Miroslav Gasparek’s Post

Novartis has acquired Kate Therapeutics, a San Diego-based #genetherapy company, in a deal valued at up to $1.1 billion. Kate Therapeutics specializes in #preclinical programs targeting Duchenne muscular dystrophy (#DMD), facioscapulohumeral dystrophy (#FSHD), and myotonic dystrophy type 1 (#DM1). The company employs its proprietary DELIVER platform, which utilizes directed evolution of adeno-associated virus (#AAV) capsids to create #liver-#detargeted, muscle-tropic #vectors. Acknowledging recent challenges in muscular dystrophy treatments, such as Pfizer's investigational gene therapy for DMD failing to demonstrate efficacy in a Phase 3 trial, Novartis CEO Vas Narasimhan emphasized the company's commitment to learning from past experiences in the #musculardystrophy domain. By acquiring Kate Therapeutics, Novartis aims to leverage innovative #delivery #technologies to overcome previous obstacles in gene therapy for #neuromuscular #diseases. Muscular Dystrophy Association Muscular Dystrophy UK Muscular Dystrophy Canada Facioscapulohumeral Muscular Dystrophy Duchenne UK Duchenne Heroes #genetherapy #cellandgenetherapy #biotech #pharma #geneticmedice #biopioneers #innovation #geneediting https://lnkd.in/giRnS_6G

Huge Acquisition 🚨 Novartis Takes Bold Step in Muscular Dystrophy with $1.1B Kate Therapeutics Deal Novartis is expanding its gene therapy efforts by acquiring Kate Therapeutics, focusing on treatments for Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1). 𝗞𝗲𝘆 𝗣𝗼𝗶𝗻𝘁𝘀: ➡️ Kate Therapeutics cutting-edge platform targets muscle and cardiac tissues while avoiding the liver, solving key delivery challenges. ➡️ Novartis aims to learn from past industry setbacks to advance its neuromuscular pipeline. ➡️ This move strengthens Novartis gene therapy portfolio beyond its spinal muscular atrophy treatment, Zolgensma. What do you think about Novartis push into this challenging space? 𝗙𝘂𝗹𝗹 𝗔𝗿𝘁𝗶𝗰𝗹𝗲 𝗕𝗲𝗹𝗼𝘄: ⬇️ #biotech #genetherapy #novartis #katetherapeutics

On World Duchenne Muscular Dystrophy (DMD) Day, I want to shed light on the crucial advancements in therapeutic research highlighted in the paper published in Nature in 2023 by Roberts and his collegues comprehensive review delves into transformative approaches such as gene therapy, exon-skipping, and CRISPR-based technologies, aimed at restoring dystrophin function—the key protein absent in DMD patients. These innovations offer new hope in slowing disease progression and enhancing muscle function. Gene therapies aim to address faulty genes by either replacing or repairing them, while exon-skipping methods are designed to circumvent genetic mutations, enabling the body to produce a functional version of the dystrophin protein. Additionally, CRISPR technology offers the potential to correct these mutations directly within the DNA, providing a more permanent solution. This review presents recent developments in therapeutic approaches for Duchenne Muscular Dystrophy (DMD), focusing on innovative techniques These efforts represent a significant leap forward in the treatment of DMD, a disorder that causes progressive muscle degeneration. Continued support for research and innovation is vital in our collective mission to better manage, or potentially cure, Duchenne Muscular Dystrophy. You can access the paper here: https://lnkd.in/dZ-C8HYe Roberts, T. C., Wood, M. J., & Davies, K. E. (2023). Therapeutic approaches for Duchenne muscular dystrophy. Nature Reviews Drug Discovery, 22(11), 917-934. DOI: 10.1038/s41573-023-00775-6 #WorldDMDday #DuchenneMuscularDystrophy #DMDResearch #GeneTherapy #ExonSkipping

Novartis spends $1.1B for Kate Therapeutics purchase. Kate Tx pipeline contains preclinical AAV based gene therapies for Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1). https://lnkd.in/g3XxfPzx

