Steven Quay MD, PhD

Proud to have fellow Biotechnology Innovation Organization board member and Arcutis Biotherapeutics, Inc. (Nasdaq: ARQT) CEO Frank Watanabe testify at the House Ways and Means Committee hearing on July 12th in Salt Lake City. Frank highlighted the contributions the biotech industry has made to human health and the economy: "In the last quarter century, American life expectancy has continued its steady increase upwards, with roughly one-third of that improvement directly attributable to biopharmaceutical innovations.” The big picture: The industry’s direct and indirect contribution to the U.S. GDP is anywhere from 2-7%, with an economic impact of $2.9 trillion in 2021. Biotech capabilities enhance national security by ensuring we can care for ourselves. Legislative proposals that would support innovation include: - The Ensuring Pathways to Innovative Cures (EPIC) Act, which would give small molecule drugs the same 13-year protection from price controls as biologics, and the ORPHAN Cures Act, which would exempt orphan drugs from price controls even if approved for more than one indication. - The Tax Relief for American Families and Workers Act (H.R. 7024), which was passed by the House earlier this year, to restore the R&D tax deduction. Allow for monetization of net operating losses (NOLs) and reform Section 382 on NOLs. - Improve the capital gains exemption under Section 1202 to encourage investment in small biotechs. - Ensure strong strong patent protections and reasonable regulation of M&A activity. Proud of the work BIO does to support the industry, and proud to be a part of community that works tirelessly to drive innovation and improve human and planetary health. #patientsfirst #biotechnology #innovation #humanhealth #sustainability

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Key Takeaways: Intellia Therapeutics, Inc.'s Gene Editing Treatment Nex-z Encouraging Early Results • Nex-z demonstrated potential to stabilize or improve outcomes in ATTR cardiomyopathy • Results from 36 patients showed a median 5-meter improvement in the six-minute walk test after one year. • 92% of patients experienced disease stabilization or improvement in their New York Heart Association class. • Mean transthyretin (TTR) reduction was 90%, achieved by inactivating the gene encoding TTR protein. • Findings are preliminary and must be confirmed in the ongoing, larger 765-patient Phase 3 MAGNITUDE trial. Comparison to Competitors • Pfizer’s tafamidis (Vyndamax/Vyndaqel) is the only approved treatment, with $3.3 billion in 2023 sales. • Upcoming competitors include BridgeBio’s acoramidis, Alnylam Pharmaceuticals’s vutrisiran, and AstraZeneca/Ionis’ investigational drug. #geneediting #genetherapy Regeneron John Leonard #advancedtherapies #regenerativemedicine #biotech #biotechnology #clinicaltrials #datareadout #lifescience #lifesciences #PatientOutcomes #RareDiseaseTreatment #Cardiomyopathy #BiotechCollaboration #ScientificBreakthrough #collaboration #PharmaInnovation #BiotechPipeline #DrugDevelopment#ClinicalTrials #Phase1Trials #Phase3Trials #PipelineInnovation #FutureOfMedicine #IntelliaTherapeutics #ATTRAmyloidosis #TTRReduction #InVivoGeneEditing #ExVivoTherapies #CRISPRInMedicine #CRISPR #InnovativeMedicine #NextGenTherapies #PrecisionMedicine

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We will present two sets of data at the upcoming AACR Special Conference in Cancer Research: Liquid Biopsy: From Discovery to Clinical Implementation, November 13-16, 2024, San Diego, California. 1. A cost-effective two-step approach for multi-cancer early detection in general population. 2. A Blood-Based Multi-Omics Test for Early Evaluation of Lymphoma Treatment Effectiveness.

