Remove tag stem
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

Spark Therapeutics’ Luxturna, indicated for inherited retinal disease (IRD), was the first gene therapy to be approved, in 2017, with a price tag of $850,000 for each eye. Prior to bluebird's approvals, there were only two FDA-approved gene therapies for inherited conditions on the market.

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CHMP backs J&J’s myeloma bispecific Tecvayli, ahead of US decision

pharmaphorum

The new drug is one of two bispecifics for multiple myeloma in the late-stage pipeline of J&J’s Janssen division stemming from its longstanding and fertile collaboration with Danish biotech Genmab, along with talquetamab which binds to CD3 and GPRC5D.

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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. While stem cell transplants can cure the condition, their use is limited due to some of the risks that come with them.

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Do healthcare mergers and acquisitions lead to better care for patients?

Clarify Health

This increase can be attributed to many things, including an aging population, the increasing prevalence of chronic conditions, and greater access to care stemming from expanded insurance coverage. With higher price tags, the incentive for quality improvement is lower, setting back efforts to drive value-based care.

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Weekly Roundup – Nov 9, 2018

CloserIQ

” The Ultimate Guide to Gifting STEM toys: Tons of Ideas for Little builders (TechCrunch). “There are a few devices in the market that are essentially fully fledged computers — but with a child-friendly layer applied to hand-hold and gamify STEM learning.” ” What happens to employees after an acquisition?

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bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

pharmaphorum

Zynteglo is a customised, one-time treatment created using a patient’s own bone marrow stem cells hat are genetically modified to produce functional beta-globin, which is mutated in the disease. million price tag attached to the therapy when it was first made available in Europe, and is also well above the proposed $2.1

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Q&A: A decade on, what’s next for CAR-T therapies?

Pharmaceutical Technology

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. Currently, all approved CAR-T therapies are autologous, where T cells taken from patients are modified and then re-infused.