Heather A. Steinman, PhD, MBA

PTC was excited to learn, share and collaborate with other leading RNA experts at the recent 7th Annual RNA-Targeted Drug Discovery & Development Summit in Boston, MA, and the RNA at the Bench and Bedside IV conference in La Jolla, CA. Anu Bhattacharyya, Kerstin Effenberger, and Marla Weetall from our research team presented on ways PTC is leveraging its leading splicing platform to identify promising therapies for rare diseases with high unmet need. Learn more about our splicing platform here: https://bit.ly/4fi4w6L

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Join us December 10-12 at the 7th Annual RNA-Targeted Drug Discovery & Development Summit in our home city of Boston! We can't wait to explore the latest and greatest in RNA-targeting therapeutics with experts and innovators from across the RNA world.      If that includes you, we'd love to connect and discuss how the Momentum team can accelerate your drug discovery and development project. We have experience working with RNA targets across a wide range of analytical services and can provide customized assay solutions to best tackle your specific research challenges.      On Wednesday, December 11th at 2:30PM, our CEO & Founder Can Ozbal will present "A Complete Workflow for the Identification and Characterization of Small Molecule Modulators of RNA." This talk will describe an end-to-end strategy that can be used to discover, validate, and characterize potent and specific RNA-targeting small-molecule drugs, including covalent drugs.      To schedule a time to meet with the Momentum team at the 7th Annual RNA-Targeted Drug Discovery & Development Summit, send us a message at: https://lnkd.in/dSD6dHDZ     Or, to learn more about the conference, visit: https://lnkd.in/gzcGvHH     #RNADrugDIscovery #RNAResearch #RNA   

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🚨 Webinar Announcement: Improving Outcomes and Access to Precision Medicine🚨- https://lnkd.in/eSvkPZPG Despite groundbreaking advances in precision medicine, a critical gap remains: the lack of diverse representation in genomic data, leading to unequal healthcare outcomes—especially for people of color. 🔬 What is the impact? Genomic testing is a cornerstone of modern medicine, yet current databases are largely based on data from individuals of European descent. This leaves underrepresented populations at a disadvantage, with less effective treatments and limited access to life-saving innovations. 💡What you’ll learn: 1️⃣ Understanding the genomic testing gap and the structural barriers in healthcare 2️⃣ How to advance precision medicine for all, ensuring inclusivity 3️⃣ Practical strategies for improving access, including policy changes, community engagement, and innovative healthcare models 📅 Date: Thursday, 21st November, 2024 ⏰ Time: 12:00pm - 1:00pm ET 📍 Location: Zoom (Register Here https://lnkd.in/eSvkPZPG) Let’s work together to create a healthcare system that serves everyone. #PrecisionMedicine #GenomicTesting #HealthEquity

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Better health outcomes start with access to the right tools. Bill A4163 would break down barriers to biomarker testing for patients across NJ. 🔗 Details here: https://lnkd.in/e4XX5AVa Bill A4163 is sponsored by Assemblywoman Shavonda Sumter, Assemblyman Gary Schaer, and Assemblywoman Shama A. Haider. American Cancer Society American Cancer Society Cancer Action Network (ACS CAN) #biomarker #biomarkertesting #NewJersey

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How do the diabetes drugs, DPP4 inhibitors, relate to the lung? They give lung stem cells back their regenerative ability! Michael Bollong at Scripps Research told Stephanie DeMarco, PhD all about their new DPP4 inhibitor that’s delivered directly to the lung. Read more: https://ow.ly/p0BI50U9wBj

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Excited to share a new publication in the Journal of Molecular Therapy which highlights the eShunt®️ Platform as a CNS gene therapy delivery method. Thanks to the teams at UMass Chan Medical School and Tufts Medical Center for your work on this study. "We're very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain." #bbb #CNS #drugdelivery #neurovascular #endovascular https://lnkd.in/eeKCGPm7

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In part two of our series with Applied Clinical Trials Magazine featuring Ken Getz of the Tufts Center for the Study of Drug Development and Steve Young of CluePoints, Ken and Steve highlight what stood out to them from the results of our recent study on RBQM adoption. https://bit.ly/3WKWfm3 #ClinicalTrials #RBQM #Innovation #TuftsCSDD #CluePoints #PwC 🚀🔬

