Cour Pharmaceuticals Development Co., Inc.’s Post

📢 Discover cutting-edge non-alcoholic steatohepatitis (NASH) models for your #preclinical studies! As the incidence of NASH continues to rise, the selection of an appropriate NASH model is crucial for developing new treatments. Here at #GemPharmatech, we provide diverse NASH models. We offer genetically engineered mouse models with specific gene deletions (such as BKS-db NASH and B6-Alms1-del NASH), which have a more stable phenotype than the traditional induction model. We also provide a number of humanized mice that target NASH treatment targets (STK25, PNPLA3, DGAT2, and KHK) to support your drug development needs. Learn more here: https://lnkd.in/gASvT37v 🔗 BKS-db NASH: https://lnkd.in/gcJE6fRh 🔗 B6-Alms1-del NASH: https://lnkd.in/gjg2aRRR   #nonalcoholicsteatohepatitis #NASH #Research #NAFLD #antiNASHdrug #drugdevelopment #MetabolicDisease #mousemodel #NASHtreatment #biotech

In a groundbreaking move, the U.S. Food and Drug Administration (FDA) approved Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-editing therapy for treating sickle cell disease, Casgevy. The therapy uses the Nobel Prize-winning CRISPR-Cas9 gene-editing technology. The FDA also approved Lyfgenia, another cell-based gene therapy developed by Bluebird Bio, for sickle cell patients ages 12 and older with a history of vaso-occlusive events. For those in the sickle cell community, these breakthroughs represent hope for one day curing this debilitating, painful, and often life-threatening disease. The timing of these approvals could not have come sooner: In a recent survey we conducted, 87% of those with the condition were dissatisfied with their current treatment plan. Sickle cell disease affects more than 100,000 people in the United States and 20 million people worldwide. In the U.S., most people who have sickle cell disease are of African ancestry or identify themselves as Black. For relevant and up to date news, resources and patient perspectives on sickle cell disease, visit: bit.ly/46XJSVb #BioNews #SickleCellDisease #FDAApproval #Vertex #BluebirdBio

✅ Key Takeaways: ✳️ Small trial, but LDL was substantially maintained ✳️ LDL reduction- 69% ✳️ The investigational therapy in question, VERVE-101, is a CRISPR-based gene-editing drug designed to permanently turn off the PCSK9 gene in a patient’s liver. Previous animal studies had concluded that treatment could reduce PCSK9 levels by as much as 83% and reduce LDL cholesterol by as much as 69%. The animals in those studies were only given one dose of the gene-editing therapy, and their reductions in PCSK9 and LDL cholesterol have now been consistent for more than 2.5 years.

This is awesome! Transplant organs from engineered pigs: "The eGenesis donor kidney (EGEN-2784) used for this procedure is the company’s lead candidate for kidney transplant and carries three classes of genome edits: (1) knock out of three genes involved in the synthesis of glycan antigens implicated in hyperacute rejection, (2) insertion of seven human transgenes involved in the regulation of pathways that modulate rejection: inflammation, innate immunity, coagulation, and complement, and (3) inactivation of the endogenous retroviruses in the porcine genome. Without genetic modification, a porcine kidney would be immediately rejected by a human recipient." https://lnkd.in/eatmKGDc #biotechnology #patents #kidney

Super happy to share our new preprint on #MASLD and #Hsd17b13. Grateful for the work of PhD student Shehroz M. (now graduate), RNA-seq project of former PhD student Nicola Morrice , postdoc work of Dawn Thompson and great collaborators Dr George D. Mcilroy, Justin Rochford, Phil Whitfield, Sara Milanizadeh PhD, Sophie Wilson. We show that direct, liver-specific, shRNA-mediated knockdown of Hsd17b13 in high-fat diet-obese mice, markedly improved hepatic steatosis with no effect on body weight, adiposity or glycaemia. This was our lead project following our RNA-seq analysis of steatotic liver from obese mice -/+ Fenretinide treatment, which identified major beneficial effects of Fenretinide on hepatic gene expression including Hsd17b13. Lipidomic analysis of liver tissue revealed changes in phosphatidylcholines containing polyunsaturated fatty acids (PUFA) e.g. PC 34:3 and PC 42:10 suggesting a potential mechanism of Hsd17b13 biological function and role in MASLD. Should be of interest to number of companies targetting Hsd17b13 with RNAi approaches eg. Alnylam Pharmaceuticals. Looking for a good peer-review process, fingers-crossed.

