Mariska ter Haak

CRS3123 is a small molecule protein synthesis inhibitor that acts on the novel target methionyl-tRNA synthetase (MetRS). CRS3123 has numerous potential advantages over current CDI therapies. Learn more about C. difficile Infection (CDI) and the Phase 2 study of CRS3123 here: https://bit.ly/3zhg5Kb

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CRS3123 is a small molecule protein synthesis inhibitor that acts on the novel target methionyl-tRNA synthetase (MetRS). CRS3123 has numerous potential advantages over current CDI therapies. Learn more about C. difficile Infection (CDI) and the Phase 2 study of CRS3123 here: https://bit.ly/3zhg5Kb

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Join Catherine Ulich (Senior Field Application Specialist) and Anthony Kayas (Account Manger) at Discovery on Target in Boston next week📍 Discovery on Target highlights advances in current and emerging “hot” targets and technologies, as well as target validation strategies for the discovery and development of novel therapeutic agents ranging from biologics to small molecules. Cathrine will be speaking at 9am on 1st October about Accelerating Clinical Translation: Harnessing Biology and Technology with Predictive Models 💡 You can also stop by Booth #212 to speak to our team and find out more about our models! 👋 #Newcells #DiscoveryonTarget #PredictiveModels

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🎤 uBriGene Presents Exclusive Sessions at SAPA 2024 @Philly CGT Annual Conference 🎤 🚀 We're thrilled to announce our participation at 2024 @Philly Cell and Gene Therapy Annual Conference with two exclusive sessions aimed at revolutionizing the field: 1️⃣ An Innovative Technology for High Yield and More Stable #CRISPR Guide RNA: Discover how our innovative approach of using in vitro transcription (IVT) in producing guide RNA (#gRNA) that can simplify the process while being more cost-effective and free of organic chemicals. Join us to delve into the future of precision gene editing. 2️⃣ How Could CDMO and Technology Innovations Make CGT Affordable: Dive into the intersection #CDMO of and technological advancements with uBriGene. Learn about our strategies to drive down costs without compromising quality or efficacy. Let's explore how cutting-edge technology can make #CellandGeneTherapy more accessible to all. 2024 @Philly Cell and Gene Therapy Annual Conference brings together the innovators and business leaders to foster collaborations and advance cell and gene therapy in the #GreaterPhiladelphia area. 💡 Join #uBriGene to be at the forefront of innovation and collaboration in the dynamic world of cell and gene therapy and let's make cutting-edge cell and gene technology more accessible!

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A Curative Solution: How Cell & Gene Therapies Support Patients & NJ’s Life Sciences Industry https://ow.ly/xOPe50UnvNc #CellTherapy #GeneTherapy #LifeSciences #Biotech #HealthcareInnovation #PatientCare #NewJerseyLifeSciences

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View the final agenda, take advantage of early savings, and join your colleagues developing targeted oligonucleotide therapies when you register for CHI's 7th Annual Oligonucleotide CMC & Regulatory Strategies conference. Taking place March 11-12, 2025 in Boston & virtually, you will join leading scientists to explore: -Advances in CMC & Analytical Characterization -Regulatory Strategies -Safety Challenges and Risk-Mitigation Strategies -Efficient Manufacturing Processes Join the conversation as we host this unique opportunity to discuss and collaborate on how to optimize product development processes and speed time market. https://lnkd.in/eYSYd2zE #OPTBoston #oligonucleotides #drugdiscovery #drugdelivery #CMC

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For anyone building diagnostics: Very cool approach to gain valuable insights into FDA decision summaries. Brendan O'Leary scraped the FDA’s decision summaries and provided some simple R code for anyone to get up and running in no time to prod and probe those summaries. Very nice work!

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CRS3123 is a small molecule protein synthesis inhibitor that acts on the novel target methionyl-tRNA synthetase (MetRS). CRS3123 has numerous potential advantages over current CDI therapies.

