Michael Ehlers

This is a really important discussion hosted by the American Society of Gene & Cell Therapy. In order to improve patient access to transformative #CGT medicines, we need to consider the downstream effects of how trial designs inform drug labels, which are later referenced by payers making reimbursement determinations that may ultimately exclude some patients.?We need thoughtful drug development and evidence generation strategies in both the pre- and post-approval settings as a means of effectively expanding access. #ASGCTPatientEducation

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At Nautilus, we're developing a platform that can not only quantify proteins falling all across the vast landscape of the proteome, but that can also dive deep and quantify the patterns of post-translational modifications found on individual protein molecules. This is incredibly exciting because those proteoforms are the actual versions of proteins found in biological systems and they are drivers of cell biology (both healthy and disease). To learn more about proteoforms and the power that comes from understanding them, check out the latest episode of our Translating Proteomics podcast featuring proteoform pioneer, Professor Neil Kelleher!! https://lnkd.in/gfrKmd_Z #Proteoforms #TranslatingProteomics #Biomarkers #DrugDiscovery

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Good afternoon everyone! Today's blog dives into the power of a new AI-based predictive model (published in June in Nature Methods) that can effectively extract valuable drug discovery insight - but only after being trained on tons of single-cell RNA-seq datasets (more than 50 million data sets in fact). This exciting new predictive model, called as scFoundation, is designed to predict cellular responses to changes, gene interactions, and potential drug targets based on single-cell RNA sequencing data. It also improves the prediction of cancer drug responses and cellular responses to perturbations. In essence, it uses patterns in the data to make predictions about various aspects of cellular behavior. This makes it a valuable tool in fields like precision medicine, targeted cancer therapy, and understanding of complex cellular behaviors. Hope you enjoy! #bioinformatics #scRNA-seq #AI #predictivemodels

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?? Big News in Biopharma! ?? Profluent is making waves at the intersection of biotech and AI. Their innovative approach promises to revolutionize gene editing, and they're doing it with a bold move: open-sourcing their work! ???? Introducing OpenCRISPR ?? Profluent's OpenCRISPR initiative is set to provide customizable gene-editing proteins designed entirely by AI. Imagine AI-generated CRISPR-like proteins that have never been seen before, capable of editing the human genome with unparalleled precision. ?? Highlights: AI-Powered: Trained on vast databases of protein sequences, Profluent's AI can generate hundreds of thousands of CRISPR-like proteins. OpenCRISPR-1: Their top-performing AI-created gene editor boasts 400+ mutations compared to CRISPR-Cas9, reducing off-target editing by 95% and keeping unwanted genetic changes below 1%. Why This Matters Attempting to edit human DNA with AI-designed systems is nothing short of a scientific moonshot. Profluent's success points to a future where AI can design bespoke cures for diseases, democratizing gene editing. ?? Real-World Impact: While AI-designed drugs have been in clinical testing for years, CRISPR tech just got its FDA green light last year with Casgevy for sickle cell disease and beta thalassemia. These treatments show the incredible potential of gene editing to transform lives. Open Collaboration Profluent invites researchers to test and provide feedback on OpenCRISPR-1. Their goal is to partner with top research institutions and drug developers to expedite the development of new genetic therapies, ensuring safe and ethical practices. Recent Milestones $35M Funding: Backed by Spark Capital, Insight Partners, Air Street Capital, and top angel investors from OpenAI, Salesforce, and Google. Join the Revolution This is a watershed moment in gene editing. Profluent is not just pushing the boundaries of what's possible; they’re opening the doors for global collaboration. Let's build the next generation of genetic medicines together! ?? Read more and get involved: https://lnkd.in/gN9p-H6j #GeneEditing #CRISPR #AI #Biotech #OpenSource #Innovation #Profluent #HealthcareRevolution

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://https://lnkd.in/giUEBUHC Amprion is proud to be working with ALS Network to elucidate the role of Synuclein co-pathologies in ALS progression. Our previous collaboration with @RichardSmithMD and his Center for Neurologic Studies revealed an ALS co-pathology rate of approximately 14% against an age- and gender-matched control rate of less than 3%. The current follow-on study seeks to determine how Synuclein co-pathology in ALS influences clinical presentation including cognitive effects or enhanced rates of progression. #ALSNetwork #Amprion #CenterForNeurologicStudies #ALSCoPathology #Synuclein

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Epoch 19: The AutoImmune Renaissance A few weeks back we went into detail on this topic, with a primary focus on CAR-T. Today we broaden to cover additional modalities. ??CD19 ??CAR-T ??FcRN ??T-Cell Engagers https://lnkd.in/dEkBy2bY

