British Society for Gene and Cell Therapy (BSGCT)’s Post

Excited to announce the launch of Diagonal Therapeutics. We have rapidly discovered agonist antibody candidates against four complex targets, providing new paths to targeting the root cause of severe diseases. Our platform is agnostic and can work across any therapeutic areas. Our initial programs target two vasculopathies with high unmet need: Hereditary hemorrhagic telangiectasia (HHT): HHT is an orphan disease that affects more than 150,000 patients in the US and EU and for which there are no approved therapies. Pulmonary arterial hypertension (PAH): PAH is an orphan disease that results in high blood pressure that affects the arteries in the lungs and the right side of the heart. Our approach also capitalizes on the excellent properties of antibodies: superior potency and selectivity, tunable and sustained pharmacology, excellent developability, and low immunogenicity to develop optimal drug candidates for patients. #raredisase #orphandisease #antibodies #innovation

I am excited to announce the launch of Diagonal Therapeutics. We are advancing a ground-breaking approach to agonist antibody therapeutics. Our DIAGONAL platform has enabled the discovery of agonist antibodies to four different heteromeric receptor complexes. We have solved the longstanding challenge that has limited agonist antibody drug discovery by skillfully blending computational and experimental techniques - allowing us to move efficiently (8 months or less to agonist antibody discovery) - a timeframe previously unimaginable using historical methods. The rapid advancement of our lead programs for hereditary hemorrhagic telangiectasia (HHT) and pulmonary arterial hypertension (PAH) demonstrate the power of the DIAGONAL platform and breadth of opportunities yet to be solved using our approach. #innovation #antibodies #biotechnology #partnerships

🚀 Exciting Partnership Unveiled! Remix Therapeutics joins forces with Roche for pioneering discoveries in small molecule therapeutics modulating RNA processing using the REMaster™ platform. 🧬💊 🌐 Remix, a clinical-stage biotech, will receive $30 million upfront, with potential milestone payments totaling up to $1 billion, plus royalties. In return, Roche gains exclusive rights to specific targets, marking a strategic collaboration to advance RNA-level interventions in disease treatment. 💡👥 👨🔬 Peter Smith, CEO of Remix, expresses enthusiasm about leveraging REMaster's capabilities, stating, "Our collaboration with Roche provides an opportunity to further translate our cutting-edge science into new medicines." 🌱 🌐 James Sabry, Global Head of Roche Pharma Partnering, highlights Remix's scientific prowess, emphasizing the alignment with Roche's strategy to innovate in areas with high unmet medical needs. 🤝🔍 🔬 Remix Therapeutics, with its REMaster platform, aims to reprogram RNA processing to alter gene expression fundamentally—a potential game-changer in addressing disease drivers at their root. 🌍🔬 💬 Stay tuned for groundbreaking developments as Remix and Roche redefine the landscape of small molecule therapeutics! 💪🌐 #RemixTherapeutics #RocheCollaboration #RNAProcessing #BiotechInnovation #HealthcarePartnership #DrugDiscovery #MedicalAdvancements #InnovationInMedicine #Biopharma #REMmasterPlatform #LinkedInNews 🌐👩⚕️

Join our workshop on #rna #manufacturing. Our experts are there to guide you through the key decisions to be taken when embarking on an RNA based journey.

🚀 Nvelop Therapeutics launched a new platform for in-vivo delivery🧬 In a significant stride towards advancing genetic medicine, Nvelop Therapeutics has been launched by a coalition of gene editing trailblazers, including David Liu and Keith Joung, alongside industry stalwarts Jeff Walsh (CEO) and Melissa Bonner (CSO). This innovative biotech firm is set to redefine the landscape of genetic therapies with its novel, non-viral delivery technologies. With a focus on creating more precise, efficient, and safer gene therapies, Nvelop Therapeutics is leveraging in vivo validated platforms to tackle a broad spectrum of diseases. These platforms aim to address the limitations of current delivery methods, promising a new era of gene therapy. Backed by an impressive roster of life science investors, such as Newpath Partners, Atlas Venture, F-Prime Capital, 5AM Ventures, GV, and ARCH Venture Partners, Nvelop Therapeutics is well-positioned to make significant contributions to the field of genetic medicine. As Nvelop Therapeutics embarks on its mission to develop groundbreaking therapies, the biotech community watches eagerly. This venture not only marks a pivotal moment in the pursuit of curative treatments but also heralds a new chapter in medical science. #NvelopTherapeutics #GeneTherapy #BiotechInnovation #FutureOfMedicine

