Arrowhead Pharmaceuticals’ Post

🩸🧬 For the estimated 6,000 people in the US living with hemophilia B, a rare inherited bleeding disorder, as many as 60% have preexisting antibodies that could interfere with available therapy BEQVEZ. Luckily, FDA recently approved Labcorp's companion diagnostic nAbCyte™ Anti-AAVRh74var HB-FE Assay to help determine patient eligibility for treatment. "Labcorp is proud to offer the first cell-based, companion diagnostic to receive FDA approval, which represents a pioneering breakthrough in the field of companion diagnostics and will help transform the therapeutic landscape and the lives of patients living with rare, genetically inherited conditions," says Dr. Brian Caveney. Get the details here ⬇️ https://lnkd.in/eyFgvrpb #CompanionDiagnostics #CDx #CellTherapy #GeneTherapy

#GLP1 works great, but John Lepore, CEO at ProFound Therapeutics, says he knows it's not the only amino chain out there that can treat obesity. In fact, that's the premise of the company's approach to drug discovery: the previous definition of a protein-coding gene limited the development of new therapies. What if there were tens of thousands more protein-coding genes that we don't know about, and they're out there just waiting to be characterized? Catch John's story on this week's #BusinessOfBiotech. https://lnkd.in/e7JD_BB4 Here's a quick preview.

Join me in the latest episode of Preparing for a Cell and Gene Future. Hear from Parexel experts Jamie Pierson and Kim MacDonnell, BScN, MSc, RN, CGC to learn more about the increased adoption of accelerated regulatory pathways for fast tracking promising novel medicines through clinical development. Discussion includes the criticality of accelerated development in rare diseases, promising signals from the FDA and stakeholder engagement strategies to ensure broader access to new treatments. https://lnkd.in/eQTVaAnZ

Multiple guidelines recommend allopurinol as a first-line treatment, and because Asian people who carry the HLA-B*5801 gene experience severe cutaneous adverse effects when taking allopurinol, with a risk of morbidity that is 80-97 times higher than that of non-carriers, and a higher mortality rate. Studies in patients with hyperuricaemia and gout have shown that the frequency of the HLA-B*5801 gene is as high as 23-29%. Therefore, all major guidelines at home and abroad recommend that patients with hyperuricaemia and gout should be tested for HLA-B*5801 before using allopurinol in those at risk.

📢 bioMérieux and NecstGen collaborate to accelerate the development of #CellandGeneTherapies. 🧬 Ensuring the quality, safety, and efficacy of Cell and Gene Therapies (CGT) is crucial. Through our #partnership with NecstGen, CGT researchers and manufacturers gain access to our innovative solutions, enabling effective product and process monitoring. 🧑🔬 Our #Endotoxin Testing Solutions help you achieve GMP compliance and tackle advanced therapy challenges like sample sacrifice, complex compositions, and reducing vein-to-vein time, whilst helping you achieve your #sustainability goals. By breaking down testing bottlenecks, we accelerate CGT development and improve patient access to essential medicines. More information about our solutions: https://lnkd.in/edpxrbge

If you are at the Meeting on the Med Conference hosted by Alliance for Regenerative Medicine (ARM), come check out the two presentations about ImmPACT Bio’s work in developing therapies to treat cancer and autoimmune diseases. #CARTCellTherapy #Cancer #AutoimmuneDiseases

The latest episode of Preparing for a Cell and Gene Future addresses a rising trend in the adoption of accelerated regulatory pathways for fast tracking promising novel medicines through clinical development. Kim MacDonnell, BScN, MSc, RN, CGC and I, discuss the criticality of accelerated development in rare diseases, promising signals from the FDA and stakeholder engagement strategies to ensure broader access to new treatments. https://lnkd.in/g6a6dhsi

#NewResearch🔎 Researchers have developed a novel precision cellular #Immunotherapy for #NMDARencephalitis, a debilitating #AutoimmuneCondition. Using chimeric autoantibody receptors (CAARs), T-cells are genetically engineered to target and eliminate autoantibody-producing B-cells, presenting a promising therapeutic approach. The study highlights potential advantages, including specificity and reduced off-target effects. Clinical trials for related #CAARTtechnologies in autoimmune conditions are underway, demonstrating encouraging preliminary results. The findings offer a new avenue for precision-targeted therapies in #AutoimmuneDiseases. 📖 Read the full study here: https://lnkd.in/dRNXC3cs #Autoimmunity #MosaicofAutoimmunity

【Targeting AMPKa1 Gene in PC3 Cells by Triphenylmethanol Derivatives】 Full article: https://lnkd.in/gRiG-pbq (Authored by William Yaw Boadi, et al., from Tennessee State University, USA.) Many studies have indicated that diet in relation to certain lifestyles is one of the factors that affect cancer incidence and mortality. And analyses of some food consumptions have revealed the presence of some bioactive compounds such as the presence of flavonoids, carotenoids, lignans and other phenolic acids that have potential health benefits. This study investigate the potential effect of the #triphenylmethanol derivatives (TPMs), novel synthesized flavonoids, on total- and phospho-AMPKal levels in prostate cells (PC-3). Activation of AMP-activated protein kinase (#AMPK) has been reported to oppose tumor progression in diverse types of cancers. #Prodrugs #Polyphenols #Prostate_Cancer 

Mark Simon, Stifel, will moderate the Payer Perspectives on Drug Delivery panel at #PODD2024 Specifically, the panelists will address: ✔️ The growing number of self-insured employers that are considering restricting drugs based on cost alone is increasing ✔️ The future of weight loss drugs and GLP-1s ✔️ Potential factors influencing market access for gene therapies for more common conditions Learn more at https://lnkd.in/gQ_wSyBb #PayerPerspective #DrugDelivery #DrugDevelopment