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I hope you can join me and Lindsey Gasparini, of NeoGenomics, for a discussion of how biomarker-linked, multi-modal, longitudinal clinical datasets can be the basis for meaningful and actionable clinical insights, foundation for advanced AI model development, and inform the design of retrospective RWD and prospective RCT studies. It will be a pleasure to do this with our wonderful colleagues at Datavant (we've worked with them since our founding!) and our wonderful moderator Alan Ma. #concertai #neogenomics #precisiononcology #AI #precisionmedicine

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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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In 1973, Stanley Cohen, Herbert Boyer, Annie Cheng, and Robert Helling published "Construction of Biologically Functional Bacterial Plasmids In Vitro," their landmark paper on Recombinant DNA that many consider the intellectual seed for the biotechnology industry. In that seminal paper, the key restriction enzyme that allowed for the creation of the new, Recombinant plasmid DNA species was EcoRI. In this 51st year since their breakthrough publication, it may serve us to ask how our organizations can cultivate a recombinant engineering and design culture inspired by the EcoRI enzyme, catalyzing intellectual exchange and incorporating genes of interest from disparate fields into transformative technologies. https://lnkd.in/g9J_53RJ

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Interested in the latest developments at the FDA's Office of Therapeutic Products (#OTP)? Join a free, public webinar on June 14th featuring Dr. Nicole Verdun, OTP's Super Office Director, and Rachael Anatol, Ph.D., Deputy Super Office Director. What you'll learn: The new "Super Office" structure of the OTP Progress on hiring new staff An overview of the OTP's organization Brought to you by Alliance for Regenerative Medicine. Please use the link in the post below. #celltherapy #genetherapy #cellandgenetherapy #advancedtherapies #lifescience #lifesciences #regulatory #structure #biotech #biotechnology #webinar #productupdate #regulatorystructure

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在BeiGene，我們致力專注於 iPSC-derived 細胞治療研發，以提升對尚未滿足醫療需求患者之可近與可負擔性。 去年九月，很榮幸邀請澳洲莫納許醫學中心臨床血液科主任Prof. Stephen Opat 蒞臨參訪BeiGene細胞治療研發中心。Prof. Opat對研發中心先進的設備與專業高效的團隊感到印象深刻。 Prof. Opat與BeiGene副總裁兼細胞治療負責人 Alex Huang 二位專家也藉此機會深入探討細胞治療發展現況與挑戰，以及同種異體細胞治療將有望作為細胞療法普及化的關鍵技術。 一起來了解二位專家對細胞治療未來變革與願景：https://shorturl.at/wixzq At BeiGene, we’re committed to advancing iPSC-derived CAR-T therapy to improve accessibility and affordability for patients with unmet medical needs. Last September, we had the honor of hosting Prof. Stephen Opat, Director of Clinical Hematology at Monash Medical Centre, at our world-class Cell Therapy Research Center. Prof. Opat was impressed by our cutting-edge facilities and the expertise of our dedicated team. During an insightful discussion, Prof. Opat and our VP & Head of Cell Therapy, Alex Shih-Min Huang, explored the challenges in autologous cell therapies and the exciting potential of allogeneic approaches. Discover their vision for the transformative potential of cell therapy at https://shorturl.at/wixzq #CellTherapy #iPSC #CancerResearch

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ANOTHER PATENT ISSUED for BIOELECTRIC STIMULATOR https://lnkd.in/g2cCCnkq for organ regeneration including skin and hair, healing including joint health and healthspan extension October 8th, 2014 by the USPTO! https://lnkd.in/g2cCCnkq with 15 new claims including... 3. The method according to claim 1, wherein the target tissue is selected from the group consisting of brain, scalp, eye, ear, skin, tooth, dental gum, tooth root, sub-mucosa, breast, aorta, limb, artery, heart, heart valve, kidney, pancreas, bladder, liver, joint, bone, and any combination thereof.

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Back in San Diego in a couple of days for the “Precision in Drug Discovery & Preclinical Summit San Diego” 2024. Looking forward to presenting our talk on “Discovery and development of novel DNA repair inhibitors as precision oncology therapeutics” (on behalf of XPose Therapeutics) and being a panelist on “Balancing The Human Expertise and Artificial Intelligence in Drug Development”. #drugdiscovery #therapeutics #oncology #PDDPSUMMIT Precision Evolution Global

