Diana Albu

🚀 Exciting new product launches in Genomics! 🌟 Leading companies are unveiling groundbreaking products at the ASMS conference and beyond. Check out these innovations: 🔬 Thermo Fisher Scientific: Ardia software platform for mass spec workflows. 🔬 Bruker Optics: Scils Scope 1.0, GlycoScape, and OmniScape for mass spec imaging and protein analysis. 🔬 SCIEX: ZT Scan DIA method for Zeno trap. 🔬 Helix: Clinico-genomic datasets for precision medicine. 🔬 QIAGEN: FDA-cleared QiaStat-Dx Gastrointestinal Panel 2. 🔬 Illumina: XLeap-SBS chemistry for NextSeq 1000/2000. 🔬 Zymo Research Corp Research: PureRec DSN for DNA/RNA research. 🔬 Mission Bio: Tapestri Single-Cell Multiple Myeloma Multiomics Solution. These innovations are set to revolutionise research and healthcare! #Genomics #Biotech #Innovation #ASMS2024 #ThermoFisher #Bruker #Sciex #Helix #Qiagen #Illumina #ZymoResearch #MissionBio #LifeSciences #FutureOfHealth

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Check out our paper in the Journal of Medicinal Chemistry: “Discovery of Clinical Candidate PF-06648671: A Potent γ-Secretase Modulator for the Treatment of Alzheimer’s Disease”. We are grateful to everyone at Pfizer who contributed to this program over the years – especially to Christopher am Ende, Todd Butler, and Doug Johnson who were instrumental in getting this manuscript over the finish line. For those of you in the drug hunting community who may not be familiar with the pharmacological distinction between γ-secretase inhibitors (GSIs) and γ-secretase modulators (GSMs), here is a brief summary: - γ-Secretase Inhibitors (GSIs): This older class of compounds binds to the active site (or to an allosteric site) on the presenilin subunit of the γ-secretase complex resulting in inhibition of the enzyme. In addition to blocking formation of all forms of amyloid beta (Aβ) peptides, this approach also inhibits processing of many other substrates (including the Notch receptor) that are critical for normal cellular functions. Several γ-secretase inhibitors were advanced into clinical trials for Alzheimer’s disease quite some years ago, but these trials were terminated due to on-target toxicity. - γ-Secretase Modulators (GSMs): These compounds have a very different pharmacological profile compared to the GSIs. GSMs also bind to the presenilin subunit, but at allosteric sites that are distinct from those of the inhibitors. Rather than inhibiting the enzyme, GSMs modulate the enzymatic function and change the processivity to reduce formation of the longer, insoluble and neurotoxic Aβ peptides (including Aβ42) while increasing formation of the shorter, more soluble peptides, Aβ37 and Aβ38. Meanwhile, total Aβ remains unchanged. Most importantly, this class of compounds does not inhibit Notch processing. As a result, GSMs may be an effective approach to selectively reduce formation of the toxic Aβ peptides while sparing normal enzymatic function (see introduction of the paper for more info along with relevant references). https://lnkd.in/enHskGYp

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Umoja Biopharma Announces Publication in Blood Highlighting Successful Generation of In Vivo CAR T-cells in Non-Human Primate Modeling First landmark, peer-reviewed publication of successful in vivo CAR T-cell data in an immune competent NHP model Data validate the VivoVec™ platform in a translationally relevant model and support its transition into human clinical testing, offering a potential paradigm shift in the field of CAR T-cell therapies Key Study Findings VivoVec particles (VVPs) incorporating CD80 and CD58 costimulation proteins exhibit enhanced capacity for in vivo CAR T-cell generation and produce CAR T-cells with increased antitumor functionality compared to those produced from VVPs displaying anti-CD3 scFvs alone. Combining the anti-CD3 scFv together with the ligand binding domains of CD80 and CD58 into a single multi-domain fusion (MDF) protein markedly augments the particles’ ability to bind, activate, and transduce T-cells. VVPs incorporating MDF compatible with NHP T-cell activation and costimulation potently generate anti-CD20 CAR T-cells in vivo, comprising up to 65% https://lnkd.in/eqsSTKjf

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There is a new paper on DIA global, “The role of RWD & RWE in shaping global regulatory practices“, by Jeffrey Brown from TriNetX. It’s well written. I especially liked the “known unknowns” and “unknown unknowns” discussion, and “matching the expertise of RWE generator & reviewer”suggestion. Two comments: 1. FDA, EMA, PMDA were mentioned, NMPA was left out. In fact, seven RWD/E guidance were published by NMPA during the past years; NMPA has been a strong advocator of RWD/E. 2. RWE for regulatory consideration has a bright future with a rough path forward. So, for everyday practitioners, what are the suggestions between the miserable reality and bright future, especially for service providers, who need to deliver? That reminded me on an External Control Arm study a couple of years ago, where a “perfect” piece of evidence was not available, but we might be able to generate a chain of “good” evidences, combining both primary and secondary data. Is perfect the enemy of good for regulatory RWE studies?

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A great piece on the use of confirmatory evidence to demonstrate drug effectiveness and expedite the regulatory approval process (as per recent FDA guidelines). Quality and quantity of data sources used to substantiate clinical trials are equally important considerations when developing your evidence generation strategy. Be sure to download our white paper via the link below!

