iBB-Institute for Bioengineering and Biosciences’ Post

This year’s ESACT was a great snapshot of a rapidly evolving cell and gene therapy (CGT) industry. Our experts Andrea Vervoort, Keara Sutherland, and Oliver Varette highlight 3 standout conference insights: ✔ The need for viral vectors is expected to continue to grow. This necessitates efficient and economical manufacturing methods to meet rising demand while also being mindful of environmental footprints. ✔ Industry leveraging AI/ML to model and optimize manufacturing processes, predict optimal outcomes and improve efficiency. Marinna Madrid, presented an AI/ML model for the automated production of stem cells for personalized regenerative cell therapies. There is also an emphasis on curating datasets and minimizing data silos within organizations. ✔ An excellent talk by Cath Green highlighted the benefits of providing robust biomanufacturing capabilities to academics. This enables important research to reach clinical trials, which is crucial for scenarios such as pandemics. Integrating industry and academia is pivotal for accelerating innovative therapies. Ultimately, CGT manufacturing is undeniably complex, and industry and academia collaboration is the cornerstone for the CGT sector to take transformative strides. #biomanufacturing #ESACT2024 #innovation

In Nature Nanotechnology: Gene-editing nanoparticles delivered via transfusion for the treatment of #sicklecelldisease. Current gene therapies are complex, time-consuming, and risky, with some clinical trials revealing serious side effects. A new approach from assistant research scientist Xizhen Lian of the Johns Hopkins Institute for NanoBioTechnology uses special nanoparticles that can send gene treatment directly to various types of cells in bone marrow via transfusion. The paper is “Bone-marrow-homing lipid nanoparticles for genome editing in diseased and malignant haematopoietic stem cells.” “Our approach promises to help patients avoid invasive treatment procedures, which will significantly reduce the side effects of blood cancer because there is no random gene insertion into the patient’s genes. We are targeting a specific gene that causes the disease and that’s it." Co-authors: From Hopkins: Greg Newby From University of Texas Southwestern Medical Center: Sumanta Chatterjee, Yehui Sun, Sean Dilliard, Ph.D., Stephen Moore, Yufen Xiao, Xiaoyan Bian, Kohki Yamada, Yun-Chieh Sung, sam john, Xiaoye Liu, Caroline Smith, Lindsay Johnson, Xu Wang, Cheng Zhang, Daniel Siegwart. From St Jude's Children's Research Hospital: Rachel Levine, Kalin Mayberry, Mitchell Weiss, Jonathan Yen From Broad Institute of Harvard and MIT: David R. Liu #nanomedicine #genetherapy #CRISPRCas9 #LipidNanoparticles #DrugDelivery #PrecisionMedicine #genomeediting

Join us for an insightful journey into the world of regenerative medicine. Tailored for professionals from non-scientific backgrounds, this immersive program demystifies the advanced concepts and technologies behind regenerative medicine. From stem cells to gene editing and cell therapy, learn how cutting-edge techniques are being used to repair or replace damaged tissues and cells. Course Highlights: - Gain a clear understanding of various stem cells and their distinct features. - Explore the potential risks and challenges associated with gene therapy. - Delve into cell therapy, focusing on the role and functionality of CAR receptors. - Analyze and compare gene editing methodologies, including CRISPR and ZFN. -Investigate the commercial landscape of regenerative medicine, understanding its business implications. Register here -> https://zurl.co/Dfuw As the leading life science education company for non-scientists, Biotech Primer has educated over 150,000 professionals globally since 2001, offering unparalleled training in the science, business, and regulatory foundations necessary to excel in the biotech, pharmaceutical, diagnostics, and medical device industries. #RegenerativeMedicine #StemCells #GeneTherapy #CellTherapy #BiotechPrimer

