French Lewis, III’s Post

Regulatory genetic toxicology tests focus on detecting mutagens, clastogens, and aneugens to assess substance safety. However, early-stage discovery benefits from screening for genotoxic hazards first, with mechanistic insights as a bonus. Substances that cause gene mutations or chromosomal changes also activate DNA damage response and repair pathways. These pathways are useful biomarkers for quickly and cost-effectively assessing potential genotoxic hazards. Find out more here: https://lnkd.in/efqP3C59

𝐔𝐒𝐅𝐃𝐀 𝐠𝐢𝐯𝐞𝐬 𝐭𝐞𝐧𝐭𝐚𝐭𝐢𝐯𝐞 𝐚𝐩𝐩𝐫𝐨𝐯𝐚𝐥 𝐭𝐨 𝐋𝐮𝐩𝐢𝐧’𝐬 𝐝𝐫𝐮𝐠 𝐟𝐨𝐫 𝐭𝐫𝐞𝐚𝐭𝐦𝐞𝐧𝐭 𝐨𝐟 𝐫𝐚𝐫𝐞 𝐠𝐞𝐧𝐞𝐭𝐢𝐜 𝐝𝐢𝐬𝐨𝐫𝐝𝐞𝐫 📌 Lupin has received tentative approval from the United States Food and Drug Administration (U.S. FDA) for its Abbreviated New Drug Application for Migalastat Capsules, 123 mg. 📌 The approval has been granted to market a generic equivalent of Galafold Capsules, 123 mg of Amicus Therapeutics US LLC. 📌Migalastat Capsules are indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data. #lupin #usfda #geneticdisorder

In this weeks #Fridayread, Marsee et al from Bart Spee's lab at Universiteit Utrecht describe a novel hepatobiliary organoid system. The need for primary human hepatocytes in ex vivo toxicology studies is limiting, due to the limited expansion potential of primary cells. The formation of organoids can increase hepatocyte functions and proliferation. In this publication they describe a novel method for mass generation and long-term expansion of functional hepatobiliary organoids expressing hepatocyte markers ALB, ECAD, HNF4A and ASGR1. They then demonstrated the usefulness of these organoids in disease modeling by inducing disease states cholestatis (stalled bile flow) and steatosis (fat build up). For applications such as toxicology testing, the cells were gene-edited prior to organoid generation. The methods could also be used for patient-specific therapies. Read the full paper > https://zurl.co/TK3O Read more about Qkine R-spondin 3 (Qk032) used in this publication > https://zurl.co/g0aj #hepatocytes #organoids #research

Longitude portfolio company Inozyme Pharma, which focuses on developing novel therapeutics for rare diseases, recently shared that the U.S. FDA has granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency. INZ-701 is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In pre-clinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in devastating genetic disorders such as ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. Learn more about INZ-701 and this major milestone: https://lnkd.in/er5Csmbf #biopharma #rarediseases

How does it look like to be part of the Toxys team? How do different members of our team contribute to our mission? What kind of methods do they use daily? If you are curious about the answers, we invite you to follow our “Working at Toxys” series based on interviews with our team. We first asked Marit Hoogenboom-Geijer, Scientist for genetic toxicology R&D and Study Director for MutaTracker, about her work. Click on the link below for the article. 👇 https://lnkd.in/difEnSvi

Step 3 is Testing in Models. HNF develops and validates CMT cell and animal models to test the efficacy of viable treatment approaches. This includes toxicology and dosing studies. In 2023, we've established 2 Rat Models, and 3 Cell Models. Our new #CMTBiobank collects patients’ biospecimens to accelerate crucial biomarker development. Since our #Biobank launch in August 2023, #HNF has collected over 65+ Biospecimens across 5 Cities and has supported 3 Exploratory Biomarker Studies. #cmtwegotthis #cmtawareness #cmtresearch #hnf4cmt #charcotmarietoothdisorder #hnf #charcotmarietooth #cmtneuropathy #genetics

Inspiring breakthrough: Sandeep Prabhu, professor of immunology and molecular toxicology and head of the Department of Veterinary and Biomedical Sciences, sheds light on how dietary selenium can combat dangerous stem cells. Researchers in the College of Agricultural Sciences who recently revealed how this trace mineral — found naturally in varying amounts in many foods — can hasten the death of leukemia stem cells. Their findings, published in Cell Reports, could help lead to drug therapies that target acute myeloid leukemia, or AML, the most common blood and bone marrow cancer in adults. Learn more: https://ow.ly/merb50RejEl #Immunology #Toxicology #Research #PennState

Phyllanthus tenellus Roxb. and Kaempferia parviflora Wall. ex Baker compounds as inhibitors of SARS-CoV-2 main protease and RNA-dependent RNA polymerase: A molecular docking study. https://ow.ly/UZr850Qx3xw #Pharmacy #Pharmacology #NaturalProduct #InfectiousDisease #COVID-19

Ruth Roberts, Michael Morton, Laurence Bishop, Nicholas James Coltman, PhD, Kimberly Rockley, Will Humfrey, Heather Brown and Abigail Walker BSc, MSc will be at British Toxicology Society (BTS) Discovery Toxicology 2024. Kim is presenting a poster "Benefits of early in vitro screening for seizure liability in problem solving and decision making" Will is presenting and giving a rapid-fire talk on "The value of gene expression data as a surrogate for Human Protein Atlas immunohistochemistry for assessing on-target off-tumour toxicity in target safety assessment" Nic is presenting a poster "Target safety assessment of unintended cereblon-based degrader neosubstrates reveals a wide range of potential organ toxicities" Hope to see you there #drugsafety #drugdiscovery

Phyllanthus tenellus Roxb. and Kaempferia parviflora Wall. ex Baker compounds as inhibitors of SARS-CoV-2 main protease and RNA-dependent RNA polymerase: A molecular docking study. https://ow.ly/vQc250Qx3xz #Pharmacy #Pharmacology #NaturalProduct #InfectiousDisease #COVID-19