Catherine Priest

Congratulations and good luck to Sanket Shah for launching Otsuka Precision Health, Inc. (OPH). With FDA's new PDURS framework, we are confident a playbook will evolve for molecules and digital medicine to co-exist. It's hard to dismantle molecules from digital medicine. Keeping that in mind, we always conducted our clinical trials to show how JOGO Health + pills provide better outcomes. In one of the clinical trials, we demonstrated that adding JOGO to migraine medications improved MIDAS(Migraine Disability Assessment) score significantly. Another trial demonstrated JOGO's foundational science reduced side effects of cancer medications and medication discontinuation. pill + bytes is where digital medicine is heading.

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In one of 2023’s more controversial decisions, the #FDA approved #Sarepta's #Elevidys as the first #genetherapy for #Duchennemusculardystrophy. Still, Sarepta is anticipating full FDA approval and a label expansion. https://lnkd.in/gKTZxVYv #genetherapies #ASGCT2024 #DMD #ASGCT

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Exciting news for those interested in inflammation and brain health! 🧠 I'm thrilled to announce that I'm working with Sage Cerebro to develop SageSignal™, the world's first at-home diagnostic that measures inflammation in your brain, also known as neuroinflammation. Why is this a game-changer? High neuroinflammatory levels are associated with a wide range of health concerns, including: - Anxiety 😰 - COVID brain fog 🌫️ - Attention and mood disturbances 😔 - Poor sleep quality 😴 Moreover, neuroinflammation is a key marker for identifying diseases such as Alzheimer's & Dementia, and it's also now effective at predicting your risk of stroke. 🩺 This incredible innovation comes from the brilliant minds at UCLA, including my colleague Jason Hinman's lab. Their tremendous work paves the way for accessible, at-home brain health monitoring. 👨‍🔬👩‍🔬 If you're interested in measuring your neuroinflammation from the comfort of your own home, join our waitlist at Sage Cerebro today! 🙌 Join the Waitlist → https://sagecerebro.com/ For more information, check out this article from the San Francisco Chronicle: https://lnkd.in/geTypdpF #SageCerebro #Neuroinflammation #BrainHealth #Alzheimers #Dementia #Stroke #Innovation #HealthTech

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📍PBMs ‘Wield Enormous Power’ Over Patient Access to Affordable Drugs: FTC The Federal Trade Commission released an interim report on Tuesday showing the power that pharmacy benefit managers exert over patients and their ability to afford and access prescription drugs. The report, part of an inquiry launched by the FTC in 2022, details how increasing vertical integration and concentration has allowed six of the largest pharmacy benefit managers (PBMs) to manage around 95% of all prescription drugs filled in the U.S. According to the report, the six most significant PBMs—Caremark Rx, Express Scripts, OptumRx, Humana Pharmacy Solutions, Prime Therapeutics and MedImpact Healthcare Systems—have achieved their dominance by becoming vertically integrated with upstream suppliers of goods and services, such as private drug labels and provider groups. PBMs are also linked to retail, mail-order and specialty pharmacies, according to the report. Last year the top three PBMs—CVS Health Corp.’s CVS, +1.28% CVS Caremark, Cigna Group’s CI, +1.42% Express Scripts, and UnitedHealth Group Inc.’s UNH, +0.57% Optum Rx—processed nearly 80% of the roughly 6.6 billion prescriptions dispensed by U.S. pharmacies last year, while the top six combined processed more than 90%, the report found. “The FTC’s interim report lays out how dominant pharmacy benefit managers can hike the cost of drugs—including overcharging patients for cancer drugs,” FTC Chair Lina Khan said in a statement. “The report also details how PBMs can squeeze independent pharmacies that many Americans—especially those in rural communities—depend on for essential care.” The report contends that PBMs have “substantial influence” over independent pharmacies by levying unfair, arbitrary or harmful contractual terms that can impact their ability to stay in business. This includes PBM contracts not reflecting the total payment amounts, making it difficult for pharmacies to determine how much they will get in compensation. The FTC claims that PBMs and brand pharmaceutical manufacturers are entering into agreements to exclude lower-cost competitor drugs from the PBMs’ formulary in exchange for increased rebates from the manufacturers. Kahn noted in her statement that FTC will continue to use all of its tools and authority to “scrutinize dominant players across healthcare markets and ensure that Americans can access affordable healthcare.” https://lnkd.in/d55FugPy #prescriptiondrugs #pharmacybenefitmanagers

