Cell&Gene Consulting Inc.’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

The Importance of Specialized Training in Advanced Cellular Therapy Management. This new article explores the importance of specialized training in managing advanced cellular therapies, with a focus on #CAR-T therapies. It discusses how these therapies are revolutionizing the treatment of certain forms of #cancer, the skills required to manage a CAR-T center, the importance of a robust quality system, and the challenges in managing clinical trial projects. Additionally, it emphasizes the need for continuous improvement to maintain high standards of quality and #safety. 📖 Read the full article to delve deeper. Obtain the qualification of Car-T Specialist and Gene Therapy Expert through a comprehensive training program that covers all aspects of treatment, from theory to practice, including organizational protocols and current regulations. You will also be officially recognized in the international registry. 👨🏫 Speakers: Dr. vincenzo iaconianni, CEO of ICMED srl, internationally recognized expert in quality system design. Dr. Giorgia Saporiti, hematologist, director of the clinical unit at Policlinico di Milano, affiliated with the Milano Transplant Network. Dr. @Alessandra Bo, director of the cell manipulation laboratory at IRCCS Policlinico San Martino in Genoa. Prof. Vincenzo Salvatore, Full Professor of European Union Law at the University of Insubria, Varese, expert in ATMP. 📚 Program: What is a gene therapy product and CAR-T? Drug or Tissue? Production or Administration European and national regulatory framework The role of the Cell Therapy and Car-T Specialist from the Clinical Center perspective The role of the Cell Therapy and Car-T Specialist from the Pharmaceutical Company perspective 📅 When: Friday 27/09/24: ⏲10-13 & 14-17  / Saturday 28/09/24: ⏲9-13 Friday 04/10/24: ⏲10-13 & 14-17 / Saturday 05/10/24: ⏲9-13 🌐 Mode: In-person at Hotel The Building, via Montebello n. 126 – ROME 📜 Certification: Car-T Specialist and Gene Therapy Expert Certificate Registration in the international registry of Car-T Specialist and Gene Therapy Experts More details and registration: https://lnkd.in/d-NmM7tz Dr. Chandana Kamili Manoj Kumar Reddy Nidhi Singh, Ph.D Selva karthick #QualityManagement #JACIEAccreditation #TransplantProgram #ClinicalPractice #StemCellTransplantation #NonCompliance #AdverseEvents #Cryopreservation #Sepsis #MRSA #HealthWorkers #Training #RootCauseAnalysis #ImmunologicalDisorders #InfectionControl #QualitySystem #CellTherapy #GeneTherapy #ClinicalTrials #GoodClinicalPractice #SOPManagement #CAR_TCellTherapy #eucancerplan #oncology #pharmacy #clinicaltrials #pharmacovigilance  

Explore the transformative advancements in Biotech that are redefining healthcare. Our latest blog explores how gene editing, tissue engineering, and stem cell therapy are paving the way for groundbreaking treatments, underpinned by data and real-world examples such as: 💉 75% of experts anticipate increased investment in stem cell research over the next three years. 🧠 Tissue Engineering and Bioprinting industries expected to reach $5.1 billion by 2030, with 39% of experts highlighting its impact on creating functional tissues for organ replacement. 🚀 90% of experts anticipate a surge in market demand for CRISPR-Cas9 technologies in future. Discover more about these cutting-edge technologies and their impact: https://lnkd.in/evRVUB7h #Biotech #CRISPR #GeneEditing #TissueEngineering #StemCellTherapy #Pharma

🚀 Exciting Developments in Cell & Gene Therapy! 🚀   The cell & gene therapy landscape is buzzing with two major acquisitions in the last week: 1. Roche's acquisition of Poseida Therapeutics, Inc. Therapeutics for up to $1.5B. This strategic move will bolster Roche's capabilities in off-the-shelf CAR-T therapies, aiming to democratize access to these life-saving treatments. Poseida's innovative non-viral technology platform, rich in T stem cell memory cells, will hopefully enhance the safety and efficacy of CAR-T therapies, potentially benefiting a broader patient population. 2. Meanwhile, Novartis has acquired Kate Therapeutics in a deal valued at up to $1.1B. This acquisition aligns with Novartis' commitment to advancing gene therapies for neuromuscular diseases. Kate Therapeutics' cutting-edge AAV-based gene therapy platform and promising preclinical candidates for conditions like Duchenne muscular dystrophy and myotonic dystrophy type 1 will strengthen Novartis' pipeline and innovation in gene therapy.   These acquisitions underscore the relentless pursuit of innovation in the biotech industry, aiming to bring transformative therapies to patients worldwide. #GeneTherapy #HealthcareInnovation #CART  