As more #clinicaltrials are initiated and safety issues are addressed, #iPSC-based #genetherapy is likely to play an increasingly important role in #personalizedmedicine. In his most recent article for Cell & Gene, CSTEAM biotechnology's Jack (Jie) Huang MD, PhD discusses the current progress, applications, challenges, and future prospects of iPSC-based gene therapy, focusing on its potential in treating #geneticdiseases, #cancer, and age-related disorders. https://lnkd.in/eYziRt-H

Novartis is stepping up its game in neuroscience, leveraging its cell-and-gene therapy expertise to tackle challenging neuromuscular diseases. In a $1.1 billion deal blending upfront cash with milestone-based payments, the Swiss pharma giant has acquired San Diego-based Kate Therapeutics, a biotech specializing in innovative approaches to Duchenne muscular dystrophy and other muscular disorders. By focusing on lessons learned from early industry pioneers, Novartis aims to deepen its foothold in gene therapy while mitigating risk through this strategic investment structure.

🚀 Novartis strengthens its gene therapy portfolio with the acquisition of Kate Therapeutics, aiming to revolutionize treatments for neuromuscular disorders like DMD and DM1! 💡 #GeneTherapy #Healthcare #Innovation #Novartis #Pharma #MarketAccessToday #MarketAccess

[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

#BreakingNews --> Addressing Unmet Needs in Neuromuscular Diseases: Novartis Acquires Kate Therapeutics Let's look into this recent development in more detail: 💡 Novartis has acquired Kate Therapeutics, a San Diego-based biotech, to strengthen its strategic focus on #genetherapy and neuroscience innovation. 🔬 Kate's preclinical therapeutic candidates target inherited #neuromuscular diseases such as #Duchenne muscular dystrophy (#DMD), #facioscapulohumeral dystrophy (#FSHD), and myotonic dystrophy type 1 (DM1). 🧬 The acquisition includes Kate's groundbreaking DELIVER platform, which uses #machinelearning and transcript-based in vivo selection to evolve potent, muscle-tropic #AAV capsids while minimizing liver toxicity. 💰 Total transaction value is up to $1.1 billion, comprising an upfront payment and potential milestone payments. 🏥 Neuromuscular diseases, often genetic in nature, disrupt muscle function and nerve signaling, posing significant challenges to patients and families. 🚀 With this acquisition, Novartis enhances its portfolio, targeting improved efficacy, safety, and precision in gene therapies for challenging neuromuscular diseases. Let's keep the discussion going! What are your views on: 🤔 How might machine learning-driven platforms like DELIVER reshape the landscape of gene therapy? 🧬 What role do you see for advanced capsid engineering in overcoming current gene therapy limitations? Please share your thoughts and like / share this post to see more like it! Reach out to #LucidQuest at [email protected] to find out more about what we do and how we can support your brand to thrive! #GeneTherapy #NeuromuscularDiseases #Novartis #Innovation #LucidQuest #Pharma #Strategy #Insights

Completing this deal is quite impressive if you consider that Kate Therapeutics was launched last year with $51M. It allows Novartis to expand its presence in both neuroscience drug development and gene therapy. Kate Therapeutics last year licensed to Astellas Pharmaceuticals exclusive rights to develop and commercialize KT430, a treatment designed to deliver a functional copy of the MTM1 gene via a novel MyoAAV capsid to treat X-linked myotubular myopathy (XLMTM). DELIVER has the potential to significantly improve the efficiency and effectiveness of gene therapy. By using machine learning to evolve AAV capsids, it could lead to increased potency, improved safety, and faster development. DELIVER capsids could deliver gene therapies more effectively to target tissues, leading to stronger treatment effects. By minimizing off-target delivery to organs like the liver, DELIVER could reduce potential side effects. Machine learning could accelerate the process of identifying optimal capsids for specific gene therapies. This acquisition underscores the growing importance of gene therapy and the innovative approaches being developed to address unmet medical needs.