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AAV is the leader of gene therapy. But what are it's pros and cons? Let's break it down. Pros: - Current leader in vector-based gene therapy - Payload doesn't integrate into patient DNA (normally) - FDA-approved therapies on the market - Can target specific tissues for precise delivery Cons: - High cost of manufacturing - Small payload size - Good quality at scale is a challenge - Single dose administration due to high immunogenicity (for now) - Liver toxicity concerns (for now) I'm excited to see how recent advances in capsid design will fix these drawbacks. Anything else you'd add? Drop it in the comments. For content that breaks down the science of CGT: Check out #cgt_simplified “Created with BioRender.com” #genetherapy #aav #biotechnology

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#FDA and the Office of the Chief Scientist commissioned Reagan-Udall Foundation for the FDA to use their "Regulatory Science Accelerator" to bring together industry and academic stakeholders to discuss needs for *in silico technologies*. The team originally thought they would develop the details for a "Good Simulation Practices" guideline. What instead they decided to work on, after their 3-month sprint of robust discussion, was to prepare an "activation paper, if you will, on simplifying but emphasizing the strategic imperative for the adoption of in silico technologies for FDA-regulated products. The audience: business decision-makers and regulatory affairs. Steven Kreuzer's post provides more details on the who and what. Check it out! Special thanks Susan Winckler for her dynamic leadership, and to Angie Hoth, Carolyn Hiller and Hannah Gray for their focus and project management support. Also, special mention to Khaled Bouri and Tracy Chen for their support during the accelerator sprints. #regulatory #medicalproducts #foodsafety #insilico #regulatoryscience #regulatoryaffairs

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Eagerly anticipating publication of FDA's final rule on laboratory developed tests, which is targeted by end of month. Great summary from Hyman, Phelps & McNamara, P.C.'s FDA Law Blog for those interested in learning the latest 🐝 #LDT #invitrodiagnostics #medicaldevices #regulatoryaffairs

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The merger of high tech and biotech is transforming healthcare and research. Innovations like AI-integrated labs on a chip enable the synthesis, analysis, and screening of DNA, RNA, and other molecules on microchips, accelerating complex molecular processes. This convergence is driving advances in personalized medicine and diagnostics, promising more efficient and precise treatments and research methods. The fusion of these fields is set to revolutionize our approach to biology and medicine, offering groundbreaking solutions for the future. Stay tuned for more on this exciting evolution!

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Our joint G2 Intelligence / Today's Clinical Lab webinar on the FDA's final LDTs rule is just over 24 hours away! I recently chatted via email with one of our speakers, regulatory expert Shannon Bennett, to get his initial thoughts on the final rule. Here's what he had to say about what was most surprising about the rule, what labs may find most challenging, and what labs should do right away to prepare: https://lnkd.in/gw3WuQjM #LDTs #FDA #ClinicalLaboratory #MedicalLaboratory

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evitria's recent article spotlight focuses on Tsuchikama et al.'s exploration of next-gen ADCS in cancer treatment. The review touches on recent advances in ADC components and highlights how bispecific ADCs have the potential to combat drug resistance It's a great reminder that antibodies are showing increasing promise in the realm of oncology and have the potential to enable multimodal treatments. Over the coming years, we're likely to see an increased research focus on them. Get in touch to discover the role evitria plays in supporting this research and how our services can help you. #ADCs #CancerTreatments #Antibodies #Oncology

