 14
WATCH: Transporting life-saving medicines around the world requires serious attention to detail. We created the Chain of Compliance™️ to make certain the newest advancements in and reach patients safely and securely. Learn more:
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Tamara Mathias Jun 14
Bluebird bio Inc on Friday set a price for its gene therapy, Zynteglo, at 1.575 million euros ($1.78 million) over five years, after winning conditional approval in Europe this month to treat a rare genetic blood disorder.
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Marty Chilberg Jun 13
Some good insights from Evercore in their series of reports on Biotech 4.0 IPOs are leading indicator IPOs are earlier stage lead assets 1)
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BCBSTX Jun 15
Do you know someone who could benefit from ? A pharmacist joins us for a episode discussing the high price tags that come with medical breakthroughs. Watch the video recording of this .
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Akron Biotech Jun 11
While Vertex and do their best to repair genes, a from Sarepta Therapeutics is gaining steam. Here are the arguments for both methods.
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Invest Grup Bilişim Hizm.Ltd.Sti / Invest Group Co Jun 11
A Grand Premium domain for sale at a way too low price now at sedo..!
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Monica Robinson Jun 14
It works. It's expensive. The costs will come down, and it's worth it already.
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Cell and Gene Therapy Catapult Jun 13
. to invest $420m to develop its position in -
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Jessica Pacheco Jun 14
From bio's CEO Nick Leschly "The rising cost of new treatments will prompt the industry to re-examine how it charges for drugs."
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Richard Kuehn 16h
can treat what were thought to be incurable diseases, but at quite a cost.
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GE Healthcare Life Sciences Jun 14
and collaborate to drive commercialization of cell and gene therapies. - A collaboration connecting critical manufacturing processes and logistics technology with reliable time- and temperature-sensitive transport.
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Tobias P Dreesman 18h
Is $1.8 Million for bio's new ridiculous? Following European approval earlier this month, bluebird bio announced its gene therapy will launch with a sky-high price tag.
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