Fractyl Health’s Post

🗞️In this week's edition of Vault's "Metabolic News Weekly," our team has curated several impactful studies and developments in metabolic health: ⏵ A novel approach using glucagon-like peptide-1 to treat obesity shows promising results in mice. ⏵ Advances in liver disease research with a new liver-on-a-chip model. ⏵ Promising preclinical results for a new #GLP-1 gene therapy in obesity treatment. ⏵ Analysis of blood osteocalcin’s potential role in reducing Alzheimer's disease risk through improved metabolism. #MetabolicHealth #ResearchUpdate #HealthcareInnovation For a comprehensive read on these articles and more in recent metabolic news, check out the full post 👇

We are thrilled to have presented preclinical findings from our Rejuva® gene therapy platform at the World Congress Insulin Resistance Diabetes and Cardiovascular Disease 2023 Annual Meeting. The oral presentation, titled “Single-dose GLP-1-based Pancreatic Gene Therapy Maintains Weight Loss After Semaglutide Withdrawal in a Murine Model of Obesity,” highlighted the promising implications of the Rejuva platform in providing a durable therapeutic solution for obesity with a GLP-1-based pancreatic gene therapy candidate (GLP-1 PGTx) designed to target the pancreas to provide long-term metabolic benefits from a single administration. #WCIRDC23 #T2D #obesity https://lnkd.in/eAKF-d_Z maintained-weight-loss-after-semaglutide-withdrawal-in-a-murine-model-of-obesity/ *The Rejuva platform is in preclinical development and has not yet been evaluated by regulatory agencies for investigational or commercial use. 

#CGT #PlasmidDNA CGT (Cell and Gene Therapy) is considered one of the important industry for future medical research. It offers the possibility of providing cures for cancer, genetic diseases, and rare disease patients. In recent years, the research and development pipeline for CGT has been growing rapidly. According to the statistics, there are a total of 5,814 CGT research and development projects globally, with 4,881 of them being actively in development. The outlook for the commercialization of CGT products is also quite optimistic, with a total of 41 CGT products having been launched between 2015 and 2022. Plasmid DNA is the highest-cost component in CGT production, with GMP-grade plasmids priced as high as $10,000 to $30,000 per gram. Most downstream CGT companies choose to directly purchase ready-made plasmids and collaborate with CDMO companies that offer fast production, high yields, and excellent quality. Some representative CDMO companies in China include GenScript, Wuxi AppTec, Obio, and WuXi Biologics, among others. The key to further reducing costs while ensuring plasmid quality lies in domestic production substitutes and process optimization. The CGT industry is transitioning from a focus on customized therapies for blood cancers, rare diseases, and specialized applications towards more generalized treatments for solid tumors and common diseases. Some related products have already entered Phase II clinical trials. As the scope of indications broadens and more universal CGT products become available, the market demand is expected to increase, which should lead to further cost reduction in the large-scale GMP-grade plasmid production.

#PlasmidDNA CGT (Cell and Gene Therapy) is considered one of the important industry for future medical research. It offers the possibility of providing cures for cancer, genetic diseases, and rare disease patients. In recent years, the research and development pipeline for CGT has been growing rapidly. According to the statistics, there are a total of 5,814 CGT research and development projects globally, with 4,881 of them being actively in development. The outlook for the commercialization of CGT products is also quite optimistic, with a total of 41 CGT products having been launched between 2015 and 2022. Plasmid DNA is the highest-cost component in CGT production, with GMP-grade plasmids priced as high as $10,000 to $30,000 per gram. Most downstream CGT companies choose to directly purchase ready-made plasmids and collaborate with CDMO companies that offer fast production, high yields, and excellent quality. Some representative CDMO companies in China include GenScript, Wuxi AppTec, Obio, and WuXi Biologics, among others. The key to further reducing costs while ensuring plasmid quality lies in domestic production substitutes and process optimization. The CGT industry is transitioning from a focus on customized therapies for blood cancers, rare diseases, and specialized applications towards more generalized treatments for solid tumors and common diseases. Some related products have already entered Phase II clinical trials. As the scope of indications broadens and more universal CGT products become available, the market demand is expected to increase, which should lead to further cost reduction in the large-scale GMP-grade plasmid production.