𝐒𝐭𝐞𝐦 𝐂𝐞𝐥𝐥 𝐌𝐚𝐧𝐮𝐟𝐚𝐜𝐭𝐮𝐫𝐢𝐧𝐠 𝐌𝐚𝐫𝐤𝐞𝐭 𝐒𝐢𝐳𝐞 𝐏𝐫𝐨𝐣𝐞𝐜𝐭𝐞𝐝 𝐭𝐨 𝐫𝐞𝐚𝐜𝐡 $𝟐𝟏.𝟖 𝐛𝐢𝐥𝐥𝐢𝐨𝐧 𝐛𝐲 𝟐𝟎𝟐𝟖 𝐃𝐨𝐰𝐧𝐥𝐨𝐚𝐝 𝐭𝐡𝐞 𝐏𝐃𝐅 𝐁𝐫𝐨𝐜𝐡𝐮𝐫𝐞 𝐚𝐭 https://lnkd.in/d-KbXnRJ The global stem cell manufacturing market in terms of revenue was estimated to be worth $12.7 billion in 2023 and is poised to reach $21.8 billion by 2028, growing at a CAGR of 11.3% from 2023 to 2028. 𝐊𝐞𝐲 𝐏𝐥𝐚𝐲𝐞𝐫𝐬: Cell and Gene Therapy Catapult Cell-Easy Cell BioEngines, Inc Cell Therapy Group Cell Cure Neurosciences Ltd. Cell X Technologies Stem Cell Cluster Stem Sel Srl Stem Cell Reserve Akron Bio Nodexus Inc. Nexcelom    Axion BioSystems       #StemCellResearch, #RegenerativeMedicine, #StemCellTherapy, #Biotech, #CellTherapy, #StemCellScience, #TissueEngineering, #Bioengineering, #MedicalInnovation, #Biopharma #stemcellmanufacturingmarket #stemcellmanufacturing

🔬 Targeted Gene Delivery: Revolutionizing Neurotherapeutics 🧠✨ The field of neurotherapeutics is being transformed by targeted gene delivery approaches, offering new hope for treating neurodegenerative diseases and other neurological disorders. Key Approaches: 1. Viral Vectors: Engineered viruses like AAV and lentiviruses can cross the blood-brain barrier (BBB) to deliver therapeutic genes directly to neurons, providing long-term gene expression. 2. Nanoparticle-Based Delivery: Functionalized nanoparticles encapsulate and protect genetic material, targeting specific neurons and minimizing off-target effects for enhanced efficacy. 3. CRISPR-Cas9: This revolutionary system allows precise genome editing to correct genetic mutations, potentially treating or even curing neurodegenerative diseases. 4. Exosome-Mediated Delivery: Engineered exosomes can cross the BBB and deliver therapeutic genes to neurons, offering a natural and efficient gene delivery method. 5. Polymeric Carriers: Biodegradable polymers protect and deliver genetic material, releasing their payload in response to specific stimuli for controlled gene expression. Impact: These advanced approaches ensure precision, durability, and versatility in treating conditions like Parkinson’s, Alzheimer’s, Huntington’s disease, ALS, and epilepsy. They are actively being tested in clinical settings, bringing us closer to effective, long-term treatments. As we push these innovations forward, collaboration between researchers, clinicians, and industry partners will be key to transforming neurotherapeutics and improving patient outcomes. Here’s to the future of targeted gene delivery in neurotherapeutics! #Neurotherapeutics #GeneTherapy #CRISPR #Nanotechnology #Exosomes #BiomedicalEngineering #HealthcareInnovation #Pharmacology

Regeneron is making significant advancements in gene therapy for genetic hearing loss, providing new options that extend beyond the capabilities of traditional cochlear implants. 🦻 The company's recent work with DB-OTO, a targeted AAV gene therapy aimed at correcting mutations in the otoferlin gene, has demonstrated substantial potential. Early results from the CHORD trial are promising, with the initial patient responding to auditory stimuli within a few weeks of treatment. By six months, this individual was able to perceive sounds without needing a cochlear implant - an encouraging sign for natural hearing restoration. These developments not only mark a leap forward in treating hearing loss but also underscore the power of diligent research and collaboration within the biotech sector. As Regeneron presents their case studies at the #ASGCT meeting, it serves as a testament to our collective endeavor to advance and distribute life-altering therapies.👏 #GeneTherapy #Innovation #Biotech #Science #Regeneron