👂🌟 Regeneron's DB-OTO Gene Therapy Shows Promise in Treating Genetic Deafness! 🔍 In a ground-breaking development, Regeneron Pharmaceuticals' investigational gene therapy, DB-OTO, has demonstrated significant efficacy in treating profound genetic deafness in children, as revealed in the Phase 1/2 CHORD trial. DB-OTO, a cell-selective adeno-associated virus (AAV) gene therapy, targets variants of the otoferlin gene responsible for congenital hearing loss. Two young participants showed promising results: one child achieved normal hearing levels within 24 weeks, while the other displayed initial improvements after six weeks. Audiometry tests indicated an average 84dB improvement in key speech frequencies for the first participant and a 19dB average improvement for the second. 💡 These findings suggest DB-OTO's potential as a transformative therapy for otoferlin-related deafness, offering hope for enhanced speech development and quality of life. Regeneron's commitment to innovative medical solutions continues to drive progress in addressing unmet medical needs. Clinical trial investigator and Columbia University’s Department of Otolaryngology – Head & Neck Surgery chair Lawrence Lustig said: “These impressive results showcase the revolutionary promise of DB-OTO as a potential treatment for otoferlin-related deafness, and we are excited to see how this translates into an individual’s development, especially since early intervention is associated with better outcomes for speech development.” To read similar ground-breaking news, subscribe to the World Pharmaceutical Frontiers page!! #Regeneron #GeneTherapy #CHORDTrial #HearingLoss #MedicalAdvancements #worldpharmaceuticalfrontiers

As we draw to the end of the year, the #lifescience, #biopharma, and #CGT industries are reviewing what happened in #2024, and looking forward to #2025. Here, Erin Harris, Chief Editor at Cell & Gene, the Life Science Connect publication, asks 3 KOLs about the outlook for 2025 in the #cellandgenetherapy sector. Miguel Forte, President, ISCT, International Society for Cell & Gene Therapy, and CEO, Kiji Therapeutics, and Vered Caplan, CEO, Orgenesis Inc. "As technology improves, and is doing so rather quickly, we will be better at accelerating the development and improving conditions of adoption and use, making the field delivering value to patients and stakeholders faster.” Miguel Forte, MD, Ph.D. Incorporating the advancements in automation, closed systems, quality control and IT solutions into a standard platform will make POC accessible for many institutes and enable a more efficient approach for the clinical development stage and will lay the basis for enhancing accessibility and reducing costs for commercial stage products.” Vered Caplan https://lnkd.in/eFWHqkpW

Novartis has announced a $1.1 billion deal with Kate Therapeutics, a San Diego-based biotech focused on developing innovative gene therapies for challenging conditions like Duchenne muscular dystrophy (DMD), facioscapulohumeral dystrophy (FSHD), and myotonic dystrophy type 1 (DM1). The deal is the combination of upfront cash and milestone payments. This deal comes as the muscular dystrophy field faces setbacks, with high-profile failures reminding us of the hurdles in tackling these diseases. However, Novartis’ CEO, Vas Narasimhan, emphasized how Kate’s platform—designed to target muscle and cardiac tissue while avoiding the liver—could help address these challenges and advance treatments for neuromuscular conditions. Building on its prior experience with Zolgensma, Novartis aims to learn from past industry struggles while pushing the boundaries of gene therapy for complex diseases. Read more in the following article in Fierce Biotech: https://lnkd.in/erYpX2Vn #musculardystrophy #aav #deliver #miRNA #novartis

To propel regenerative medicine into a new era of possibilities, we must find ways to deliver effective #CellTherapies at scale.  President of the Astellas Institute for Regenerative Medicine, Erin Kimbrel, shared her insights with Cell & Gene, on the innovative technologies Astellas is employing to deliver these next-generation therapies to more patients, faster: https://bit.ly/3WRqrw7. #ScienceFirstForPatients #ChangingTomorrow

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

🧬 Dyno Therapeutics has announced a new partnership with Roche valued at up to $1.05 billion to develop next-generation AAV gene therapy vectors targeting neurological diseases. Utilizing Dyno’s AI-powered platform, the collaboration aims to overcome key challenges in AAV gene therapy, including delivery efficiency, immunity issues, and manufacturability. The advanced AAV capsids will be tailored specifically for neurological applications, marking a step forward in effective in vivo gene delivery. 💰 The deal provides Dyno with an initial $50 million payment, and includes additional milestone payments and royalties. This marks the second partnership between the companies, following a 2020 collaboration for neurological and liver therapies. 🧪 Dyno’s Low-Shot Efficient Accelerated Performance (LEAP) technology enables billions of in vivo sequence-function measurements each month, allowing rapid optimization of AAV capsids for specific tissue targeting. Under the new agreement, Dyno will lead in designing and discovering enhanced AAV capsids, while Roche will focus on preclinical and clinical validation, as well as commercialization of neurological gene therapies using these novel vectors. 🧠 Roche’s business development lead, Boris L. Zaïtra, highlighted the importance of the partnership, noting that combining Roche’s therapeutic expertise with Dyno’s advanced capsid engineering opens up opportunities for tackling historically difficult-to-treat neurological diseases. #GeneTherapy #Neurology #DynoTherapeutics #Roche #AAVVectors #BiotechInnovation #AIinBiopharma #PrecisionMedicine #NeurologicalTherapies #BiopharmaPartnership