Astellas Pharma’s Post

Editas Medicine announces a major strategic pivot, shifting focus entirely to in vivo gene editing after discontinuing its lead CRISPR therapy for sickle cell disease. With layoffs and renewed efforts in accelerated breakthroughs, the biotech aims to redefine its future. Learn more about the transition: https://bit.ly/3BtHQlZ

We are watching this collaboration between Somite Therapeutics and OmniaBio with great interest to see its impact on the cell and gene therapy industry. "I am excited about the long-term potential of this collaboration, which extends beyond this initial program," said Prof. Peter Zandstra, a Scientific Advisory Board member of Somite and Chief Scientific Officer of CCRM. "Somite's innovative use of AI to drive the discovery and optimization of cell therapy manufacturing presents our field with exciting opportunities to enhance the effectiveness and cost-efficiency of cell therapy products. This collaboration exemplifies how combining cutting-edge technology with deep scientific expertise can accelerate the development of transformative therapies." Read the announcement here: https://lnkd.in/gnxH9rGf #AI, #artificialintelligence, #manufacturing, #celltherapy

Biocom California recently invited Sapient to contribute an article for its Lifelines Magazine edition focused on innovations in #genetherapy and #celltherapy. https://lnkd.in/gC9YfQnM While cell- and gene-based treatments are advancing rapidly, spurred by a landmark year of FDA approvals, their development is not without challenges. We discuss how #multiomics for gene and cell therapy can deliver the novel insights needed to realize the incredible therapeutic potential represented in these pipelines today: https://lnkd.in/gC9YfQnM

We understand that the journey of working with induced pluripotent stem cells (iPSCs) for clinical applications is fraught with challenges. From maintaining robust cell cultures to ensuring reliable performance under stressful conditions, the path is not easy. Gibco™ CTS™ StemFlex™ Medium, launched on Friday, Nov 15th, is designed to address these challenges. Gibco CTS StemFlex Medium supports the robust expansion of feeder-free pluripotent stem cells (PSCs) and is optimized for the most demanding clinical production applications, including gene editing, clonal expansion, and single-cell passaging. Key benefits of Gibco CTS StemFlex Medium include: -Robust PSC adherent cell culture for cell therapy -Reliable performance in stressful applications -High clonal expansion efficiency -Consistent expansion of high-quality PSCs -Smooth transition from StemFlex Medium (RUO) to CTS StemFlex Medium for clinical production Elevate your cell therapy research and clinical production with the Gibco CTS StemFlex Medium. Visit our product page to learn more about how this innovative medium can make a difference in your work. Learn more: https://lnkd.in/eQiNiNHc

This two-part BPI article is a great and concise summary of the analytical considerations that cell therapy manufacturers must take during the development of gene-modified HSPC therapies. #celltherapy #genetherapy Part 1 covers in process and drug product controls https://lnkd.in/gch7xhbd Part 2 covers starting materials and gene modification reagents https://lnkd.in/gWSxSAHb

The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research within the fields of gene and cell therapy. One particular presentation from biotechnology and gene/cell therapy company Sangamo Therapeutics (“Sangamo”) centered around promising data from a Phase 1/2 clinical study evaluating ST-920 for patients with Fabry disease. In addition to sustaining α-Gal A activity, ST-920 was safe, well-tolerated, and had many other benefits for patients. #genetherapy

Excited to share a new publication in the Journal of Molecular Therapy which highlights the eShunt®️ Platform as a CNS gene therapy delivery method. Thanks to the teams at UMass Chan Medical School and Tufts Medical Center for your work on this study. "We're very excited about these promising preclinical results, which suggest that the eShunt System delivery approach could provide biopharmaceutical and biotechnology organizations with a minimally invasive method for delivering gene therapies directly to the cortex and brain." #bbb #CNS #drugdelivery #neurovascular #endovascular https://lnkd.in/eeKCGPm7

