Synthego Corporation’s Post

"The European Commission has approved CRISPR Therapeutics and Vertex Pharma’s exagamglogene autotemcel (exa-cel; Casgevy) CRISPR-edited gene therapy for the treatment of patients who are 12 years of age and older with severe sickle cell disease (SCD) characterized by recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT), for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.1" #DahliaConsulting #BiopharmaceuticalIndustry #CommercialLifeSciences #ManagementConsultant #LifeSciencesBusinessStrategy #GlobalPharma #GlobalBioTech #SupplychainOperations #CommercialOperations #CGTcommercialization #Manufacturing #QualityAssurance #OperationalExcellence #FinancialAnalysis #OpModelDesign #LifeScience #BioTechnology #Bioprocessing #Biosimilars #BioPharmaIndustry #MedicalResearch #CDMO #GMP #GMPbatch #GMPmanufacturing #GenomeEditing #GeneTherapy #CellTherapy #Car_T #mRNA #cGMP #ClinicalGradeManufacturing #ClinicalGradeProduction #BiologicsLicenseApplication #BLA #Pharmaceuticals #RegenerativeMedicine #CGTLive #EuropeanCommission #CRISPRTherapeutics #VertexPharma #CASGEVY

Impactful work from Daniel Siegwart, ReCode Therapeutics, and the rest of the team The University of Texas Southwestern Medical School recently published in Nature Communications! Authors highlight the success of their lung targeting SORT LNP platform to deliver Cas9 mRNA, sgRNA, and donor ssDNA templates and ultimately correct genes associated with Cystic Fibrosis in both ex vivo and in vivo models. This works demonstrates a promising opportunity for mRNA LNPs as a CF treatment via CRISPR/Cas gene correction. https://lnkd.in/ds_d_uP4

Gene editing targeting lungs shows promise in preclinical study A preclinical study by ReCode Therapeutics shows promise for a lung-targeted gene-editing therapy designed to correct the CFTR gene mutation in cystic fibrosis (CF) patients. Using Selective Organ Targeting (SORT) lipid nanoparticles (LNPs), the therapy effectively targeted lung stem cells in mice through intravenous infusion. This method demonstrated significant gene correction, with potential for long-lasting treatments for CF and other genetic disorders. ReCode's lead candidate, RCT2100, which delivers healthy CFTR mRNA, is currently in Phase 1 trials. Further safety studies are needed before moving to clinical applications. To read more: https://lnkd.in/gDXH6DjT #CFA #CFAustralia #CysticFibrosisAustralia #News #ReCodeTherapeutics

Defence Therapeutics is honored to announce that our CEO, Sébastien Plouffe, will be attending the prestigious ISCT, International Society for Cell & Gene Therapy 2024 Conference in Vancouver, taking place from May 29 to June 1. This event marks the 30th annual meeting of ISCT and stands as the largest global gathering for cell and gene therapy professionals, drawing over 2,500 experts from academia, regulatory bodies, and the industry. The ISCT conference is a cornerstone for advancements in cell and gene therapies, offering unparalleled opportunities for collaboration and knowledge exchange. The program includes over 50 roundtable sessions covering critical topics such as induced pluripotent stem cells (iPSCs), exosomes, chimeric antigen receptor T-cells (CAR-Ts), and regulatory harmonization. Additionally, the Translational Pathway Program will provide deep insights into the latest scientific breakthroughs and innovative therapies. We are also proud to announce that Dr. Moutih Rafei مطيع الرافعي, Defence's Chief Scientific Officer, will be presenting our ARM-X vaccine advancement on May 31st at 13:00 at the Global Showcase Exhibit Hall session. The session, titled "ARM-X: Putting Pedal to the Metal for the Design of Cell-Based Anti-Cancer Vaccines," will highlight the cutting-edge developments and potential of this innovative approach in cancer therapy. Mr. Plouffe and Dr. Rafei’s participation underscores our commitment to driving forward the frontiers of biomedical innovation. We look forward to engaging with global leaders in cell and gene therapy, fostering new collaborations, and advancing our mission to translate scientific research into effective treatments that improve patient outcomes. 👉 Stay informed with updates from the conference by following our page. #ISCT2024 #CellTherapy #GeneTherapy #Biotechnology #DefenceTherapeutics #Vancouver

Exciting news! Our latest study, published in the Journal of Extracellular Vesicles, explores the potential of Extracellular Vesicles (EVs) as therapeutic delivery vehicle.   These small but mighty nanoparticles, known for facilitating cell-to-cell communication by carrying essential molecules, show great promise as a method for delivering advanced therapies like CRISPR/Cas9.   What makes this study stand out? -      EVs stand out due to their biocompatibility, low immunogenicity, and natural targeting abilities, making them an ideal drug delivery system. -      Our study investigates the use of EVs to administer CRISPR/Cas9, targeting the Pcsk9 gene. This could offer a new approach to lower cholesterol levels and, in turn, lessen the risk of coronary artery disease. -      We have successfully demonstrated EV-mediated delivery of CRISPR/Cas9 RNP targeting of the Pcsk9 gene ex vivo in primary mouse hepatocytes.   Special thanks to the dedicated efforts of Thomas Kluiver, Olivier Gerrit de Jong, Saskia de Jager, Raymond Schiffelers, Pieter Vader, Weng Chuan Peng, Lei Zhiyong, and Joost Sluijter. UMC Utrecht Regenerative Medicine Utrecht   Read the full article here ⬇