We recently shared our work on using JESS automated western blotting to track phospho-proteins in a signaling pathway. By leveraging biomarkers, we developed a screening cascade that identified drug compounds with high specificity and potency, crucial for advancing therapies with minimal side effects. This approach ensured the targeted inhibition of a signaling pathway branch, offering a more refined and effective therapeutic option. Our findings pave the way for successful transitions from in vitro to in vivo studies, reinforcing the drug development pipeline. Read the full case study here: https://lnkd.in/ew3HpCuu #Biomarkers #DrugDiscovery #westernblotting

𝗠𝗼𝗹𝗲𝗰𝘂𝗹𝗮𝗿 𝗴𝗹𝘂𝗲𝘀 - I always found their concept fascinating - you can basically change molecular pathways by forcing different proteins to interact! The latest deal between Takeda and Degron Therapeutics specifically revolves around degraders, which 'glue' the target protein to an E3 ubiquitin ligase complex, leading to ubiquitination and subsequent degradation of the target protein by the proteasome - such an elegant way to get rid of proteins you don't want, isn't it? 💁♀️ Molecular Glue Degraders (MGDs) have been discovered serendipitously in the past, but Degron Therapeutics' approach is completely changing the game by using AI/ML and developing multiple complementary screening and validation platforms. Definitely one to watch! #MGD #molecularglues #biotech #pharmadeal https://lnkd.in/eKCr82N4

Today at #ASGCT we will be sharing additional data from our team at Vesigen Therapeutics, Inc., including #targeted #delivery of #genomeeditors to a variety of cells, which we hope will enable new therapeutic strategies for diseases with unmet need. Please come by our posters, listed below, if interested in learning more: ARMMs as Non-Viral Vehicles for the Delivery of Therapeutic Payloads to Liver Sinusoidal Endothelial Cells Abstract Number: 1238 Fractionation of distinct extracellular vesicle populations by charge and density to evaluate the heterogeneity and purity of ARMMs Abstract Number: 1246 ARMMs as a Versatile and Modular Non-Viral Platform for the Functional Delivery of Genome Editors Abstract Number: 1247 Engineering ARMMs with Modular T Cell-Specific Engagers for Non-Viral Delivery of Therapeutic Payloads Abstract Number: 1248 Engineering Directed Tropism in the Non-Viral ARMMs Delivery Platform Abstract Number: 1249 #nonviral #invivo #drugdelivery #genomeediting

The life sciences industry is witnessing a significant shift in 2024, marked by strategic advancements and a cautiously optimistic outlook. Key movements, like Roche's notable acquisition of Carmot Therapeutics, Inc., signal a focused approach towards areas with high potential, such as obesity and neurology. This trend represents a departure from the broader investment strategies of the past and underscores a more targeted direction in research and development. In this evolving landscape, the importance of aligning specialized talent with emerging fields is more pronounced than ever. Areas like gene editing and neurotechnology are not just future promises; they are rapidly growing sectors that demand a specific skill set. Despite the challenges in funding, particularly for public biotech firms, opportunities for strategic growth and innovation remain. For our company, resilience and adaptability are key virtues as we navigate these changes. Our focus is on staying agile and responsive to the industry’s needs, ensuring that we are well-positioned to support and drive progress in this dynamic phase. https://lnkd.in/ee4PUY92 #lifesciences #AspireLifeSciences #biotech #clinicaltrials #2024trends #2024

The medical breakthroughs of tomorrow are happening today, which is why Clarivate recently published the annual “Drugs to Watch” report so that you can stay on top of the newest breakthroughs, technologies, and treatments in the pharmaceutical industry. In 2024, get ready for #geneediting, #machinelearning, and #AI! Read more here: