A potential cystic fibrosis therapy with higher efficacy and a less-frequent dosing regimen than other mRNA approaches? We're excited to share promising preclinical data that suggest that Sail’s Endless RNA? (eRNA?) may offer a new type of treatment for those with cystic fibrosis, including those for whom existing therapies are not an option. The Pioneering Medicines initiative of Flagship Pioneering will present a poster at #NAFC2024 on the effects of lab treatment with CFTR endless RNA on respiratory cells. Learn more about the Cystic Fibrosis Foundation funded research: https://lnkd.in/gJXtMsjz #eRNA #RNA #endlessRNA #CF #cysticfibrosis
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?? #D2RProjectSpotlight: "B-cell derived extracellular vesicles with enriched miRNA contents that mitigate Th2 inflammation" led by Bruce Mazer, Research Institute of the Centre universitaire de santé McGill | McGill University Health Centre. Co-Investigator: Janusz Rak (Research Institute of the McGill University Health Centre) Collaborator: Yasser Riaz Al-Hosseini (McGill University) This project aims to develop and test laboratory-produced B-Cell-derived extracellular vesicles (B2-EV) as a potential low-toxicity therapy for severe asthma by targeting multiple genes through miRNA in preclinical mouse models. ?? Learn more: https://buff.ly/3Nzempi #DNA2RNA #AsthmaTreatment
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Developing liver-targeting exosomes as anti-TGF-β nanocarriers to treat the pre-metastatic niche: in this recent work, Paloma A., Pierrick Fournier at CICESE and collaborators developed a liver-targeted anti-TGF-β therapy using functionalised αVβ5+ exosomes. By overexpressing the integrin αVβ5 in 293T cells, they enhanced exosome accumulation in the liver of mice. To load these exosomes with anti-TGF-β agents, they engineered 293T cells to express either a short hairpin RNA against Tgfb1, silencing its expression in recipient cells, or an mRNA encoding sBG, enabling recipient cells to secrete a TGF-β-neutralising agent. https://lnkd.in/e5QB2746 Their study highlights the potential of αVβ5+ exosomes as innovative liver-targeted nanocarriers, using their cargo delivery capabilities to administer anti-TGF-β therapies while minimising systemic toxicity. An article co-authored by Eréndira Olvera Félix, Veronica Castro Flores, Arturo Hernández García, Ruben D. Cadena-Nava, Octavio Galindo Hernández and Patricia Juárez Camacho #extracellularvesicles #exosomes #drugdelivery #integrin #Vesiculab
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A novel mRNA therapy developed by researchers at Mass Eye and Ear has proven successful in preclinical studies in reducing excess scar tissue and preventing the formation of new scar tissue caused by proliferative vitreoretinopathy. This marks the first mRNA therapy delivered directly to the eye. The researchers plan to refine the treatment and ramp up animal testing, with the hope of moving toward human clinical trials in the future. Learn more about the study findings here: https://lnkd.in/gCR-9WEA #Ophthalmology #PVR #EyeDisease #GeneTherapy #MediaMICE?
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Heading to #SITC2024 in Houston this week? Adaptive Biotechnologies Corp.'s deep #Tcell and #Bcell receptor sequencing and comprehensive data informs the development of innovative immune-mediated therapies across modalities and indications. Message me if you want to meet! #SITC #cancerresearch #immunotherapy
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Here our latest publication on targeting the pathogenic exon 1 HTT protein in Huntington disease models. Thank you to all the co-authors and collaborators and special thank to Melvin Evers, Astrid Valles Sanchez and Sander van Deventer for their support and teamwork to make this possible! I feel honored to have been part of this research on AMT-130, an advanced gene therapy with the potential to make a difference in HD patients. https://lnkd.in/dHCfpJcf
uniQure has published the paper “Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models” in the December issue of Brain, describing the development of AMT-130 to target the HTT exon 1 sequence. Unlike most other programs in development, AMT-130 targets the accumulation of the exon 1?HTT fragment, the most toxic source of abnormal protein aggregation in HD. Preclinical testing in mouse models showed that AMT-130 reduced both full-length HTT and HTT exon1 mRNA and protein levels, improving HD-like symptoms, suggesting that AMT-130 could be a promising therapy for HD and potentially offer a broader therapeutic benefit by targeting both forms of the toxic HTT protein.??https://lnkd.in/eUA77vDS
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uniQure has published the paper “Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington's disease models” in the December issue of Brain, describing the development of AMT-130 to target the HTT exon 1 sequence. Unlike most other programs in development, AMT-130 targets the accumulation of the exon 1?HTT fragment, the most toxic source of abnormal protein aggregation in HD. Preclinical testing in mouse models showed that AMT-130 reduced both full-length HTT and HTT exon1 mRNA and protein levels, improving HD-like symptoms, suggesting that AMT-130 could be a promising therapy for HD and potentially offer a broader therapeutic benefit by targeting both forms of the toxic HTT protein.??https://lnkd.in/eUA77vDS
