At the end of last year, Vertex/CRISPR and Bluebird received approvals on the same day for two different gene therapy products for sickle cell disease (SCD). Click the link below to download our most recent white paper and explore with our Cell and Gene Therapy experts, Subreen Khatib, Ph.D. and Sarah Jims, the approaches by each company to commercializing these complex technologies in this unique and quite interesting scenario. https://lnkd.in/eEhVZMjB #cellandgene #CGT
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Did you know that Veristat executed trials for the first gene therapy approved in Europe? With experience supporting over 100 cell and gene clinical trials, we have their strategy and execution down to a science. Download the case study 📩 https://bit.ly/48DfMXZ #ClinicalTrials #GeneTherapy
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Check out a story about the first #genetherapy approval in the EU.
Did you know that Veristat executed trials for the first gene therapy approved in Europe? With experience supporting over 100 cell and gene clinical trials, we have their strategy and execution down to a science. Download the case study 📩 https://bit.ly/48DfMXZ #ClinicalTrials #GeneTherapy
First Gene Therapy Approved in Europe
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Are you interested in learning more about: 🦠 Overcoming challenges in the industrialisation of iPSC-derived therapies? 🏥 UK patient referral pathways for gene therapy and CAR-T? 📝 How to develop a robust Target Product Profile (TPP) for your #ATMP? Take a look at our webinar recordings to hear from the experts at CGT Catapult addressing the key topics above and many more relating to the #cell and #gene therapy industry: https://buff.ly/3uE5HLS #CellTherapy #GeneTherapy #AdvancedTherapies #Innovation #Webinars
Webinar recordings
ct.catapult.org.uk
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Urgency... Perseverence... Determination.... Good things for all drug developers to keep in mind.... Fix this generation not just the next generation.... Are not rare diseases as the frontier for developing approaches to genetic disease --the most common of which may be Alzheimer's? We should all be cheering on the advances made--yes it's expensive, it's hard, it's "impossible" The first computers were the same way.... These are the pioneers finding out what could work for us all.... #GottaSolveThemAll #GottaSolveAllDisease #GeneticDiseaseIsOneTypeOfDisease #StillGreatIfYouCanSolveWithSmallMoleculesOrBiologics #TrikaftaApproachAmazingIfItWorks #DoNotKillThatOption #ButMayNotNeEasyOrStraightforword #GCLiveForeverClub #WeCanAllBeAPartOfIt #ScientistCanPlayASmallPartIn #HealingTheWorld #NoMoreDiseaseInducedMorbidityOrMortality #IsThisWhyThereIsOrWasSoMuchMarketExcitementInCrisprTechnologies #PublicSensesTheExcitementOfWhatCouldBePossible #DoesItMakeSenseAsAnInvestment #NotUnlessItWorks #ForNowThePOSMaySeemTooSmall #MechansimTooUnknown #WhatIfWeCanLive140Years #But70WithAlzheimers #NotGreat......
Urgency. Perseverance. Determination. Hear from FNIH Patient Ambassadors Sharon King and Terry Pirovolakis on their greatest take-home messages from #ASGCT2024. Learn more about how FNIH's Bespoke Gene Therapy Consortium is building bridges to breakthroughs for rare disease gene therapies: http://fnih.org/bgtc #BGTC #BridgestoBreakthroughs #genetherapy
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Learn about today’s top challenges in #CellAndGeneTherapy and how to solve them from Johan Hultman at this year’s #ESACT. Explore more about Sartorius cell and gene therapy solutions: https://sar.to/5t6ox #SimplifyingProgress #InThisTogether #AdvancedTherapies #ESACT2024
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Did you know expanded access programs can provide hope for patients who've exhausted all other options? Dive into our new article to understand the crucial role these programs play in cell and gene therapy development. https://lnkd.in/eqtxypnE Discover how they're accelerating innovation while prioritizing patient safety. #MedicalInnovation #RareDisease #ExpandedAccess #CellandGeneTherapy
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“Gene therapy is a bit of a blunderbuss approach,” Adrian Bird at The University of Edinburgh told Stephanie DeMarco, PhD, but gene therapies for Rett syndrome based on his and others' foundational research are in clinical trials now with preliminary efficacy results expected soon. Read more: https://ow.ly/flOp50UjtTr
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It was a pleasure to write this review for Cell & Gene Therapy Insights where I discuss approaches and considerations for generating in vivo genetic therapies. From the mechanism of action of the agent being delivered, to overcoming immune barriers to in vivo gene delivery, from biodistribution and safety to CMC and regulatory considerations, this article covers my recommendations on the topic as a virologist. I hope you find it educational and helpful regardless of your background. https://lnkd.in/gbx-jNnG
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Heart failure may soon have a new cure💡 Researchers at the University of Utah College of Health have developed a cutting-edge gene therapy capable of reversing heart failure in a large animal model with just a single IV injection. By using an adeno-associated virus (AAV9) to deliver the CBIN1 gene directly into heart cells, this therapy replenishes a crucial protein often depleted in heart failure. This innovative approach offers hope for millions affected by cardiac diseases, potentially transforming how we treat and manage heart failure in the future. Dhruv Jain Reference:-https://lnkd.in/gvuqHtZT
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A short memory might be good sometime as you forget you did the interview, but it becomes a brutal awakening when seeing it posted on LinkedIn. 😅 #cellandgenetherapy #processintensification #umetrics #ai #ml #designofexperiments #multivariatedataanalysis #advancedtherapies
Learn about today’s top challenges in #CellAndGeneTherapy and how to solve them from Johan Hultman at this year’s #ESACT. Explore more about Sartorius cell and gene therapy solutions: https://sar.to/5t6ox #SimplifyingProgress #InThisTogether #AdvancedTherapies #ESACT2024
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