Pushing the boundaries of what's possible in genetic research and therapy development. Recently published in *Brain* (2024), titled "A CAG Repeat Threshold for Therapeutics Targeting Somatic Instability in Huntington’s Disease." 🔬📜 New insights into the genetic thresholds could guide the development of targeted therapies for Huntington's disease. This paper not only enhances our understanding of the disease mechanism but also opens new avenues for therapeutic intervention that could potentially alter the course of this devastating illness. As we continue our research and collaborations, I am optimistic about the future of Huntington’s disease treatment. Stay tuned for more updates as we advance our mission to translate scientific discoveries into clinical solutions.  Let’s keep driving science forward for a better tomorrow!  https://lnkd.in/e_E6_d-2 #HuntingtonsDisease #Genetics #Biotech #RanchoBioSciences #ScientificResearch #Innovation 

Nice early evidence of clinical benefit from a Ph1/2 trial testing Moderna's mRNA candidate in propionic acidaemia, a rare genetic disorder which affects about one in 100,000 individuals worldwide and arises from mutations in either of two genes that encode an enzyme necessary for nutrient processing. Room for improvement, but there are thousands of diseases like this that are now within reach, thanks in no small part to rapid innovation needed to generate effective vaccines for COVID19. https://lnkd.in/ee-vHEWY

We are excited to announce that the US FDA has granted Orphan Drug Designation (ODD) to our BGT-OTCD program (https://lnkd.in/e_SQp8sR), an investigational liver-targeted AAV-LK03 gene therapy for the treatment of Ornithine Transcarbamylase Deficiency (OTCD). BGT-OTCD has previously received FDA Rare Pediatric Disease Designation in the US and Orphan Drug Designation in the EU. BGT-OTCD has gained momentum in recent months following the UK Medicines and Healthcare products Regulatory Agency’s (MHRA) approval of the clinical trial application for the initiation a phase 1/2 clinical trial in paediatric patients diagnosed with OTCD, which is expected to begin enrolment in the UK in Q3 2023. Read the full press release here: https://lnkd.in/gHNrq5-r Read more about HORACE here: https://lnkd.in/dFVkTnWQ Leigh Shaw Paul Gissen UCL Business Ltd UCL Technology Fund #genetherapy #rarediseases #orphandrugs #aav #metabolicdisorders #advancedtherapies #clinicalresearch #OTCdeficiency #ureacycledisorder 

#B_adolescentis may protect against #acute_liver_injury (failure) via its metabolite #hypaphorin. [ Mouse ] [ Preclinical Interventional Study ] - B. adolescentis generates microbial metabolite Hypaphorine. - Hypaphorine inhibits the inflammatory response and hepatic #Oxidative_stress to alleviate #Acetaminophen (APAP)-induced #Liver Injury (AILI) in mice. - Transcriptomic analysis indicates that Cry1 expression is increased in APAP-treated mice after Hypaphorine treatment. - Overexpression of Cry1 by its stabilizer KL001 effectively mitigates liver damage arising from #Oxidative_stress in APAP-treated mice. - Cry1 gene expression was also decreased in patients with APAP-induced acute liver failure.

Metaba is enthusiastic about beginning a pilot research program to apply our metabolomics expertise with the aim of better understanding cystic fibrosis and finding innovative solutions. Cystic fibrosis (CF) is a genetic disease affecting multiple organs, primarily the lungs and pancreas, caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. These mutations result in a dysfunctional CFTR protein that is unable to help move chloride to the cell surface resulting in thick and sticky mucus. This mucus can clog the airways and trap bacteria that lead to infections, inflammation, respiratory failure, and other complications. Approximately 40,000 individuals in the US and 105,000 worldwide are living with CF, cutting across all racial and ethnic groups. #CysticFibrosis #GeneticDisease #Awareness More information can be found at: Cystic Fibrosis Foundation https://lnkd.in/ePiXD5mg