Resolution Therapeutics’ Post

While the cell therapy field is searching for ways to solve exhaustion, self-tolerance, and cost-effectiveness hurdles, our thymus-empowered allogeneic approach could be a game-changer. How? Our therapeutic T cell progenitors can be generated in just a week from healthy compatible donors' mobilized peripheral blood stem cells. They benefit from patients' thymic education, becoming fully functional T cells expected to persist over the years. Our Chief Scientific Officer, Olivier NEGRE, Ph.D., will explain this disruptive approach at the first Innovations for Cell & Gene Therapies Congress in Paris, on May 22d. Register now to attend the event at Institut du Cerveau – Paris Brain Institute https://lnkd.in/gYV7rZxy MabDesign SFTCG-Société Française de Thérapie Cellulaire et Génique #ICGT2024 #CGT #Cell #Gene #Congress #Thymus #Leukemia #CART

I was in Barcelona last week for the 7th Annual Cell & Gene Therapy Innovation Summit, where I had the opportunity to talk about the importance of bringing the potential of cell therapy to more people living with cancer. This topic is especially critical today, as we know that only a fraction of eligible patients receive #celltherapy. To overcome these barriers, everyone in the cell therapy space must work together – and always keep our focus on supporting patients and their physicians – as we aim to change the current landscape. This includes scaling manufacturing capacity to meet patient demand; developing a global network of treatment centers to bring therapies closer to patients; working on ways we can increase patient access; and coordinating the connections between oncologists and treatment centers to support each patient’s journey. #cgti

RNA-based translation activators for targeted gene upregulation. A molecular technology called "translation-activating RNAs" (taRNAs) shows promise in treating diseases caused by haploinsufficiency, where one functional gene copy is absent. taRNAs are small molecules designed to bind to specific mRNA molecules, regulating their translation into proteins. This approach was successful in increasing mRNA levels for PTEN and ABCA7, genes associated with tumor suppression and Alzheimer's disease. In a human sample of triple-negative breast cancer, taRNAs slowed tumor growth. The technology also restored protein levels in SYNGAP1, a gene linked to epilepsy and impaired neurodevelopment. Read more: https://lnkd.in/dNqvbgcc #rna #tumor #biology #alzheimer #medicalresearch

We're thrilled to congratulate Jessica Boehler, Principal Scientist, R&D at Solid Biosciences, on the recent publication of her manuscript titled "N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy" now featured in the current issue of Skeletal Muscle.   The research introduces a non-invasive biomarker for assessing the efficacy of microdystrophin gene therapy. Biomarkers from non-invasive biofluids, like the N-terminal fragment of titin, have potential to streamline drug development, enhance confidence in efficacy and ease the burden of clinical trial participation on patients and families.    Kudos to Jessica for this significant advancement in Duchenne muscular dystrophy research! The team at Solid remains dedicated to advancing groundbreaking research that holds promise for all its patient communities.    Read the full publication: https://lnkd.in/eSrUeyiQ   #ResearchandDevelopment #DuchenneMuscularDystrophy #GeneTherapy #ClinicalResearch

Check out the 3rd session of our webinar series, “Harnessing iNKT cells for ‘off-the-shelf’ cancer therapy” presented by Dr. Lil Yang, Associate Professor at the University of California. This presentation describes a study combining hematopoietic stem cell (HSC) gene engineering and in vitro differentiation, to generate human allogeneic HSC-engineered iNKT (AlloHSC-iNKT) cells at high yield and purity. These cells closely resemble endogenous iNKT cells, effectively target tumor cells using multiple mechanisms, and exhibit high safety and low immunogenicity. Collectively, these preclinical studies demonstrate the feasibility and cancer therapy potential of AlloHSC-iNKT cell products and lay a foundation for their translational and clinical development. Watch here! https://lnkd.in/gkrz3d82 #cancerresearch #syntheticbiology #lifesciences #rnatherapeutics #geneandcellresearch

Check out the 3rd session of our webinar series, “Harnessing iNKT cells for ‘off-the-shelf’ cancer therapy” presented by Dr. Lil Yang, Associate Professor at the University of California. This presentation describes a study combining hematopoietic stem cell (HSC) gene engineering and in vitro differentiation, to generate human allogeneic HSC-engineered iNKT (AlloHSC-iNKT) cells at high yield and purity. These cells closely resemble endogenous iNKT cells, effectively target tumor cells using multiple mechanisms, and exhibit high safety and low immunogenicity. Collectively, these preclinical studies demonstrate the feasibility and cancer therapy potential of AlloHSC-iNKT cell products and lay a foundation for their translational and clinical development. Watch here! https://lnkd.in/gkrz3d82 #cancerresearch #syntheticbiology #lifesciences #rnatherapeutics #geneandcellresearch

📖 Read the article: https://lnkd.in/gByvf64T 🌟 The gene-modified cell therapy field continues to grow apace, particularly in the oncology arena, which dominates both preclinical and clinical applications. We interviewed industry thought leaders in collaboration with the team at Cell & Gene Therapy Insights to provide you with the latest trends for #gene-modified cell therapy. #Oncology #GeneTherapy #Biotechnology

Generation of induced pluripotent stem cell lines from two unrelated individuals with familial hypertrophic cardiomyopathy carrying MYBPC3 nonsense mutations https://lnkd.in/dHkHneNB This study reports the generation of iPSC lines derived from two unrelated individuals carry the c.1731G > A and c.2670G > A nonsense mutations in the MYBPC3 gene. These mutations have been previously linked to hypertrophic cardiomyopathy (HCM). These iPSCs can be differentiated into cardiac cells, thus providing valuable resources for elucidating the pathophysiological mechanisms underlying HCM.

Did you already read the interview with Inge Jedema and Jurgen Kuball❓ “Success will be putting the Netherlands firmly on the international map as a leader in cell and gene therapy.” Read the full double-interview with Jurgen Kuball, chair of the Cell and Gene Therapy Workstream management team and head of the Department of Hematology at UMC Utrecht, and Inge Jedema, co-chair and head of Translational Cellular Therapy at The Netherlands Cancer Institute. ➡ https://lnkd.in/dSyxyQ5U #preclinical #drugdevelopment #healthdata #patientengagement #ai #patientcohorts #organoids #cellgenetherapy #smallmolecules #biologics #therapeuticvaccines #regulatoryinnovation #nationaalgroeifonds #NGF #oncology #cancerresearch #cancertherapy