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Vineti 8h
Excited to connect with leaders at next week’s ’s Meeting on the Med, where our CEO is a guest speaker. DM or contact us via the partnering system to meet our team!
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Andrew Tomlinson Apr 16
Complete vindication of interim data from Phase 3 STR1VE trial of Zolgensma (AVXS-101) in spinal muscular atrophy (SMA) Type 1 show prolonged event-free survival, early & rapid increase in CHOP-INTEND scores, signf milestone achievement
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Kate Buckland Apr 4
Wow... gene therapy for PKU entering clinical trials. I'll admit, I've shed a few tears. Never thought I'd see this in my lifetime. I know it's still far away, but for me it gives hope.
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Dr. Nicole Paulk 4h
Impressive not only for the final successful results today, but impressive for having the vision and sticking with it even when wasn't popular.
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NC State BTEC Apr 18
BTEC provided leadership for a one-day Cell and Gene Therapy Symposium and Vendor Show April 11, 2019, at the NC Biotechnology Center in Research Triangle Park, N.C.
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Dr. Nicole Paulk Apr 15
Excited to see old friends and meet new ones as well. Don't be bashful y'all, come introduce yourself I'd love to meet you and learn about your research 🤓
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AGTC 9h
is an exciting process. Learn about the science behind gene therapy & AGTC’s clinical trials at . has 17+ years of experience designing & constructing critical gene therapy elements in order to develop innovative treatments for patients.
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Sarepta Therapeutics Apr 8
Our clinical data has been accepted for late-breaking oral presentation at the 2019 Clinical Conference, where we’ll also present 6 posters highlighting data from our and programs for and . Learn more:
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Jonathan Hoggatt Apr 2
The is looking for postdocs interested in , , bone marrow transplant, and related fields. Please retweet and spread the word, and if you are a PI with a graduating student, please consider referring them to our team!
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#RecombEU Apr 9
’s annual consortium meeting has just started in the beautiful city of Leiden. We are excited to welcome all project partners and looking forward to interesting discussions related to our project on .
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Amy Board Apr 16
Update on Sangamo process. Clear reporting on steroid need in several patients as well.
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GEN 10h
Cures Babies with “Bubble Boy” Disease:
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Sangamo Therapeutics Apr 9
patients have a genetic mutation that results in missing or defective Factor VIII, a blood clotting protein. Our investigational is designed to deliver a functioning gene into cells that enables the body to produce the clotting protein on its own.
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Cell and Gene Therapy Catapult Apr 18
CBO Matthew Durdy: “A lot of discussion about value is stalled by inability to adapt to multi-year budgeting in health and social care.” A crucial challenge that must be overcome to ensure adoption of gene therapies in the NHS -
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ASGCT Apr 4
More news on yesterday's update regarding the Phase I/II clinical trial to treat hemophilia A. Safety monitoring committee recommends up to 5 more patients be enrolled in maximum-dose cohort based on high-dose Factor VIII results.
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Vineti 9h
As the number of in grows (1K+), now is the time to standardize processes on behalf of . A key SF Cancer Summit theme with and Tim Moore – thank you for a great event!
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ASGCT 8h
, and are different fields with a similar goal: To treat disease by changing our bodies at a microscopic level. What do these approaches have in common, and what makes them different?
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Cell Culture 8h
Have you listened to Jacqueline Barry from ’s webinar titled ‘Regulatory Strategies to implement your manufacturing process?’ No? Go ahead and Download it here -
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Fujifilm Healthcare 7h
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NCATS 7h
We hate to burst your bubble…actually, we’re more than happy to do it! A new , supported in part by NCATS’ program, corrects the that causes severe combined immunodeficiency disease ().
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