Next week DIVINCELL and Aanastra Inc will be at the American Society of Gene & Cell Therapy Gene and Cell Therapies (@ASGCT), 27th Annual Meeting, taking place May 7-11, 2024, in Baltimore, Maryland. https://lnkd.in/dHgkVjV We will be presenting oral and posters highlighting preclinical data supporting our novel RNA-based therapeutic strategy to target aberrant genes and oncogenes : ▶Oral presentation" KRAS Mutant Gene Editing Abolishes Tumor Growth In Vivo and Overcomes Acquired Resistance to KRASG12C and KRASG12D Small Molecule Inhibitors" Session: Targeted Gene and Cell Therapy II, Abstract: 415, May 11th, 10:15 AM - 12:00 PM ▶ “Peptide Based Nanoparticle-Platform for Extrahepatic Delivery of Functional mRNAs" Abstract: 761, May 8th ▶ " Peptide-Based Nanoparticles Mediated mRNA Rescue of Factor VIII; a New Approach to Treat Hemophilia A" Abstract : 1093, May 9th ▶ " In Vivo CRISPR Mediated Editing of PCSK9 Using Peptide-Based Nanoparticles Durably Lowers Cholesterol in Mice" Abstract : 1094, May 9th #p53 #KRAS #PCSK9 #CRISPR #nanotechnology #drugdelivery #mRNA #ASGCT #haemophilia #geneediting

🎉 Gene and cell therapies have opened a new therapeutic era!!!   🏆 Yesterday was the day: a joint strategy paper, written by over 120 stakeholders from science, industry, politics, authorities, foundations and patient organizations on the “National Strategy for Gene and Cell Therapy Research and Development” was ceremoniously presented to the Federal Minister of Education and Research, Bettina Stark-Watzinger, at the Futurium in Berlin I am glad to be one of these stakeholders and to have been given the opportunity to contribute  developing the strategy. This paper identifies the challenges in the various fields of action and proposes the most suitable measures for implementation. 🔬 Together with me, eight stakeholders (Axel Schambach, Hildegard Büning, Ulrich Kalinke, Tobias Cantz, Robert Zweigerdt, Ulrich Martin, Nataliya Di Donato) from Medizinische Hochschule Hannover were selected as experts to contribute to this strategy paper in various working groups. This emphasizes the importance and role of the Medizinische Hochschule Hannover and its employees in research, development and translation in the field of gene and cell therapy. 🔬 It is now important to continue and to ensure that new findings from research reach patients quickly, that the production of genetically modified and unmodified cellular drugs is established sustainably in Germany and that clinical trials lead to drug authorization. We must now take the momentum to not lose the international connection and innovation. Our mission is to provide patients in-time with high-quality, highly specific and personalized medicines to safe lifes and improve its quality. 💐 Importantly, my participation in this work is also a tribute to all members of our alloCELL lab, the Molecular Immunotherapy Group and the entire ITT of Medizinische Hochschule Hannover.   Rainer Blasczyk Johanna Barnstorf-Brandes Agnes Bonifacius Sabine Tischer-Zimmermann #celltherapy #immunotherapy #genetherapy #transplantation #CAR_T_cells #cellengineering #GCTGermany #NationaleStrategieGCT #Zelltherapie #Gentherapie

[PRESS RELEASE] Genethon presents positive results from first phase of GNT0004, the drug candidate for patients who suffer from Duchenne Muscular Dystrophy, with ability to walk during American Society of Gene & Cell Therapy Breakthroughs in Muscular Dystrophy Congress, in Chicago. This drug-candidate has the potential to overturn the disease: good tolerability, efficacy data, stabilization of motor functions - the results of the phase 1/2 dose escalation are remarkable.  Genethon expects to launch the pivotal phase of the trial, with the inclusion of the first patients in 2025 with the aim of treating over 60 patients in Europe and the United States. Braun Serge Frederic Revah https://urls.fr/yVRgfJ #duchenne #Genetherapy #clinicaltrials #DMD #raredisease #ASGCTBreakthroughs24 #musculardystrophy