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The white paper in the AAPS Journal by Amanda Hays, Mark Wissel, et al. is a comprehensive and detailed review, offering a wealth of well-thought-out recommendations. Its thoroughness and relevance to advancing Cell and Gene Therapeutics for development and approval will undoubtedly leave you feeling informed and confident in its content. It stands out for its unique contribution to Cell and Gene Therapeutics. It addresses the lack of information and guidelines for qPCR and dPCR. It brings together cross-industry conversations and an American Association of Pharmaceutical Scientists 2024 working group to fill these gaps. They acknowledge that the modalities for Cell and Gene Therapy have advanced rapidly and describe the inherent complexity that can overwhelm the method development and validation of these therapeutics and complicate the efficacy and safety of these therapeutics, which is critical. The white paper's practical approach is a testament to its value. It provides theoretical insights and offers a clear roadmap for implementing Cell and Gene Therapy using qPCR and dPCR as the bioanalytical platforms, as depicted in Figure 1. The emphasis on designing and validating assays to fit their intended purpose is a powerful tool that can directly support therapeutic development, making you feel empowered and capable of implementing these recommendations. The two central assay designs and development that I found informative were the design and selection of primers and probes for a bioanalytical PCR assay, which the paper goes into detail on, and designing single-nucleotide sequences (singleplex reactions) or multiple-nucleotide sequences (multiple reactions). They stated that the qPCR efficiency in multiplex vs. singleplex should be 90-110% with similar curve slopes, similar detection limits (LOD), and similar precision and accuracy performance. For multiplexing in dPCR, they say to ensure that the droplets/partitions can be distinguished between the two targets and that the fluorophores do not compete. In Table 1(pg. 8), they describe in excellent detail the advantages of qPCR and dPCR for Cell and Gene Therapy applications. They also propose evaluating the assay reagent and platform changes on a case-by-case basis and suggest that they be determined based on the context of use (COU). Lastly, the authors summarize the relevant publications and guidance documents on PCR validations in Table 4 (pages 15-21). This is a well-done evaluation of the different validation criteria. This white paper is a must-read for those involved in Cell and Gene Therapeutics development and validation. It's a valuable resource that directly addresses our needs and can significantly enhance our work. Please download the paper here: https://lnkd.in/eVVMSXvp

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Fresh off the presses and just in time for today's discussion about the future of proteomics at Assn of Biomolecular Resource Facilities (ABRF): a perspective piece on how mass spectrometry can -- and will -- coexist alongside "disruptive" next-gen proteomics platforms. Instead of thinking about "our" slice of (mass spectrometry-based) proteomics getting smaller from competition with new technologies, I am excited to see the proteomics market growing as more users join. We have such a wealth of knowledge and "lessons learned" to share. Collaboration, rather than competition, will grow proteomics, expanding the scope and impact of the entire field! https://lnkd.in/gtynYPDf #ABRF24 #TeamMassSpec #proteomics

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New vinylsulfonyl piperazine handle for DCF16 E3 ligase targeting covalent degraders.

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Among the multitude of single-cell technologies, which one should you choose? A comprehensive benchmarking study by a team from Genentech can help make that choice easier. They used PBMCs to evaluate the performance of droplet, microwell, and combinatorial indexing-based methods from various companies that offer single-cell technology. 07/08/24 edit: please see response from Singleron by Harlin Jhyont below in the comment. “This study provides a comprehensive and systematic evaluation of commercially-available scRNA-seq kits and sheds light on the various strengths and weaknesses of each kit with respect to time, cost, functionality and performance, which should facilitate an optimal and informed selection of a suitable scRNA-seq approach.” “Overall, 10x FRP exhibited the highest ranked performance followed by 10x 3’, BD, Scale, 10x 5’ / Parse (tie), Fluent, Honeycomb, and Scipio. Singleron was excluded from the cell-based comparisons due to unreliable cell calling; however, based on UMI count, gene count, and usable mapped reads, it exhibited the lowest performance at the library level.” https://lnkd.in/eTk3jYVQ #singlecell #science

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With portable, low-cost, and scalable platforms, #nanoporesequencing has revolutionized genomics, transcriptomics, and epigenomics. But did you know that nanopores are not just for DNA and RNA? A new field of protein sensing and sequencing through nanopores is emerging, and some of our close collaborators, including Hagan Bayley, Yujia Qing, and Jeff Nivala, are at the forefront. Check out this article to learn more about the groundbreaking work being done in this area. Protein Sequencing is coming. #science #proteinsensing #proteinsequencing Link to the article: https://lnkd.in/eEZG7a82

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Excited to share the latest feature in our "Partnering Spotlight" series, where we share some of the connections we are building to foster scientific innovation in healthcare. In this edition, our very own Neha Kaushal sits down with Daniel Anderson, Professor of Chemical Engineering at MIT, to discuss the potential of innovative #mRNA applications in vaccines and novel treatments for genetic diseases. Check out the video to learn more about our collaboration! https://lnkd.in/eHwpyjeY #vaccines #immunoscience

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Amazed at what Profluent is doing with generative AI, expanding the repertoire of gene editing tools with OpenCRISPR-1 and making them available for ethical applications! Way to go Ali Madani! https://lnkd.in/eF8Jq4rv

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Here's a new press release from Pacific Northwest National Laboratory on our recent work on the molecular effects of circadian misalignment (aka shift work) in humans!

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