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QurAlis Corporation and UMass Chan Medical School have partnered to develop potential therapies using antisense oligonucleotide (ASO) splicing technology. This collaboration builds on groundbreaking research by Dr. Joel Richter and his team at UMass, funded by FRAXA Research Foundation. Their discovery revealed that the FMR1 gene in individuals with Fragile X syndrome isn't entirely inactive but produces a mis-spliced messenger RNA. With an ASO, they aim to neutralize this deviant RNA, potentially restoring normal protein production and reversing signs of Fragile X syndrome. Today's announcement marks a significant leap towards clinical trials, bringing hope for effective treatments. #FragileX #FXResearch #FragileXAwareness #Neurogenetics #PrecisionMedicine #ASOTherapy #MedicalBreakthroughs #GeneticResearch

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Addressing the problem of drug safety and efficacy has been top of mind for Aaron Goldman, PhD, an associate bioengineer at the Brigham. In addition to uncovering cancer’s vulnerabilities, Goldman’s lab has developed and patented several cancer drugs, from small molecules to cell therapies. Learn more about Goldman’s work advancing drug development:

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Jonathan Montagu, CEO of MassBio member HotSpot Therapeutics, Inc., writes on the #LifeSCiVC blog about hopes around an immuno-oncology spring this year, aligning with the emergence of #biotech from its own long winter. Jonathan points out that after the #lifesciences industry dove headfirst into I-O “to address the substantial need that still persists for many cancer patients with a range of tumor types,” much of the second-generation agents fell short. He writes about why he’s optimistic that new approaches have started to turn the tide. What are your thoughts on the I-O space right now? https://lnkd.in/eMh62Z8j #immunooncology #oncology #patientdriven

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The breakthrough research by Penn Medicine, the Rockefeller Neuroscience Institute, and Precision Neuroscience, showcased at the 2024 Congress of Neurological Surgeons, reveals unprecedented insights into brain signal mapping using their Layer 7 Cortical Interface. With remarkable accuracy in decoding movement and speech, this technology offers new hope for brain-computer interface applications, potentially restoring function for individuals with paralysis or speech impairments. These advancements bring us closer to personalized neurotherapeutic solutions in precision psychiatry and neurological care. This milestone is just the beginning—one that gives hope for a future where conditions such as paralysis and neuropsychiatric disorders can be addressed with unprecedented precision, ultimately restoring autonomy and quality of life for so many. This is so exciting and can't wait to see the impact this breakthrough will have on the world of neuroscience and beyond.

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📣PEGS BOSTON 2025 PRELIMINARY AGENDA JUST RELEASED! https://lnkd.in/ecqAmYuP Showcasing over 300 presentations, breakout sessions, and interactive seminars, the PEGS Boston Summit offers an unparalleled platform for sharing cutting-edge advancements in drug development, protein and antibody engineering, immunotherapy, radiotherapy, and AI/ML-driven biologics research. Here's a sneak peek at the programs and list of confirmed speakers: https://lnkd.in/ecqAmYuP

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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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The #biosecure act is bad legislation and anti-competitive and will force US #biotech companies to abandon strong and honest #CDMO partners without making us more secure. Longstanding, productive drug-discovery collaborations will inevitably be compromised by an ill-conceived initiative — all for short-term political gain. Patients in need of innovative medicines are the ones that will lose out; I urge leaders in the #biotech industry to stand up and be counted in opposition to this proposed legislation.

