Neil Hunter’s Post

🚀 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  

Thank you to Erin Harris for a great article on 2025 Outlook for Cell and Gene Therapy (CGT): Key Trends to Watch The CGT industry is set for transformative advancements in 2025, focusing on: 1️⃣ Non-Oncology Indications: Broader applications in genetic disorders, autoimmune diseases, and cardiovascular conditions, with scalable manufacturing and affordability driving accessibility. 2️⃣ In Vivo Gene Editing: Enhanced precision and delivery methods, such as lipid nanoparticles and novel AAV capsids, unlocking potential for complex diseases. 3️⃣ Point-of-Care (POC) Innovations: Miniaturized bioreactors, automation, AI-driven optimization, and decentralized production models, enabling cost-effective and accessible therapies globally. It is noted that resilience, scalability, and innovation are essential for advancing CGT in a challenging biotech landscape. The future of CGT promises revolutionary treatments and broader access to life-saving therapies. #CellAndGeneTherapy #BiotechInnovation #GeneEditing #PointOfCare #HealthcareTransformation #PersonalizedMedicine #LifeSciences

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

🔬 2025 Cell and Gene Therapy Outlook: Insights from Industry Leaders as Interviewed by Erin Harris 🚀 Here’s what industry experts are saying about the trends shaping the future: 1️⃣ Non-Oncology Indications Are Gaining Traction Dr. Miguel Forte, CEO and Co-Founder of Kiji Therapeutics, highlights the need for scalable production: "Developing our MSC product sourcing the cells from iPSC is geared at achieving that scalability." 2️⃣ In Vivo Gene Editing Reaches New Heights Dr. Cassie Gorsuch, VP of Gene Therapy at Precision BioSciences, shares her excitement about tackling chronic diseases: "Our program for chronic hepatitis B aims to potentially cure the disease by eliminating the root source of viral replication." 3️⃣ Point-of-Care (POC) Technologies Drive Efficiency Vered Caplan, CEO of Orgenesis, underscores the importance of streamlining CGT manufacturing: "Miniaturized, automated bioreactors, AI-driven optimization, and closed-system processing are transforming precision and safety."

Would you like to know more about the Australian cell and gene therapy landscape? Heather Main and HOYA consulting worked with the Stem Cell network at the University of Melbourne to map out the sector. Key take aways - there are >22 companies in Australia developing advanced therapies, with most based in Victoria. These include CAR-T, and pluripotent stem cell derived therapies including CAR-NK. Australian developers are also focused on in vivo gene therapies, with dedicated clinical trial units such as Cerulea Clinical Trials leading the way. Significant investment and support from Therapeutic Innovation Australia, AusBiotech via the Cell and Gene Therapy Catalyst, and state investment in WA, QLD, NSW and Victoria mean that we have innovation hubs linked to cGMP manufacturing capabilities. Download the report here, or check it out at https://lnkd.in/gPfmMg8N

Fresh news out of the clinic this week in the #cellandgenetherapy sector. 🧫New allogeneic CAR-T candidate for blood cancers announced from an ongoing partnership 🧬Positive data released from a clinical trial for gene therapy to treat Parkinson’s disease 🧪New partnership for scalable automation of cell and gene therapy manufacturing See the full stories below and subscribe to ARM's Sector Vector newsletter for more weekly news on cell and gene therapy clinical, business, and manufacturing. 📨 https://lnkd.in/eD7ZNaU Poseida Therapeutics, Inc. announced the nomination of a new development candidate under its collaboration with Roche. The candidate is an allogeneic, dual CAR-T therapy targeting known antigens expressed in hematologic malignancies, including multiple myeloma. https://lnkd.in/e8VZwgJY MeiraGTx announced top-line data from its clinical bridging study of a gene therapy to treat Parkinson’s disease. The study met its primary objective of safety and tolerability and saw clinically meaningful improvements in patients. https://lnkd.in/eQFXyBDW Cellular Origins and Fresenius Kabi signed a development agreement designed to digitally and physically integrate a suite of cell therapy processing technologies within a cell and gene therapy robotic manufacturing platform. https://lnkd.in/e7NT-_Fn

