Neil Hunter’s Post

#ICYMI, our President & Division Head, Gene Therapy Research & Technical Operations, Morten Sogaard, participated on a panel earlier this year at the PMWC - Precision Medicine World Conference to discuss the progress and challenges in emerging applications for gene and cell therapies. Here are some key takeaways from the discussion:

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

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

We are proud to announce our recent acquisition of the STAR CRISPR technology and all associated IP from Artisan Bio This cutting-edge technology allows for precision gene editing with minimum off-target occurrences compared to other systems. We plan to integrate STAR CRISPR into our Cellistic and Ncardia offerings and seek further partnerships to maximize the potential of this game-changing technology. Congrats to the Cellistic and Ncardia team! Learn more about this exciting development in our latest announcement. #geneticengineering #biotechnology #precisionmedicine

The healthcare industry is witnessing a transformative shift, with the emergence of Cell and Gene Therapies (CGT) in medicine. It addresses previously untreatable conditions and is paving the way for personalized, curative approaches to diseases like cancer, rare genetic disorders and more. CGT offers the promise of not just treating but curing diseases by repairing or replacing defective cells and genes. This leap in science could redefine patient outcomes and elevate the standard of care. The global CGT market is expected to grow exponentially over the next decade. Several major pharmaceutical companies are actively investing in the sector, recognizing its transformative potential in medicine. What are your thoughts on CGT’s potential to reshape the future of medicine? #CellAndGeneTherapies #Biotech #Innovation #CGT #LifeSciences

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

Reflections on ASGCT 2024 Annual Conference: The American Society of Gene and Cell Therapy (ASGCT) held its annual conference in Baltimore/MD on May 7-11, 2024.  With a record attendance on-site (>8'000) and presumably a large number attending online, the conference highlighted the latest trends in the field, from basic research to preclinical and clinical development and ultimately results from clinical use of these new modalities. The scientific sessions were supported by a very large number of posters (>2'000) and a very large exhibition with companies (>350) offering tools and technologies for that area. And finally, the event has also been an excellent opportunity for networking. It is difficult to pick specific highlights from a conference which runs over 5 days with dozens of workshops, keynote lectures, multiple parallel scientific tracks and numerous sponsored events, often at the same time.  Nevertheless, I would like to highlight my personal favorites (in random order): 1. Huge advancements in development of novel LNP (lipid nano particles) 2. Huge advancements in development of better AAV (adeno-associated virus) 3. Huge advancements in development of better genome/epigenome tools 4. Huge advancements in development of tools for gene insertion 5. Advancements in scale-up of vectors (viral/non-viral) and cells 6. Advancements in CMC (Chemistry, Manufacturing, and Controls) 7. Advancements towards in-vivo genome editing 8. Advancements towards better CAR-T/-NK/Mp therapies (same for TCR) 9. Advancements in understanding of CAR-T safety  10. Advancements towards smarter ATMPs (2nd/3rd generation cells/vectors) https://lnkd.in/e-jdsEPa https://lnkd.in/eywVhG9V https://lnkd.in/e2n844mw https://lnkd.in/emzfY8G8 https://lnkd.in/edXcbBwi https://lnkd.in/eqSVfR8v https://lnkd.in/eC8Hf7xD

🚀 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  

📊 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

"The End of the Beginning for Cell and Gene Therapy, Part2" Manufacturing and Commercialization Challenges: Cell and gene therapy face hurdles similar to early biologics, with high costs and complex manufacturing processes needing streamlining to meet large-scale demand. Infrastructure Development: Unlike past antibody therapeutics, cell and gene therapy is benefiting from earlier infrastructure development efforts, which may speed up the reduction of manufacturing costs. Cost and Scalability Pressures: Even established CDMOs like Lonza must rapidly innovate to reduce costs, especially for personalized autologous treatments, to make these therapies widely accessible.