A homoeostatic switch causing glycerol-3-phosphate and phosphoethanolamine accumulation triggers senescence by rewiring lipid metabolism. Cellular senescence affects many physiological and pathological processes and is characterized by durable cell cycle arrest, an inflammatory secretory phenotype and metabolic reprogramming. Here, by using dynamic transcriptome and metabolome profiling in human fibroblasts with different subtypes of senescence, we show that a homoeostatic switch that results in glycerol-3-phosphate (G3P) and phosphoethanolamine (pEtN) accumulation links lipid metabolism to the senescence gene expression programme. Mechanistically, p53-dependent glycerol kinase activation and post-translational inactivation of phosphate cytidylyltransferase 2, ethanolamine regulate this metabolic switch, which promotes triglyceride accumulation in lipid droplets and induces the senescence gene expression programme. Conversely, G3P phosphatase and ethanolamine-phosphate phospho-lyase-based scavenging of G3P and pEtN acts in a senomorphic way by reducing G3P and pEtN accumulation. Collectively, our study ties G3P and pEtN accumulation to controlling lipid droplet biogenesis and phospholipid flux in senescent cells, providing a potential therapeutic avenue for targeting senescence and related pathophysiology. Source: Nat Metab https://lnkd.in/e9nKYg5s

Breaking news in #retina #genetherapy - Ascidian Therapeutics' new candidate for Stargardt disease has IND tag + Fast Track designation from the FDA, giving it the greenlight to go into the clinic. Interesting implications that go beyond eye care and Stargardt here, as their new approach to gene editing - what they call exon editing - has some tantalizing applications far beyond inherited retinal diseases. Check out our article in PIE Magazine for the full scoop. https://lnkd.in/gsQ8ADsF #eyecare #retinopathy #ophthalmology

Pfizer Receives Approval For.... Beqvez (AAV-based medicine) for hemophilia B. This is the second approved AAV therapeutic for hemophilia B, following CSL's Hemgenix. The therapy was approved in Canada earlier this year. All eyes will be on the commercial launch and ramp of this innovative medicine. Leveraging a novel capsid from a collaboration with Spark (Roche), the medicine inserts a functional copy of Factor IX. CSL's product uses an AAV5 serotype. AAV commercial launches have historically been slower than expected. Sarepta's Elevidys could be bucking this trend and certainly the industry will be watching Beqvez to see if it can also scale up dosing. Ready to delve deeper into the world of gene therapy and precision medicine? Connect with us (we'll be at #ASGCT) to explore innovative strategies for navigating this evolving landscape and discover how we can help drive your success in the era of genetic medicine. #genetherapy #pharma #FDA https://lnkd.in/d5k9vQTa

Thoughts on this? >> Duchenne gene therapy SGT-003 wins rare pediatric disease status - Muscular Dystrophy News >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #pharmaceutical #biotech #pharma #healthcare #competitivemarketing

Thoughts on this? >> Pediatric Neuron Disease Gets Its First Gene Therapy - Medpage Today >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #biotech #pharma #healthcare #competitivemarketing #pharmaceutical

Niemann–Pick Disease (NPD) type C is a neurological disorder caused by mutations in the NPC1 or NPC2 genes, affecting cholesterol trafficking and esterification. Despite efforts to find effective therapies, only limited options are available, with miglustat being the sole approved disease-modifying treatment. This review provides an overview of the genetic basis and lung involvement in NPD, focusing on clinical, radiologic, and histopathologic features, and current and future therapeutic strategies. Cyclodextrin treatment is highlighted as a potential future therapy, along with arimoclomol, acetyl-L-leucine, and gene therapy. This paper is recommended for those interested in the various types of Niemann-Pick diseases. For a deeper insight, click on the link and read Eva Fenyvesi's blog post on Cyclodextrin News: https://lnkd.in/dwj82s4t #cyclolab #cyclodextrin #cyclolab #cyclodextrin #NiemannPickDisease #NPC #raredisease #neurological #neurologicaldisorder #treatment #therapy #miglustat #pharma #pharmaceutical #pharmaindustry #science #chemistry #research