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Just a few days after Element Biosciences celebrated its 7th anniversary of incorporation, we announced an oversubscribed series D of $277M. What a remarkable journey. In seven years, we have raised $680M, and words alone cannot express my gratitude for these groups of very special people, our team, our customers and partners, and our investors. First and foremost, our loyal, resilient and mission-driven Element team. You have persevered uncertainties, naysaying, and ambitious timelines to change how science is done and make a real impact in the world. Disrupting the norm is never easy. As RFK said: “if you are not the recipient of some derision on your path to impact change, you are probably not changing enough." Together, we have built disruptive technologies and formed an incredible company. I am so tremendously proud of you, and the best is yet to come. I am grateful for all our customers and partners, current and future. We will work with you as our long-term partners, hear your feedback, and make our products better each day. Together, we can change how science is done around the world, so that everyone has access to the best scientific tools regardless of how well they are funded. Finally, I want to thank our new and existing investors for your trust and confidence in Element. We raised these funds in the backdrop of a challenging macro-economic environment and fierce competitions in the genomics field. Element is proud to be chosen as your partner. Today marks a significant milestone in Element’s journey. With the support and dedication of our teams, customers and partners, and investors, we will continue to innovate, deliver the best solutions for our customers, and solidify our future as the next name-brand biological tools company. We are just getting started. #genomics #multiomics #innovation #grit

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??ONE Week Left to?Invest in Our Breakthrough?Biotech ?? invest.cytonics.com Since 2006, Cytonics has been leading the charge in developing groundbreaking therapies for osteoarthritis. With the steadfast support of our investors, we have successfully?raised over $25 million, pushing our innovative CYT-108 therapy into Phase 1 human clinical trials. We invite you to join us on our mission to rid the world of osteoarthritis, once and for all. With 1 week remaining (campaign ends August 25 at 11:59pm PST / August 26th at 2:59am EST), seize the chance to join over 6,000 dedicated shareholders committed to eradicating osteoarthritis, once and for all. This is more than an investment in a breakthrough biotech; it's an investment in the improvement of human health and well-being for generations to come. #biotechnology #drugdevelopment #clinicaltrials #arthritis #regenerativemedicine #investing #crowdfunding

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Add more quantifiable data revvvvvv your AI engine!!!! Not just binding and sequences… add color to your data to tremendously increase your algo’s prediction power! Only on the #Beacon #optofluidic platform

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Using 'representation vectors' instead of prompt engineering to modulate #LLM responses enables tuneable behavior: https://lnkd.in/eUwSUvtg

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Fantastic Webinar with Dr. Farooqi. Anyone interested in tumor driving gene fusions and gene rearrangements should not miss.

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A great article featuring?Carl June, the Father of CAR-T, was just published. It highlights his optimism for CAR-T therapies targeting Glioblastoma (our lead indication at?KiraGen Bio) and reinforces his confidence in the future of ex vivo cell therapies. There's been much discussion lately about the emergence of in vivo therapies soon to enter the clinic, and how they'll coexist with ex vivo approaches. June, like us at KiraGen, believes both will play important roles for the foreseeable future. Ex vivo editing likely will always have some key advantages: -Larger payloads -No off-target cell toxicity concerns -Lower efficacy burden The technology for both approaches evolves in tandem. As in vivo editing improves, ex vivo products will likewise evolve to harbor more complex circuits and even higher multiplex edits. The result? Two distinct classes of cell therapies, each with its own pros and cons, providing oncologists with a diverse toolkit to combat cancer. At KiraGen, we're excited to be at the forefront of ex vivo innovation, pushing the boundaries of what's possible in cell therapy. Stay tuned for updates as we build upon such exciting recent clinical success for patients in need! (Credit for thoughts above: Jacob Kimmel and Dr. Shelby) https://lnkd.in/eKhEGr32

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Brains are complex, and sometimes complex models are what is needed to accelerate your R&D with confidence. Check out this case study in which PhenoVista measured the effects of adding microglia into a co-culture of human, iPSC-derived neurons and astrocytes. You can download a copy from our Learning Library here: https://hubs.ly/Q02J0sD60

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For investors interested in our Q2 roadshow covering New York, Boston, Princeton, Chicago, San Diego, and San Francisco, BIORCHESTRA Co., Ltd.'s #RNAi update covers: ??In-vivo progress on our neurodegenerative diseases franchise and proprietary IV-formulated RNAi research?in #Alzheimer's and #Parkinson's ??Status of our #ALS program FDA IND pathway? ??Licensing negotiations in #CNS, #INHBE/ #MAFLD, and #Glaucoma ??Our bridge round syndicate and NYSE / NASDAQ pathway timing To those requesting one-on-ones through LinkedIn, email [email protected] for slots. Bramalea Partners, PJT Partners, Perella Weinberg, William Blair Investment Banking, Jefferies, Leerink Partners, Nomura, Cain Brothers, CITIC Securities Company Limited, CITIC CLSA Forums, UBS, Oppenheimer & Co. Inc., Wells Fargo Corporate & Investment Banking, Mikael Dolsten Guggenheim Partners, Outcome Capital, Brian Doyle, Michael Bardi, Michael Zhang, Veet Misra, Cantor Fitzgerald, No?l Brown, Samuel Siu, CPA, RBC Capital Markets, John C. Reed, Novo Holdings, Lilly Asia Ventures 礼来亚洲基金, Richard A. Gonzalez, Sumitomo Mitsui Banking Corporation – SMBC Group, #pfizercolleague, Centerview Partners, Morgan Stanley USA, Citi, Bank of America Business, Greenhill & Co., Evercore, Rothschild & Co., Moelis & Company, New York Stock Exchange, Nasdaq Exchange