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Big news in lung cancer treatment! A new drug called ivonescimab from China appears to be effective against non-small cell lung cancer (NSCLC) 🇨🇳 This bispecific antibody is the first to outperform Keytruda, the current market leader, in a head-to-head trial #NSCLC #Keytruda Here's the breakdown: -Ivoncimab significantly extended progression-free survival (PFS) in patients compared to Keytruda -Experts are interested to see if this translates to an overall survival benefit, which is crucial for FDA approval #FDA -MSD , maker of Keytruda, acknowledges the potential of ivonescimab but highlights the need for more data #drugdevelopment The challenge? Ivoncimab was only tested in China, and the FDA may require additional trials for US approval #clinicaltrials This is a significant development in lung cancer treatment, and it will be interesting to see how it unfolds. Stay tuned for further updates! #cancerresearch #innovation

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RWE: External Controls in Rare Disease. Just talked to the first author, learned the story behind the paper. Amazing. Look forward to seeing your next paper on modified TMLE, Mark van der Laan!

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Demonstrating the technical leadership of SK pharmteco! 🧬 Catch up with our team at ASGCT to discuss how SKPT can support your #cellandgene development!

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As we go through 2024, clinical research recruitment is evolving rapidly, shaped by technology and a heightened focus on diversity. Here are five key trends that are expected to lead the way in enhancing recruitment strategies: 💡 Targeted Social Media Campaigns - Precision in reaching potential participants will improve with social media's powerful data analytics. 💡 Patient Advocacy Partnerships - Relationships with advocacy groups will help trials reflect the true demographics of conditions. 💡 Mobile and Telehealth Integration - Digital tools will extend recruitment reach, making participation easier and more accessible. 💡 AI-Powered Screening: AI will refine the screening process, offering quick and precise participant selection. 💡 Diversity and Inclusion Initiatives - A push for diversity will ensure trial data is comprehensive and applicable to all patient groups. As these trends gain momentum, they promise to open new avenues for recruitment, making clinical trials more efficient, inclusive, and representative of the populations they aim to serve. 😊 #skycrng #clinicaltrials #healthcare #futureofmedicine #therapies #research #healthcareresearch

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The biopharma industry is rebounding in 2024. Investments in cutting-edge technologies like antibody drug conjugates and AI, along with strategic alliances, are shaping the future of medicine. Henry Levy discusses how the sector is poised for transformative advancements in healthcare. https://ow.ly/ybSP30sCzb2 #Biopharma #PharmaInvestments #FutureOfMedicine #Healthcare #Biotech

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BDRA Consulting LLC has been a member of AMDM since 2013. Come join AMDM for the entire story. Twice a year, members get to meet with the FDA to discuss upcoming regulatory and quality policies. The FDA provides industry updates. This year's meeting was held at Building 66, White Oak campus on May 21, 2024. Key Takeaways from the May 2024 AMDM Industry FDA Roundtable: 1. Cybersecurity for medical devices is a key issue. OHT7 and CDRH have increased focus on cybersecurity for medical devices. 2. The Oncology Diagnostic Pilot Program is nearly a year old, and no company has been accepted. This is primarily because drug manufacturers have not been able to provide validation data. 3. Reclassification plans for companion diagnostics are ongoing and will be completed by November 2027. - FDA is considering down-classifying three types of CDxʼs: i. Nucleic acid-based CDX ii. In situ hybridization-based CDX iii. Immuno-histochemistry-based CDX - Technologies will be grouped based on the intended use. 4. PCCP guidance for medical devices will be published before the end of the fiscal year 2024. A PCCP may be used to make changes to sample type changes, which may be acceptable in certain cases. 5. QSIT is being replaced by an entirely novel risk-based approach, which is currently being worked out. Manufacturers should be working through transition plans. #AMDM

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Understanding the complex biomarker landscape is key to advancing Alzheimer's disease research. Our recent webinar sheds light on core and emerging biomarkers for amyloid, phospho-tau and neurodegeneration (ATN), offering key insights for those working to identify and validate diagnostic and prognostic biomarkers. Watch the full recording to hear from our scientific leaders. #Alzheimers #Biomarkers #Neurodegeneration #Amyloid

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Read our Iatest blog to learn more about the harmonization of drug-drug interaction (DDI) guidelines, and the impact this will have across drug development. The new ICH M12 aims to simplify the process by providing a single set of guidelines for the designing, conducting and interpreting metabolic enzyme- or transporter-mediated drug-drug interaction (DDI) studies. The guideline covers both in vitro and clinical DDI studies This blog will focus on the differences with the previous FDA 2020 DDI guidelines.

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🔬 Discovering Biomarkers for Better Outcomes in Melanoma Treatment 🔬 Exciting news from ASCO 2024! Led by Jeffrey Weber, our team is proud to present pioneering findings on the role of predictive and pharmacodynamic biomarkers for response and toxicity in melanoma treatment. Our phase II trial combined tocilizumab (TOCI) with immune checkpoint inhibitors, reducing toxicity and revealing new biomarkers. Initiated in 2020-21, this work involved multiple high parametric flow cytometry panels to identify various myeloid, NK and functional CD4 and CD8 T cell populations linked to treatment outcomes. Our insights are paving the way for more personalized, effective melanoma management. 🔥 Hot Take: Inflammation Modulators are Here to Stay in Immuno-Oncology!🔥 📍 Check out our findings at ASCO Poster Abstract #9538; Poster Board #322. #ASCO2024 #MelanomaAwareness #TranslationalBiomarkers #TranslationalResearch

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Looking forward to an exciting discussion on how AI and ML are reshaping the world of Drug Development. Come join me!!!

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Happy Friday! Our team at Vesigen Therapeutics, Inc. is excited to share progress on both our platform activities and drug discovery programs next week at the ASGCT and ARVO conferences. If you're attending, please come by to say hello. Our posters are listed below: https://lnkd.in/egjcCjJP https://lnkd.in/eJpg6Y5G #human #nonviral #delivery #genomeediting #targeted #ocular #immunology #neurological #therapeutics

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Exciting news to share for #biotech, #biopharma, and #cdmo organizations looking to elevate the safety and efficacy profile in related #biologics, #genetherapy, #celltherapy, and #tissueengineering sectors.

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