Fractyl Health, Inc. (Nasdaq: GUTS) today announced that it will present new preclinical obesity data from its pancreatic gene therapy platform, Rejuva®, at the upcoming American Diabetes Association (ADA)’s 84th Scientific Sessions taking place June 21-24, 2024 in Orlando, FL. https://lnkd.in/euCWK-yh #ADA2024 #obesity #type2diabetes #biotech *The Rejuva platform is in preclinical development and has not yet been evaluated by regulatory agencies for investigational or commercial use.
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🗞️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 👇
Metabolic News Weekly: May 21, 2024
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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.
Fractyl Health Demonstrated That a Single Dose of a GLP-1-Based Pancreatic Gene Therapy Candidate (GLP-1 PGTx) Durably Maintained Weight Loss After Semaglutide Withdrawal in a Murine Model of Obesity at WCIRDC 2023 Annual Meeting | Fractyl
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Offering a chronic solution to a chronic problem? - Identification and targeting of a unique NaV1.7 domain driving chronic pain. While the researchers have attempted to design chronic pain drugs that directly block the channel, this has proven challenging because Nav1.7 is one member of a family of channels that appears throughout the body. In fact none of the blockers so far have blocked this channel without also blocking other related channels. The research presents a different approach of not trying to block the channel but to indirectly regulate its activity. The study identified a small peptide that blocks a pain-associated interaction between the sodium channel Nav1.7 and its regulatory protein collapsin response mediator protein 2 (CRMP-2) Using an adeno-associated virus to deliver code for the peptide into sensory neurons in vivo, the study demonstrates a potential gene therapy for long-term reduction or even prevention of chronic pain without the risk for addiction.
Identification and targeting of a unique NaV1.7 domain driving chronic pain | PNAS
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Partner - Biopharma and Advanced Therapies, DeciBio Consulting; Investor, BioTools Fund; Founder / Director, Suono Bio
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
U.S. FDA Approves Pfizer’s BEQVEZ™ (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B
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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
BREAKING: Ascidian’s RNA Exon Editor for Stargardt Disease Makes Historic First in Gene Therapy
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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
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💡 A 2024 paper on short stature with type 1 insulin-like growth factor receptor gene alteration. The study found that cases caused by gene deletions caused more severe growth impairment and were accompanied by developmental delay. The patients receiving growth hormone therapy showed improvement in growth rate without elevated serum IGF-I. Access the paper👇 https://ow.ly/xj7H50RELuy #MedEd #ShortStature #IGF-I
Clinical characteristics of and growth hormone treatment effects on short stature with type 1 insulin-like growth factor receptor (IGF1R) gene alteration - PubMed
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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
The Genetic Basis, Lung Involvement, and Therapeutic Options in Niemann–Pick Disease: A Comprehensive Review
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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
A homoeostatic switch causing glycerol-3-phosphate and phosphoethanolamine accumulation triggers senescence by rewiring lipid metabolism - Nature Metabolism
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Thoughts on this? >> Duchenne gene therapy SGT-003 wins rare pediatric disease status - Muscular Dystrophy News >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #pharmaceutical #pharma #biotech #competitivemarketing
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