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?? New Blogpost Alert!?? Excited to announce our latest blogpost in which we benchmark different generative algorithms (e.g. #claude and #REINVENT) for their capabilities to generate novel and valid molecules. Turns out most modern generative algorithms are pretty good and can help chemists if paired with good models for activity and ADMET! Highlights: ?? Claude, a large language model, appears to be an ok molecule generator ?? Classic medicinal chemistry driven approaches are competitive against more modern AI driven approaches such as #REINVENT ?? All algorithms are good at scaffold hopping, i.e. generating molecules that are very different from original ones! Link to post and code in the comments! Big shoutout to Cecilia Cabrera and Ryan Greenhalgh for data analysis and writing. Andrea Dimitracopoulos and Jacob Green for editing. Special thanks to Pat Walters, Leonard Wossnig, and Nelson Lam for helpful comments and suggestions. #AI #DrugDiscovery #Innovation #generativeAI DeepMirror

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What a fascinating and productive workshop we are having at Cold Spring Harbor Laboratory in New York from June 3 to 6th, titled "Using Cell-free Systems to Synthesize and Express Bacteriophage Genomes: Practical Biomanufacturing for Bioscience Educators." This event, held at the DNA Learning Center NYC at City Tech, Brooklyn, is empowering bioscience educators with the tools and knowledge to introduce students to innovative biomanufacturing techniques. Under the guidance of Vincent Noireaux, participants are exploring a cell-free transcription-translation (TXTL) system to manufacture bacteriophages with new host specificities. This workshop not only honors the legacy of pioneering bacteriophage research at Cold Spring Harbor but also highlights the contemporary applications of phages in tackling multi-drug resistant bacterial infections, improving food safety, and advancing biotechnological innovations. Through hands-on experiments, attendees are engineering T7 phages to infect new E. coli hosts, conducting PCR mutagenesis, and utilizing nanopore DNA sequencing to investigate phage infectivity, making this a truly transformative experience for all involved. Bacteriophages, once again at the forefront of biotechnology, offer solutions to critical challenges in pharmaceuticals, agriculture, and food safety. By equipping educators with cutting-edge techniques to manufacture phages with new host specificities.

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ElevateBioElevateBioPowering the creation of cell & gene therapies at a speed the world deserves. Home / Investors & Media / Press Releases / ElevateBio Announces Presentations at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting ElevateBio Announces Presentations at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting Waltham, Mass., April 22, 2024 – ElevateBio, LLC (ElevateBio), a technology-driven company focused on powering the creation of life-transforming genetic medicines, today announced four poster presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, in Baltimore, MD. The poster presentations will detail ElevateBio’s expertise in optimizing the development and manufacturing of genetic medicines, including scalable screening and manufacturing processes for adeno-associated virus (AAV) vectors for gene therapies, a proprietary CAR/TCR platform that accelerates the nomination of therapeutic receptors for faster development of engineering cell therapies, and an efficient, scalable process to produce high purity circularRNA (circRNA) for potential therapeutic applications both ex vivo and in vivo. “These data underscore our commitment to developing scalable solutions and capabilities to accelerate cell and gene therapy development,” said Mitchell Finer, Ph.D., President, R&D, ElevateBio. “As a technology-forward company, we’re thrilled to showcase these latest advancements that are fueling the programs of our partners and moving the industry forward.” Presentation Details: Title: Early Characterization of Research-Grade AAV6 Productions Allows for Manufacturing Alignment During Candidate Nomination Poster/Abstract #: 1435 Session: AAV Vectors - Virology and Vectorology Date/time: Friday, May 10, 2024 at 12:00-7:00 p.m. ET Title: Development and Scale Up of a Robust End-to-End Manufacturing Process for High Quality AAV Production Poster/Abstract #: 1534 Session: AAV Vectors - Product Development Manufacturing and Approval Considerations Date/time: Friday, May 10, 2024 at 12:00-7:00 p.m. ET Title: Design and Production of Circular RNAs (circRNAs) with Validated Functional Activity in Cell Models Poster/Abstract #: 1711 Session: Oligonucleotide Therapeutics Date/time: Friday, May 10, 2024 at 12:00-7:00 p.m. ET Title: A Rapid and Scalable 96-Well Platform to Screen CARs and TCRs Poster/Abstract #: 1894 Session: Molecular and Cellular Methods Date/time: Friday, May 10, 2024 at 12:00-7:00 p.m. ET

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