Ten years ago, I was involved in this collaborative article for Cytotherapy, which reflected on twenty years of ISCT, International Society for Cell & Gene Therapy. Ten years on, and as we approach ISCT, International Society for Cell & Gene Therapy’s annual conference in Vancouver, I’m looking back on some of the predictions we made and feel a huge sense of progress and achievement. Of course, there is still a lot more to be done to improve manufacturing efficiency, costs, and patient access. Nonetheless, we can be proud, as an industry, of the innovations and advances we’ve seen. Here are a few highlights of this paper's ‘Looking to the Future’ section in 2014. -Novel concepts conceived: New technologies in leading academic labs will be developed in partnership with risk-tolerant commercial biotechnology groups - Clinical trials: Following clinical proof of concept by these partnerships, large pharma support for late-stage clinical trials and commercialization will realize the wider availability of effective gene-modified cell therapies - The field of induced pluripotent stem cell (iPSC) biology: Advance beyond effective application in traditional drug screening into an effective therapeutic modality in its own right - The patient access: To usher in this new era of treatments, it will also be critical for the patient voice to be heard. What do you predict for the next ten, twenty years? "Twenty years of the International Society for Cellular Therapies: the past, present and future of cellular therapy clinical development "- https://rb.gy/tlcwsz

🔬 Exciting news from Interius BioTherapeutics! 🔬 Interius BioTherapeutics has reached a significant milestone by dosing the first patient in its INVISE Phase 1 clinical trial, a groundbreaking step for in vivo chimeric antigen receptor (CAR) gene therapy. Their innovative therapy, INT2104, is designed to generate CAR-T and CAR-NK cells directly within the patient to target CD20-positive B cells, offering a promising new treatment option for B-cell malignancies without the need for preconditioning or extended manufacturing delays. The trial received approval from Australia’s Human Research Ethics Committee in July 2024, paving the way for this first-in-human study. Philip R. Johnson, MD, , CEO of Interius, emphasized that this marks the first clinical use of a durable in vivo CAR therapy, with preclinical studies demonstrating effective B cell depletion without chemotherapy, cytokine release syndrome (CRS), or neurotoxicity. The company plans to share interim safety and proof-of-concept data in the first half of 2025. Dr. Michael Dickinson, the principal investigator for the INVISE trial, underscores the transformative potential of this approach, which promises faster, single-dose solutions that ease the burden on both patients and healthcare systems. This first-in-human trial represents a significant leap forward in making gene therapies more accessible and off-the-shelf. Stay tuned for more updates on INT2104's progress! Press release: https://lnkd.in/duJA-fja #Biotech #GeneTherapy #CancerResearch #CARtherapy

ASGCT24: Prime Medicine to Apply Gene Editing to Wide-Ranging Indications ! 🧬First discussed in a Nature paper in 2019, the technology now includes dual-flap prime editors for larger insertions and deletions as well as Prime Assisted Site Specific Integrase Gene Editing (PASSIGE), a system that integrates large genetic payloads by leveraging a recombinase target sequence. With these platforms, the company is working on a pipeline of investigational therapeutic programs in hematology, immunology, liver, lung, ocular and neuromuscular diseases. 🧫The Cambridge, Mass.–based biotech on April 29 announced that the FDA has cleared a study of a drug candidate based on its prime editing platform, marking the first time the agency has greenlit the use of the genetic technology in humans. Prime Medicine will report initial data next year from the Phase I/II trial of PM359, its ex vivo candidate designed to correct a prevalent disease-causing mutation of the rare inherited hematologic disorder chronic granulomatous disease.

🔬 DNA methylation plays a crucial role in regulating gene expression and the development of major diseases including cancer. Our recent in Nature Communications article highlights how our novel CE IVD certified EpiDirect® Assay technology enables direct PCR quantification of DNA methylation using small amounts of untreated DNA. These unique features of EpiDirect® hold the potential to in vitro diagnose and treat brain cancer patients with alkylating agents prior to surgery and thereby significantly improve patient outcome. Read more about the EpiDirect® technology in the full article. https://hubs.la/Q02F-_MS0 #Epigenetics #Glioma #CancerDiagnostics #Biotech #Innovation

"With three FDA approvals in the past 10 months, there is a lot of momentum in the Duchenne muscular dystrophy space. Here are five companies looking to keep it going." 1. Pfizer - gene therapy: A mini-dystrophin gene therapy, fordadistrogene movaparvovec is being studied in this trial for use in ambulatory DMD patients. 2. Capricor Therapeutics - cell therapy: CAP-1002 is composed of cardiosphere-derived cells (CDCs) derived from healthy human hearts. The CDCs secrete exosomes containing bioactive cargo, including microRNAs, that alter gene expression in macrophages and other target cells, dialing down generalized inflammation and stimulating tissue regeneration. 3. Avidity Biosciences - exon-skipping biologic: AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers to the skeletal muscle and heart tissue to specifically skip exon 44 of dystrophin mRNA in order to enable dystrophin production. 4.  Dyne Therapeutics - exon-skipping biologic: DYNE-251 comprises a phosphorodiamidate morpholino oligomer conjugated to a fragment antibody that binds to the transferrin receptor 1, which is highly expressed in muscle. 5. Edgewise Therapeutics - small molecule: EDG-5506 is designed to reduce injurious mechanical stress caused by the absence of functional dystrophin in order to enable a normal, healthy range of muscle contraction.