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Join Sharmeen Roy, PharmD, BCPS Roy on September 18 alongside a great line up speakers for the Technology Networks event **Advances in Drug Discovery & Development 2024** Full agenda here: https://lnkd.in/eDWdkQbH Sharmeen will dive into accelerating precision medicine through precision dosing. Great topic as hospitals and pharmacists are seeing the impact of personalized health to address every patient's unique needs and characteristics. #futureofmedicine #pharmacist #precisiondosing 

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This morning at the Alzheimer's Association® International Conference (AAIC) 2024 in Philadelphia, we presented data from a recent study that further demonstrates the high accuracy of our blood tests incorporating the ALZpath Inc. proprietary pTau217 antibody for Alzheimer’s disease diagnosis.   Our latest study, involving over 50,000 samples from 40 cohort studies, highlights the precision of the pTau217 antibody in determining amyloid burden across all stages of Alzheimer’s disease progression. This supports early detection, treatment, and tracking.   "The need for accurate and convenient testing methods, such as blood tests, has never been more evident," says Chad Holland, CEO of ALZpath. Our pTau217 antibody is a game-changer, offering a less invasive and more accessible alternative to traditional diagnostic methods. Read the full press release here: https://lnkd.in/gFjhVS3C   To learn more about ALZpath and our innovative solutions, visit https://alzpath.bio/ and follow us here on LinkedIn for the latest updates.   #Alzheimers #AlzheimersResearch #AAIC2024 #HealthcareInnovation #BloodTests #EarlyDetection #pTau217

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FDA Guidance on CGT Products: A New Era for Regulatory Clarity? The FDA’s draft guidance on CGT products is a critical resource for sponsors. It offers clear expectations for IND submissions, expedited pathways, and product characterization. Our team sees this as an opportunity for sponsors to take a more proactive approach with regulators - especially through early meetings like INTERACT and pre-IND. But we know the execution isn’t always straightforward. What are your thoughts on the guidance? Are you seeing clearer pathways, or do the hurdles remain? 👉 Access the guidance here: https://lnkd.in/eQbNpvJK #FDA #CGT #CellTherapy #GeneTherapy #INTERACT #preIND

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📢 Introducing one of our many #PanelDiscussions during the 2 day #PDDPSUMMIT at the Hilton Boston Park Plaza Email us at info@Precision-Globe.com to learn more! #DrugDiscovery #Preclinical #Antibodies #DrugDevelopment #AI #ML #MedTech #Biopharma #Pharma #Biotech #SmallMolecules #Biologics #LifeScienes #Medicine #Scientists #LifeScience #LargeMolecules

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We are thrilled to share the presentation of Stephanie Chrysanthou, PhD, our R&D Manager at Integrated Genomics Operation Memorial Sloan Kettering Cancer Center, on detection of germline alterations in solid #cancer patient samples by adaptive sampling 🙌 presented at the Nanopore Community Meeting conference in Boston! Our core has closely partnered with the Molecular Pathology & Diagnostics lab to benchmark Adaptive Sampling long read #sequencing for detecting germline #mutations against prior MSK-IMPACT profiles generated by hybridization capture short-read sequencing. Adaptive Sampling is a promising approach due to reduced technical complexity and TAT, there is no need for sample batching and it facilitates the resolution of large complex structural #variants. Due to the live #basecalling capacity of the #nanopore technology, the sequencing can be stopped if a fragment does not match the uploaded target .bed file, thus enriching on-target fragment #sequencing. Briefly, our on-instrument data workflow includes: 1) filtering of the raw .pod5 files based on the on-target decision, 2) SUP basecalling, 3) GRCh38 alignment, 4) SNV/SV calling using #EPI2ME and #SEVERUS pipelines.  On a routine basis, this workflow, from #hmwDNA extraction to data analysis, takes 3.5 days. Feel free to watch the full presentation here 👇 (it's free to register and you'll get access to all ONT conference presentations!) Oxford Nanopore Technologies https://lnkd.in/ezE_vsYT

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In the spirit of the 3 R's (Replacement, reduction, and refinement), EMA is championing reducing reliance on animal models in pharma/biopharma development. Find out more from Parexel Regulatory Strategy in our latest Regulatory Navigator blog.