Latest DON Therapeutics news The future of healthcare is being transformed by groundbreaking advancements in gene therapy, cell therapy, and immunotherapy. These innovative treatments are pushing the boundaries of what's possible in medicine. By harnessing the power of genetic material, living cells, and the body's immune system, we can now target the root causes of diseases with unprecedented precision. Gene therapy offers the potential to repair or replace faulty genes, addressing genetic disorders at their core. Cell therapy utilizes living cells to regenerate and repair damaged tissues and organs, opening new frontiers in treating a wide range of conditions. Immunotherapy leverages the immune system to fight cancer and other diseases, creating highly targeted treatments with fewer side effects. Together, these therapies represent a new era in personalized medicine, offering hope and healing to millions worldwide. The convergence of these technologies is set to change the landscape of healthcare, making once-unthinkable treatments a reality. More about DON Therapeutics: https://lnkd.in/eKs-9g9e We are always looking for new healthcare opportunities. Submit your opportunity: https://lnkd.in/dJfhwVvv #biotech #seriesa #seriesb #healthcare #genetherapy #celltherapy #funding

Latest DON Therapeutics news You couldn't have missed how fast-growing the field of Medical biotechnology is right now. It successfully combines the principles of biology and technology to develop innovative solutions for the diagnosis, treatment and prevention of disease. Medical biotechnology includes a wide range of applications, from genetic engineering and tissue engineering to drug discovery and bioinformatics. One of the most perspective areas of medical biotechnology is regenerative medicine, aimed at repairing or replacing damaged tissues and organs. Researchers are exploring the use of stem cells, biomaterials and 3D bioprinting to create functional tissues and organs for transplantation, potentially alleviating the shortage of donor organs and improving patient outcomes. Another important area is gene therapy, which involves introducing genetic material into cells to treat or prevent genetic diseases. Gene therapy has shown promising results in the treatment of diseases such as hereditary blindness, haemophilia and cancer. We are always seeking new healthcare opportunities so if you are a decision-maker maker please submit your opportunity via this link: https://lnkd.in/ep8Bqsb8 Join the biotech revolution and participate in shaping a better tomorrow with us. DON Therapeutics' official website: www.don-therapeutics.com #BiotechFunding #BiotechInvestment #VentureCapital #StartupCapital #SeedFunding

🚀 Revolutionize your transfection with Lonza’s 4D Nucleofector® Technology! 🧬 🔎Are you looking for a highly efficient and versatile solution for transfecting hard-to-transfect cells? Lonza’s 4D Nucleofector® Technology offers superior performance over traditional electroporation and other non-viral methods. 💡Key benefits: ✅Superior performance: Achieve up to 90% efficiency for plasmid DNA and 99% for oligonucleotides, like siRNA. ✅Versatile Substrate Range: Transfect a wide range of substrates including DNA, mRNA, RNPs, miRNA, siRNA, peptides or proteins. ✅Maximum Flexibility: Effective for primary cells, stem cells, neurons, cell lines as well as cells in adherence with 750+ cell-type specific protocols. ✅Cell Viability: Ensure excellent cell viability. ✅Unmatched Scalability: Various Units designed for low to high-throughput transfections. 💪Backed by sterile Nucleocuvette® vessels, 4D Nucleofector® Technology empowers groundbreaking research in Gene Therapy, CAR-T generation, genome editing via CRISPR, iPSCs transfection, among many others. 🚀 🔗Follow the link to learn more: https://rb.gy/zz4jbh #Lonza #Transfection #4DNucleofector #Nucleofection #Bioanalytica #BioSciences #CRISPR #GeneTherapy #CART #iPSCs

Digital Histology for Gene Expression Analysis Research by Pang et al. (2021) explores the potential of using digital histology images, routinely obtained in clinical settings, to predict gene expression data. Traditionally, spatial transcriptomics (ST) has been the gold standard for this type of analysis, but its cost and complexity can be limiting. The study introduces HisToGene, a novel deep learning framework leveraging Vision Transformers to extract meaningful features from H&E-stained histology images. HisToGene demonstrates impressive accuracy in predicting gene expression, even surpassing the resolution of ST in some cases. This opens doors for researchers to gain valuable insights into gene expression patterns within tissues using existing histological data. The ability to glean gene expression data from digital histology holds immense potential. It could make spatial gene expression analysis more accessible and facilitate the identification of clinically relevant biomarkers directly from routine pathology slides. While further validation is necessary, this research paves the way for potentially predicting clinically meaningful gene expression data from digital histology, a prospect that could significantly impact personalized medicine approaches. doi: https://lnkd.in/egjCRNKg #digitalpathology #genomics #precisionmedicine #oncology #artificialintelligence