$NTLA News: We are pleased to announce that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to nexiguran ziclumeran (nex-z, also known as NTLA-2001) for the treatment of #ATTR #amyloidosis with polyneuropathy: https://lnkd.in/e7Me6DtA #CRISPR #GeneEditing
Intellia Therapeutics, Inc.
生物技术研究
Cambridge，Massachusetts 58,574 位关注者
Developing curative genome editing treatments
关于我们
Intellia Therapeutics is a leading genome editing company whose mission is to develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases. We are focused on the development of proprietary therapeutics using a recently developed biological tool known as the CRISPR/Cas9 system. The promise of the CRISPR/Cas9 system is the driving force behind the creation of Intellia. Our founders have a shared belief that the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. This technology offers the potential for us to develop curative therapeutic options for patients with chronic diseases by addressing the underlying cause of the disease. It can bring new hope and cures to people who now have nowhere to turn for help. Our combination of deep scientific expertise and clinical development experience, along with our leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Intellia has the right people, assets and ambitious vision needed to take full advantage of the CRISPR/Cas9 technology’s attributes – high potency, specificity, simplicity of use, broad applicability, and multifunctional programmability – and accelerate the advancement of curative products into the clinic. View our social media community guidelines at https://www.intelliatx.com/terms-and-conditions/ .
- 网站
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https://www.intelliatx.com
Intellia Therapeutics, Inc.的外部链接
- 所属行业
- 生物技术研究
- 规模
- 501-1,000 人
- 总部
- Cambridge，Massachusetts
- 类型
- 上市公司
- 创立
- 2014
地点
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主要
获取路线
40 Erie Street
US，Massachusetts，Cambridge，02139
Intellia Therapeutics, Inc.员工
动态
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It’s #CRISPRlingo time! In our last chapter, we spotlighted in vivo therapies; today we’re learning about their counterpart, ex vivo therapies. With an ex vivo therapy, cells are removed from the body and then modified or repaired. This type of editing most often involves blood cells. Once modified, the engineered cells are administered back to the patient. Find out more about how Intellia harnesses ex vivo therapies: https://bit.ly/3LhSspB #CRISPR #ExVivoTherapy
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Our Chief Technology Officer, Eliana Clark, Ph.D., will present on #invivo #geneediting at the Biologics Manufacturing World Summit on Nov. 20. More here: https://lnkd.in/e-DyS_QA #CRISPR #GeneEditing #BMWS20
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Intellia Therapeutics, Inc.转发了
We’re thrilled to announce that the Cystic Fibrosis Foundation has agreed to invest up to $15 million in additional funding to accelerate our gene correction research program, developed in collaboration with Intellia Therapeutics, Inc. This support advances our work, combining our innovative SORT LNP delivery platform with Intellia’s cutting-edge CRISPR DNA-writing technology. This funding will help develop up to three unique gene correction programs, bringing hope for transformative new therapies. Together, we’re tackling the root genetic cause of cystic fibrosis (CF), initially focusing on mutations current treatments cannot adequately address. We’re proud to continue our partnership with the CF Foundation, building on their support of our inhaled mRNA therapy, RCT2100, which is now in a Phase 1b trial. Together, we are working to create new therapeutic solutions for everyone affected by this genetic disease and ensure no one is left behind. Read the full release here: https://bit.ly/48V3PPi #CysticFibrosis #Biotech #ReCode #GeneticMedicine #CRISPR
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Excited to wrap up Day 2 at the American Heart Association Scientific Sessions! Here’s a snapshot of our dedicated team members, Jessica Kachadourian, David Lebwohl, Ricardo Rocha, MD, FAHA, John Leonard and Maria Natale, taking a quick moment to connect at the Intellia booth.??#AHA2024 #OneIntellia
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#ATTR News: $NTLA announces positive new clinical data from the ongoing Phase 1 trial of nexiguran ziclumeran (nex-z) in patients with #ATTR amyloidosis with cardiomyopathy. Full details here: https://lnkd.in/eTQAP6-E. Join us at 12 p.m. ET for a webcast discussing the data, as well as an update from the ongoing Phase 1 polyneuropathy cohort. https://lnkd.in/eTF4j9uq #CRISPR #GeneEditing
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Intellia is gearing up for the American Heart Association Scientific Sessions in Chicago from Nov. 16-18. Join our Late-Breaking Science Session, where we’ll present clinical data on nexiguran ziclumeran (aka NTLA-2001) for #ATTR #amyloidosis with cardiomyopathy. And don't miss our sponsored symposium, featuring leading experts in the field. Swing by our booth to learn even more! #AHA2024 #GeneEditing https://lnkd.in/dVZSYuq
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The next chapter of our #CRISPRlingo series offers a lesson in Latin.?? ? In vivo translates to “within the living,” and, in the context of gene editing, it refers to a targeted approach for editing??genes within a patient’s body. Investigational in vivo #CRISPR therapies are typically delivered intravenously and designed to target specific cell types. Learn more: https://bit.ly/3LhSspB #InVivoTherapy To learn about ex vivo therapies, check out the next installment of our series. Scientia potentia est!
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We’re grateful to Intellia co-founders Rodolphe Barrangou, Luciano Marraffini and Erik Sontheimer for joining us at our 8th Annual Symposium. Thank you for your continued support and inspiration! #OneIntellia #CRISPR
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We’re thrilled to announce Intellia has been recognized on BioSpace’s 2025 Best Places to Work list for small employers! ?? A huge thank you to our dedicated employees for making Intellia an exceptional place to work. Here’s to pushing boundaries and shaping the future of #geneediting together! #OneIntellia https://lnkd.in/e5AC_YG7