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📢 FDA Alert The FDA has released a draft guidance, FAQ on Developing Potential Cellular and Gene Therapy Products. This resource is an invaluable tool for anyone in the CGT industry, offering clarity and insights into key regulatory expectations. A must-have reference to navigate this evolving landscape effectively! 🧬📄 #OCBio, #OrangeCountyBio, #CGT, #CellandGeneTherapy, #CellTherapy, #GeneTherapy, #Consulting, #Marketing, #FDA, #FAQ

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Bristol Myers Squibb: U.S. Food and Drug Administration FDA Approves Opdivo Qvantig™ (nivolumab and hyaluronidase-nvhy) Injection, for Subcutaneous Use in Most Previously Approved Adult, Solid Tumor Opdivo® (nivolumab) Indications PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) granted approval for Opdivo Qvantig™ (nivolumab and hyaluronidase-nvhy) injection for subcutaneous use, a combination product of nivolumab co-formulated with recombinant human hyaluronidase (rHuPH20), in most previously approved adult, solid tumor Opdivo indications as monotherapy, monotherapy maintenance following completion of Opdivo plus Yervoy® (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib.1,2 The approval is based on the results from the Phase 3 randomized, open-label CheckMate-67T trial, which demonstrated non-inferior co-primary pharmacokinetic (PK) exposures, similar efficacy in overall response rate (ORR), and showed a comparable safety profile vs. intravenous (IV) Opdivo . In the trial, noninferiority was demonstrated for the co-primary endpoints of time-averaged concentration over 28 days (C avgd28 ) and minimum concentration at steady state (C minss ) of Opdivo Qvantig vs. IV Opdivo .1,3 The geometric mean ratios (GMRs) for C avgd28 was 2.10 (90% CI: 2.00-2.20) and the GMR for C minss was 1.77 (90% CI: 1.63-1.93).1 As a key powered secondary endpoint, the overall response rate (ORR) in the Opdivo Qvantig arm (n=248) was 24% (95% CI: 19-30) compared with 18% (95% CI: 14-24) in the IV Opdivo arm (n=247), showing that Opdivo Qvantig has similar efficacy compared to IV Opdivo .1 Subcutaneous administration may offer flexibility to receive treatment where it is best for patients and their providers, and may reduce steps required for preparation and time needed for administration.5,6,7,8,9,10 In the CM–67T trial, average administration time with Opdivo Qvantig was approximately five minutes, and most patients received all doses of the study medication without an injection interruption or dose delay.3 With this approval, Opdivo Qvantig is now the first and only subcutaneously administered PD-1 inhibitor, offering a faster delivery for patients to receive this immunotherapy treatment option in three to five minutes compared to 30-minute IV Opdivo . https://lnkd.in/epeCtCyk

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𝗧𝗵𝗲 𝗔𝗗𝗟𝗠 𝗲𝘅𝗽𝗹𝗮𝗶𝗻𝗲𝗱 𝘄𝗵𝘆 𝗶𝘁 𝗿𝗲𝘀𝗶𝘀𝘁𝘀 𝗿𝗲𝗴𝘂𝗹𝗮𝘁𝗶𝗼𝗻: 𝘁𝗵𝗲 𝗽𝗿𝗼𝗰𝗲𝗱𝘂𝗿𝗲𝘀 𝗮𝗿𝗲 𝘁𝗼𝗼 𝗰𝗼𝘀𝘁𝗹𝘆 𝗮𝗻𝗱 𝗰𝗼𝘂𝗹𝗱 𝗱𝗲𝗽𝗿𝗶𝘃𝗲 𝗺𝗮𝗻𝘆 𝗽𝗲𝗼𝗽𝗹𝗲 𝗼𝗳 𝗮𝗰𝗰𝗲𝘀𝘀 𝘁𝗼 𝘁𝗲𝘀𝘁𝘀. It is hard to tell whether the ADLM is correct, and if many people will lose access to tests because of regulation. We understand that all parties are for the safety and quality of tests for the patient's benefit. The question is what controls we can afford so that it makes economic sense to do these tests. We need to justify our judgments with accurate numbers. If LIMS could collect the relevant information, we could conduct quick population-based studies based on FHIR. That would provide a personalized approach: which tests are rare, by how much, and who exactly needs them. https://lnkd.in/dqAnuPA9 #Diagnostics #LaboratoryMedicine #FDARegulations #LDTs #AACC #VivicaLIMS #LifeDataLab https://about.vivica.us https://lifedl.net

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