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In the June issue of Drug Development & Delivery, Avidity’s Distinguished Scientist and Strategic Leader, Art Levin, PhD, discusses how we are developing a new class of RNA therapeutics with the potential to precisely target and modify the underlying genetic drivers of diseases. At Avidity, we’re leveraging our proprietary AOC platform to advance our clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (#DM1), facioscapulohumeral muscular dystrophy (#FSHD), and Duchenne muscular dystrophy (#DMD). In the last year, we have shared exciting data from all three programs, further reinforcing the promise of our AOC platform to profoundly improve people's lives by revolutionizing the delivery of RNA therapeutics. Read the full article: https://lnkd.in/eq-MCCVf
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Managing Director, ACF Equity Research, Board Director at ORACA Independent Equity Research
#StemCells Oral #ALStherapy by #TransposonTherapeutics (TPN-101 PIIa trial) shows safety & slows lung function decline over w/ 1yr’s treatment in ALS C9orf72 mutations. Also see NurOwn PIIIb - Read our #ACFResearch update on $BCLI https://lnkd.in/e2sThM9S The ALS Association ALS TDI
#ASLTreatment - Motor Neuron Disease - recent scientific research showed that Allogeneic B cell immunomodulatory therapy benefitted lab models, i.e. laboratory animals. As a result, the researches got permission from the FDA and Massachusetts General Hospital to try this therapeutic approach in an individual w/ #ALS. There are a range of alternative approaches to developing ALS therapies. We particularly like MSCs including $BCLI's NurOwn, its P3b trial has just begun rollout - of course $BCLI needs further funding to complete, but it is an interesting area with some promise of marked progress in therapy effectiveness for early to mid-stage ALS sufferers, if BCLI's P3b is a success. To see where $BCLI is right now you can read our #ACFResearch #NurOwn Update https://lnkd.in/dFgfxHpk $AZN $AMGN $SNY $GILD $VRTX $ABBV $AMRX $ROG.SW $MRK $PFE $TEVA $NVS $BIIB. Brainstorm Cell Therapeutics Pfizer AbbVie ALS TDI The ALS Association ALS CURE Project
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Highlighting a decade of NIH Common Fund support advancing extracellular RNA biology: in this review article, Sara Amolegbe, Christine Happel, PhD at The National Institutes of Health and collaborators examined the achievements of the NIH Common Fund exRNA Communication program and the growth of exRNA as a scientific field, analysed through scientific publications and NIH funding trends. ExRNA and its carriers hold clinical promise as biomarkers, diagnostics, and therapeutics. Notable translational advancements include the adaptation of exRNA technologies for COVID-19 diagnostics, the clinical adoption of extracellular vesicle-based biomarker assays, and the development of exRNA carriers for drug delivery https://lnkd.in/ei_xh6hJ This comprehensive analysis showcases how breakthroughs in exRNA biology are being successfully translated into clinical and commercial applications, underscoring the significant impact of the NIH Common Fund program. An article co-authored by Nicolas C. Johnston, Angela Ambrosi, Aniruddha Ganguly, T. Kevin Howcroft, Lillian S. Kuo, Patricia L., Dobrila Doda Rudnicki, John Satterlee and Danilo Tagle #extracellularvesicles #exosomes #NIH #exRNA #Vesiculab
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This project will be very helpful to all the #extracellular vesicle and #exRNA scientific community. Choosing the right antibodies and assays is a whole different ballgame when dealing with extracellular molecules. #ev #antibody #atlas
Creating a robust antibody database optimised for the analysis of extracellular vesicles: Al Charest at Beth Israel Deaconess Medical Center, Louise Laurent at UC San Diego, Roger Alexander at Caris Life Sciences and collaborators, the investigators of the Extracellular RNA Communication Consortium (ERCC), aimed to collectively promote the dissemination of findings concerning antibodies pertinent to EV & and other extracellular nanoparticles research by developing the EV Antibody Database https://lnkd.in/eqJYF5EN Hosted on the ExRNA Portal (https://lnkd.in/e4JJc_sk), this dynamic database compiles and disseminates results from antibodies tested by research groups within the EV/ENP domain. An article also authored by Amber Morey, Martin Ng, Michail Spanos, Piyan Zhang, Tuoye Xu, Willi Cheung, Emeli Chatterjee, Priyanka Gokulnath, PhD, Natacha Carnel-Amar, Ana Luisa Soares Chiaretti, Collin Nelson, Jubin George, Michelle Luo, Abhik Chakraborty, PhD, Luiza Perucci, Jennifer C. Jones, Peter De Hoff, jeff Franklin, Robert L. Raffai, Saumya Das, David Routenberg and John Nolan #extracellularvesicles #exosomes #antibody #flowcytometry #Vesiculab
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