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🚀 Join us at the Antibody Therapeutics Xchange - Maryland 2024! 🔬💡 We're excited to highlight Birthe Jessen-Park, Technical Sales Specialist Custom Antibodies at Bio-Rad Laboratories🌟, an industry leader in antibody discovery, who will be presenting at two exciting sessions during this year's event: 📅 Time: 11:20 AM - 12:20 PM ✨ Roundtable Discussion: "The Perfect Workflow for Generating Antibody Drug Candidates" 🧬 Key topics covered include: 🤔 How many hits (binders) should be further analyzed? 🎯 What are the most relevant selection criteria? 🧠 Solving challenges like affinity, developability, and epitope selection. 📅 Time: 3:35 PM - 4:05 PM 🔍 Poster Presentation: "Rapid Generation of Bispecific Antibodies for High Throughput Screening with SpyLock Technology" 🧫 Birthe will introduce SpyLock Technology 🛠️, an innovative approach for creating bispecific antibodies. This technology, compatible with Bio-Rad’s Pioneer™ Antibody Discovery Platform, offers a faster, more efficient path to discovering bispecifics 🔝. With a rich background at Bio-Rad Laboratories, Alloy Therapeutics, and Medimmune, and a PhD from the University of Freiburg 🎓, Birthe brings unparalleled expertise to these sessions. 📍 Location: College Park Marriott, Maryland 🏨 📅 Date: October 28, 2024 Secure your spot and get your complimentary pass! https://lnkd.in/eqyZAeXX 💬 Don't miss this chance to connect with Birthe and other top industry experts! Let's shape the future of antibody therapeutics together! 💼✨ 💊 Only available for Senior Scientists and above, from Bio and Pharma companies with a drug pipeline. #AntibodyTherapeutics #Biopharma #Biotechnology #PharmaInnovation #AntibodyDiscovery #DrugDevelopment #LeadOptimization #BioRad #TherapeuticsXchange #ADC #MultispecificAntibodies #BiopharmaLeaders #ProteinEngineering #BiotechEvents #PharmaIndustry #LifeSciences #TherapeuticInnovation #BiotechNetworking #ScientificResearch #AntibodyEngineering #PharmaCollaboration #NextGenTherapeutics #BioPharmaExperts #PharmaRND #hubXchange #Xchanges #Roundtable #FreePass #RegisterNow #ComplimentaryPass

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Listen to David Segarnick, PhD discuss the Evolution in Biomarkers and Precision Medicine in Oncology. 🔬 The latest developments in using biomarkers to guide cancer treatment decisions. 🎯 Novel biomarkers for targeted therapies. 👩 Personalized medicine approaches in treatment resistant and refractory disease. Watch our #ASCO24 Key Findings and Missed Gems webinar here: https://lnkd.in/dju8fTaK #ASCO24 #DownloadableContent #Webinar #InizioMedical

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ADCs represent a rapidly growing sector in oncology therapeutics, combining the targeting capabilities of monoclonal antibodies with the potency of cytotoxic drugs. We are excited to announce a strategic alliance between NJ Bio, Inc. and Discovery | Charles River to advance antibody-drug conjugate (ADC) development and manufacturing services. This collaboration combines Charles River’s expertise in antibody engineering with NJ Bio’s proficiency in developing novel linker-payloads, offering an integrated solution that accelerates ADC development timelines. #ADC #Antibody #drugdiscovery https://lnkd.in/ePTiBX2d

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Tissue-resident #NKcells do their own (G)landscaping | Jake Myers & friends "slaver over" Major New Study from Sonia Tugues Solsona & Co. with Breaking Insights* at Rockefeller University Press #JExpMed | #TGFβ | #IL15 | #ILC1 | In the latest issue of JEM, Colin Sparano, Darío Solís-Sayago et al** present compelling evidence that salivary gland trNK cells originate from cNK cells and are developmentally distinct from ILC1 cells. Mechanistically, they demonstrate that continuous autocrine TGF-β signaling drives salivary gland tissue residency and works in synergy with IL-15 to enhance Hobit-dependent cytotoxicity. *https://lnkd.in/eXYTdwjs **https://lnkd.in/e4iXZ4UJ Celentyx Ltd Professor Nicholas Barnes PhD, FBPhS Omar Qureshi Catherine Brady FIGURE | Autocrine TGF-β signaling is required for the transition of conventional NK (cNK) cells to tissue-resident NK (trNK) cells in the salivary glands. IL-15 then synergizes with TGF-β to drive NK cells toward Hobit-dependent cytotoxicity | Courtesy of Jake Myers, Shanelle Reilly, PhD & Laurent Brossay |

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What is a checkpoint inhibitor? In our new CEO spotlight series, Vlad Vitoc, MD, MBA, dives into this drug that helps the immune system attack cancer cells. Find out by watching the video clip below and stay tuned for more as Vlad brings you firsthand insights from the #biotech world. #Oncology #OncNews #Immunotherapy

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