YolTech Therapeutics

This Thursday, YolTech Therapeutics' Co-founder, CDO & CTO, Dr. Emma Wang, will be speaking at the TIDES Euro 2024 event. Don't miss this chance to hear about our latest advancements in gene editing therapies! #TIDSEuro2024 #YolTech #GeneEditing #InVivoTherapies #ClinicalTrials #Innovation

Gene editing for hematopoietic stem cells (HSCs) has proven transformative for curing blood disorders like β-thalassemia and sickle cell disease (SCD). Despite successes, current approaches require intensive preconditioning and come with high costs and potential toxicity. A simpler, safer method is emerging: in vivo RNA delivery directly to HSCs. A recent breakthrough from the University of Pennsylvania demonstrated base editing of mouse HSCs using CD117 antibody-modified lipid nanoparticles (LNPs) for Cas9 mRNA delivery. While effective, human translation remains a challenge. On November 1, 2024, YolTech scientists, in collaboration with East China Normal University, published promising research on bioRxiv. We developed a novel antibody-free LNP system that efficiently delivers base editor ABE8e mRNA to bone marrow cells, enabling precise HBG gene editing to boost fetal hemoglobin (HbF) expression—offering potential treatments for β-thalassemia and SCD. Our innovative lipid libraries yielded the highly efficient LNP-168, achieving a 48.5% base editing rate in bone marrow cells. By incorporating microRNA-122 elements, we also minimized off-target effects in the liver. This work marks a significant step toward clinical gene therapies, simplifying production and expanding treatment possibilities. YolTech remains committed to advancing HSC-based therapies and improving outcomes for patients worldwide. #YolTech #GeneEditing #BaseEditing #LNP #BloodDisorders #InnovationInMedicine #HSCs #ScientificBreakthrough

🚀Exciting Progress in Gene Therapy for Blood Disorders We’re thrilled to announce a new collaborative study by YolTech and East China Normal University, recently published on BioRxiv! This breakthrough research demonstrates the successful in vivo delivery of base editor mRNA to bone marrow and hematopoietic stem cells (HSCs) via YolTech’s proprietary lipid nanoparticle (LNP) system, opening up transformative possibilities for treating blood disorders like β-thalassemia and sickle cell disease. 🔬 What does this mean? Using our innovative LNP platform, a single or repeated IV injection can now deliver mRNA directly to bone marrow cells, achieving precise gene editing. This method activates fetal hemoglobin production in adults, a major step toward effective, low-burden treatment options for monogenic blood disorders. 🌱 🌍 Why it matters? - Direct delivery to bone marrow and HSCs, bypassing complex cell therapy steps   - Precision editing with high fetal hemoglobin activation   - Potential to improve patient outcomes with fewer treatments We’re proud to share this advancement and the potential it holds for transforming patient lives through cutting-edge gene therapy. #GeneTherapy #GeneEditing #Hemoglobinopathies #YolTech #InnovationInHealthcare

YolTech is excited to attend BIO-Europe 2024, Europe’s premier biotechnology event, taking place in Stockholm, Sweden, from November 4–6! Our VP of Business Development and Investor Relations, Mr. Lin Jian, will represent YolTech to showcase our advanced gene therapy pipeline and commitment to innovation. We look forward to connecting with international biotech companies and exploring new opportunities for collaboration and growth! #BIOEurope #YolTech #GeneTherapy #Biotech

YolTech has been granted a patent by the China National Intellectual Property Administration (CNIPA) for our latest innovation in lipid nanoparticle (LNP) technology! Patent number ZL202211695784.5 covers a novel class of ionizable lipids with unique chemical structures, efficient synthesis methods, and their use in LNP drug delivery systems.  These advanced lipids are designed for high encapsulation efficiency, precise particle size control, and enhanced delivery of nucleic acid therapeutics, adding a powerful new tool to YolTech’s LNP platform. This achievement highlights our ongoing commitment to pioneering breakthroughs in targeted gene therapies. #YolTech #GeneTherapy #NucleicAcidTherapeutics #Innovation

YolTech Therapeutics’ Co-founder, CDO & CTO, Dr. Emma Wang, will be speaking at the upcoming TIDES Euro 2024 event. 📅 Date: Thursday, November 14, 2024 🕙 Time: 10:15 AM - 10:45 AM CET 💡 Topic: In vivo Genome Editing: Translating Science from Bench to Bedside Dr. Wang will present interim clinical data from our YOLT-201 (ATTR-CM) and YOLT-101 (HeFH and ASCVD) trials, highlighting observed safety, pharmacodynamic responses, and efficacy data. The session will also cover advances in enzyme discovery and our proprietary LNP-mediated delivery systems targeting both hepatic and extrahepatic tissues. Don’t miss this opportunity to learn more about our groundbreaking work in gene editing therapies. 👉 Discover more about TIDES Euro 2024 here：https://lnkd.in/eMTQdTX7 #TIDES2024 #GeneTherapy #InVivoGenomeEditing #LNP #mRNA #OligonucleotideTherapeutics #DrugDelivery #Biotech

The Nobel Prize was awarded for the "regulatory wonders" of microRNA this October. Following the 2023 Nobel Prize being awarded to mRNA vaccine technology, this marks the second consecutive year the Nobel Prize has recognized RNA-related research. The widespread adoption of mRNA technology could significantly accelerate the development and production of new medicines, speeding up their approval process, which is undoubtedly a major benefit for the global healthcare system. 📖 Read more about the impact of mRNA classification and regulatory updates: https://lnkd.in/eRtb5eM6 #RNA #mRNA #GeneTherapy #Science #Healthcare

Great insights into the evolution of lipid nanoparticles (LNPs) for enhanced drug delivery! As pioneers in gene editing therapies, we recognize the significance of innovations in endosomal escape for efficient nucleic acid delivery. Thank you to the authors for this detailed review on the advancements in LNPs! Read more here: https://lnkd.in/es-s9kGe #LipidNanoparticles #Nanomedicine #EndosomalEscape #GeneTherapy #LNP #DrugDelivery

Thanks CRISPR Medicine News for highlighting our latest advancements! 🙏 We're thrilled to see the preclinical success of our non-viral CRISPR-Cas9 therapy for Primary Hyperoxaluria Type 1 (PH1) recognized. Our team is advancing this approach into the clinic with YOLT-203, and we're excited to share more updates soon. Read more in today's feature 👇🏻 #GeneTherapy #CRISPR #PH1 #YolTech #LNP #InVivo

YolTech Therapeutics, in collaboration with the Shanghai Frontiers Science Center, has published groundbreaking preclinical data on a non-viral gene-editing therapy for Primary Hyperoxaluria Type 1 (PH1). Our study, published in Molecular Therapy, shows how LNP-CRISPR/Cas9 delivered precise and lasting editing in a PH1 mouse model, significantly reducing oxalate levels without notable side effects. The approach demonstrated promise both in PH1 and humanized mouse models. We are excited to bring these advances to the clinic as we continue to develop YOLT-203. Stay tuned for more updates! #GeneEditing #PH1 #CRISPR #LNPtherapy #YolTech #GeneTherapy Read more in our latest publication: https://lnkd.in/g75gmQWX