Exciting News from Berlin! Bayer AG and Charité – Universitätsmedizin Berlin have unveiled plans for the Berlin Center for Gene and Cell Therapies, set to become a leading biotech hub aimed at expediting groundbreaking cell and gene therapies to patients. Supported by Germany’s Federal Government and the State of Berlin, this state-of-the-art facility will feature GMP-certified production and an incubator for start-ups, with construction beginning in 2025. This initiative represents a unique collaboration between science, industry, and government, aiming to bridge the gap between research and patient care while positioning Berlin as an international biotech leader. By fostering innovation and supporting start-ups, the Berlin Center for Gene and Cell Therapies is set to pioneer the future of medicine and strengthen Germany's position in the global biotech ecosystem. #Biotech #GeneTherapy #CellTherapy #Healthcare

📊 Gene & Cell Therapy Landscape Report for Q3 2024 1️. New FDA Approval Marks Progress in Cell Therapy -Adaptimmune’s Tecelra (afamitresgene autoleucel) received FDA approval for synovial sarcoma. This MAGE-A4-directed, genetically modified autologous T-cell therapy highlights progress in personalized immunotherapy. 2️. Gene Therapy Trials Shift Towards Non-Oncology Indications -Non-oncology trials are on the rise, with 51% of gene therapy trials in Q3 2024 targeting non-cancer indications, up from 39% in Q4 2023. -Oncology remains prominent, as 52% of all gene therapy pipeline assets and 53% of rare disease gene therapies focus on cancer indications. 3️. Dealmaking Flat, but Start-up Financing Sees a Rebound -Q3 saw 101 deals in the advanced molecular therapy space, similar to Q2, with four notable acquisitions, including Agilent’s $925M acquisition of Biovectra in the CDMO sector. -Seed and Series A funding tripled to 19 deals, raising $484M in Q3—a nearly 2x increase over Q2. 📈 Data Source: Pharmaprojects | Citeline, October 2024 #GeneTherapy #CellTherapy #Biotech #FDAApproval #ClinicalTrials #Pharma

Exploring the Frontiers of Cell Therapy: Insights from Michael Mendicino, Ph.D. Dive into the evolving regulatory landscape and strategic CMC considerations in our latest SAB Perspectives blog with RoslinCT's Scientific Advisory Board member, Dr. Mendicino. Michael Mendicino is a multi-disciplinary cell & gene therapy subject matter expert with over 24 years of experience, educated in molecular/cellular biology/genetics, immunology, and regulatory science. Discover how innovation is shaping the future of therapies and the importance of collaboration in driving progress. Read the blog here: https://lnkd.in/eceARPD7 #CellTherapy #RegulatoryInnovation #CMCStrategy

What if we can manufacture life-saving therapies faster, safer, and more cost-efficiently? Day 21 of celebrating good news in cell and gene therapies: Limula raised $6.8m in seed round to enable commercial manufacture of their new system which combines bioreactor and centrifuge in a closed process vessel to handle a wide range of volumes and cell numbers, remove transfer steps and limiting stress, losses and contamination. This can potentially lead to production capacity increase. Really happy to see investments coming back into manufacturing technologies enabling CGT manufacturing - hopefully this is the start of many more other promising technologies getting funding to optimise manufacturing of these therapies and drive greater patient impact! If anyone is heading to ISCT, International Society for Cell & Gene Therapy Vancouver, there are a couple of really exciting launches in the CGT manufacturing space (Ori Biotech & Cytiva) that I personally can't wait to see. If you are there too, let's chat! https://lnkd.in/dS4ZKP8i #CGT #celltherapies #manufacturing #celebrate #monthofCGTcelebrations