World Pharmaceutical Frontiers’ Post

Transforming Healthcare: The Rapid Growth and Impact of the Cell & Gene Therapy Market Compared with gene therapies, 46.1% of cell therapies in development are in clinical phases. The most targeted therapeutic areas are oncology, the central nervous system and infectious diseases. The rapid growth and high investment in the CGT market can attract more stakeholders, including investors, researchers, healthcare providers, and patients. This dynamic ecosystem will foster collaboration and drive the overall advancement of cell and gene therapies. The FDA's involvement and the increasing number of approved therapies signal a maturing regulatory environment that is adapting to accommodate the unique challenges and opportunities presented by CGTs. This can lead to more streamlined approval processes and better-defined pathways for bringing new therapies to market. With over 2,500 companies developing cell and gene therapies and nearly 1,800 therapies in clinical trials, the industry is seeing significant expansion. This growth in the number of companies and therapies underscores the increasing interest and competition in the market, which can lead to innovation and advancements in the field. Drop your thoughts in the comment below! For more detail updates of the healthcare and medical device industry, follow our page HealtHive Market Research #FDA #CGT #celltherapy #genetherapy #healthcare #clinicaltrials #OTAT #oncoology #cardiology #centralnervoussystem #infectiousdiseases #Luxturna #Yescarta #Abecama #Kymriah #cancer

Join Us at ICAR 2024! We invite you to visit our poster presentation on preclinical data for CAP-004, our next-generation gene therapy for Friedreich’s ataxia, at the International Congress for Ataxia Research (ICAR) in London! This innovative therapy has the potential to address neurodegenerative, cardiovascular, and sensory manifestations of this challenging condition with a single administration. Poster Title: Systemic AAV Gene Therapy with Next Generation Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich’s Ataxia 🗓 Date: November 13, 2024  🕕 Time: 6:00 PM GMT  📍 Location: Leonardo Royal Hotel London Tower Bridge  🔍 Session: Emerging and Existing Therapeutics – Preclinical Research  📜 Poster Number: 477  🎤 Presenter: Celeste Stephany, Ph.D., Director of CNS and Ophthalmology Preclinical Research, Capsida This is a unique opportunity to explore our preclinical data demonstrating the promise of CAP-004. Our findings reveal: 🔎 CNS Impact: CAP-004 resulted in human frataxin protein levels 8-fold higher than endogenous protein levels in the motor cortex of non-human primates. 🔎 Cardiac Impact: Human frataxin protein levels in non-human primates reaching 1.7 times higher than endogenous levels, with expression across nearly 30% of the heart left ventricle tissue. 🔎 Sensory Vision Benefits: Meaningful RNA expression detected in the retina. 🔎 Safety Profile: Significant liver detargeting and other non-target tissues; and a favorable safety profile For more detailed insights check out our press release here: https://lnkd.in/gGP84sr4 Don’t miss this chance to connect! We look forward to seeing you there. #ICAR2024 #CapsidaBiotherapeutics #GeneTherapy #FriedreichsAtaxia #Biotech

2025 Outlook For Cell & Gene Therapy! I'm excited to see the incredible advancements in cell and gene therapies coming in 2025. Companies like Kiji Therapeutics, Orgenesis, and Precision BioSciences are pushing the boundaries, expanding treatments beyond oncology to genetic disorders, autoimmune diseases, and cardiovascular conditions. With improvements in gene-editing and scalable manufacturing, these innovations are set to make personalized medicine more accessible and impactful than ever before! https://lnkd.in/ee8WPsxU

I am happy to share that our latest research paper, "Rabies virus-mimicking liposomes for targeted gene therapy in Alzheimer’s disease," has been published in the International Journal of Pharmaceutics, one of the prestigious journals in our field. Thanks to all authors Hasan Akbaba, Ozan Karaman, Ezgi Turunç Özoğlu, Tian Tian for their valuable contributions and especially  Prof. Bakhos Tannous for his continuous support. Also, I would like to thank TÜBİTAK for supporting this study by 3501 - Career Development Program.   In this study, a gene carrier system combining the synergistic effect of virus-mimic RVG29 ligand with bioinspired liposome that imitates the natural lipid bilayers of cell membranes was developed for brain-targeted RNAi therapeutics. You can access the article from the link below:

Gene therapy is revolutionizing modern medicine, but finding a safe and effective delivery vehicle remains a critical challenge. While liposomes offer significant promise over viral-based delivery methods, they struggle with limited delivery efficiency across various cell types. A key obstacle is our incomplete understanding of the cellular structures necessary for liposome uptake, particularly in migratory versus epithelial cells. In our latest research, we've discovered the cellular structures required for efficient liposome-based gene delivery using human pluripotent stem cells. This advancement could pave the way for more effective and safer gene therapy applications in the future. #GeneTherapy #BiomedicalResearch #StemCells #Liposomes #MedicalInnovation #NIGMS #NIH #NEI Basic and Translational Research, Dept of Ophthalmology, IUSM https://lnkd.in/gvCfnAwM

Driving innovation in cell and gene therapy!💡 Today I had the privilege of visiting our valued partner 🤝 Harro Höfliger to see their cutting-edge aseptic bag filling application including a Franz Ziel GmbH isolator. Together, we’re completing the final integrated acceptance of this turnkey production solution. Thanks to Christian Kollecker for hosting me! In cooperation with the customer of this line we have a clear mission: To advance scientific understanding and medicine by enabling groundbreaking solutions in cell and gene therapy and biomedical research. 🌱🔬 As therapeutics shift from traditional chemistry-based approaches to individualized, cell-based treatments and targeted drugs, we are proud of being part of this innovation. 🚀 By empowering safer and more efficient production processes, we’re helping to bring transformational treatments to life - moving closer to a future where cancer, autoimmune diseases, and neurodegenerative disorders are a thing of the past. 🧬✨ #cellandgenetherapy #biomedicalresearch #innovation #partnership #futureofmedicine

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

John James OBE, CEO Sickle Cell Society, is a key speaker at our forthcoming event exploring the exciting potential of gene therapy and other advanced therapeutic medicinal products (ATMPs) on Thursday 11 July. Gene therapy addresses genetic issues by adding, removing, or altering genes. Cell therapy uses cells, like stem cells, to treat diseases or repair damaged tissues. Tissue-engineered products grow new tissues or organs outside the body for transplantation. They are all ATMPs and are giving hope to patients with a variety of conditions, including genetic diseases, blood disorders and cancer, who previously had limited or no treatment options. Future developments are also likely to transform the treatment of common conditions such as diabetes, cardiovascular disease and dementia. Book now for an essential briefing into these revolutionary treatments shaping the future of healthcare: 👉 https://bit.ly/4cnylBI #genetherapy #atmps #healthcare

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.

The world of medicine is witnessing a seismic shift, with Cell and Gene Therapy (CGT) at the forefront. These transformative therapies are tackling some of the most challenging diseases, offering hope where traditional treatments fell short. 📈 Key Highlights from Novotech’s Whitepaper: Global Growth: Clinical trials have surged by 32.5% (2019-2023), with over 1,500 therapies in development. Asia-Pacific Leads: This region accounts for 44% of global trials, driven by innovation in China and efficient patient recruitment timelines. CAR-T Therapies Rule: Representing 32% of the pipeline, these therapies are redefining cancer treatment. #CellAndGeneTherapy #Innovation #MedicalBreakthroughs #HealthcareTransformation #NovotechInsights