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Artiva Biotherapeutics, a clinical-stage NK cell therapy biotech, is trying again for the public markets, it revealed Friday afternoon in an #SEC filing. The San Diego biotech is going for a Nasdaq debut but in a very different biotech financing environment than when it originally sought an #IPO in the spring of 2021, at a time when large public floats were easier to achieve thanks to low interest rates and generalist investors’ intrigue in drug development. And this time around, it’s without partner Merck, which axed their CAR-NK solid tumor collaboration last October, according to Friday’s S-1 filing. #NKcelltherapy #celltherapy #cellandgenetherapy #advancedtherapies #publicmarkets #biotech #biotechnology #financing #funding #lifescience #lifesciences #IPOs #solidtumors #bloodcancers #autoimmunedisease GC Cell 5AM Ventures venBio RA Capital Management

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Stats at the basis of Science. Statistics are crucial in science as they provide the tools to analyze complex data, draw meaningful conclusions, and validate experimental results. In science, and particularly in biology, where variability is inherent in living systems, statistical methods help distinguish between random variations and significant patterns. Stats enable researchers to design experiments, interpret outcomes with confidence, and make informed decisions. Without statistics, biological research would lack precision and reliability, but more important, it would move forward slower.

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Early data suggests that bispecific antibody petosemtamab, in conjunction with Keytruda, generates a 60% response rate in patients with second-line head and neck cancer. However, more mature clinical data will give us a clearer picture of how accurate this is. Get in touch to discover how evitria can support your antibody needs. #Biotech #BispecificAntibodies #Antibodies #HeadAndNeckCancer

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#ICYMI: Last month we announced that we raised $132.5 million in an oversubscribed #SeriesC financing to advance our pipeline of potential therapies for fibrotic lung diseases and oncology. We are thankful for the support of our investors, board, #biotech community and the entire Endeavor BioMedicines team.     In this BioSpace article, get more details about what this financing means for the future of our pipeline and our goal to restore hope for patients with life-threatening diseases: https://bit.ly/4aVqAmo    #biotechnews #fundraising #venturecapital #oncology #lungdiseases

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“Anything in the IRA [Inflation Reduction Act] that stops us from using drugs to their maximum potential is a mistake,” says Emil Kakkis. At the Stanford Biodesign Policy Program’s Health Technology Innovation Policy Conference 2024, Kakkis and other speakers on the biotechnology panel — Jeff Allen, Ken Drazan, Peter Kolchinsky, and Peter Marks — discussed the implications of the IRA on orphan drugs, and the unintended negative consequences on patients and drug manufacturers. Acknowledging that the IRA has accomplished some important things that will make a real difference in affordability and access to healthcare, the panelists highlighted, however, that the impact for orphan drug development may be felt in the next few decades as companies limit investment/research into additional indications for orphan drugs. The panelists also discussed the potentially transformative impact of cell and gene therapies and what can be done from a regulatory perspective to support that ecosystem. If you were unable to join us, here’s a replay of the discussion https://lnkd.in/gji8d5XE Many thanks to Stanford Biodesign’s D. Michael Ackermann, director, biotechnology, who moderated the session. #healthpolicy #biotech #healthtech #SBDHealthtechInnovationPolicy

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The theme from D1 Cell and Gene Meeting on the Mesa (Alliance for Regenerative Medicine) was about coming down to earth & atomizing the commercialization and industrialization problems of cell and gene therapy into attainable building blocks. Check out my convos w/ Dyno Therapeutics, Profluent, Astellas Gene Therapies / Astellas Pharma, Autolomous. Stay cool!

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In a late-stage trial, Insmed’s oral small molecule DPP1 inhibitor cut yearly exacerbation rates by around 20% in bronchiectasis patients, according to topline data. The biotech's shares soared more than 100% in reaction to the news. The biotech will continue analyzing data from ASPEN and will build toward a regulatory submission for brensocatib, which it plans to submit in the fourth quarter of 2024. The U.S. product launch is slated for mid-2025, pending approval. Designed to be taken orally, brensocatib is a small molecule inhibitor of DPP1, an enzyme that activates neutrophil serine proteases triggering lung damage and inflammation in various chronic immune-driven lung diseases. In addition to bronchiectasis, Insmed is also developing brensocatib for chronic rhinosinusitis without nasal polyps. #biotech #biopharma #bronchiectasis

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