Vishwa Savla’s Post

👩🔬 As Gene therapy is one of my favorite fields, I want to share this amazing timeline of products produced by this technique. " Since gene therapy was first used as a disease treatment modality, there have been a lot of ups and downs. The growing number of gene therapy products available on the market, however, indicates that the field of gene therapy has reached a significant turning point in its development. Given the products that are now approved and those that are pending approval, along with the many clinical trials being conducted in this area, gene therapy appears to have a bright future. Pharmaceutical businesses, legislators, and researchers may find it useful to track trends in modifications to gene therapy techniques, vectors, and targets. The majority of gene therapy clinical trials are currently being conducted for cancer, despite the fact that gene therapy was first used to cure hereditary illnesses. Modifications to gene therapy tactics, especially in countries that are leaders in this sector, could indicate the path for upcoming clinical products." You can find the full article in the below link, It is open-access fortunately 😊 https://lnkd.in/gvQtWqJD #geneediting #gene_therapy #gene

🔬  𝗪𝗵𝗮𝘁 𝗦𝘂𝗽𝗽𝗹𝘆 𝗖𝗵𝗮𝗶𝗻 𝗣𝗿𝗼𝗳𝗲𝘀𝘀𝗶𝗼𝗻𝗮𝗹𝘀 𝗦𝗵𝗼𝘂𝗹𝗱 𝗞𝗻𝗼𝘄 𝗔𝗯𝗼𝘂𝘁 𝗖𝗲𝗹𝗹 𝗮𝗻𝗱 𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝗶𝗲𝘀 🔬 Cell and gene therapies (𝗖𝗚𝗧𝘀) are a rapidly growing field of medicine that offers the potential to cure a wide range of diseases, including cancer, rare diseases, and autoimmune disorders. However, the supply chain for CGTs is complex and challenging, requiring a deep understanding of the unique requirements of these therapies. 🧬 𝗣𝗲𝗿𝘀𝗼𝗻𝗮𝗹𝗶𝘇𝗮𝘁𝗶𝗼𝗻: Cell and gene therapies are personalized, meaning that each patient's therapy is 𝗺𝗮𝗱𝗲 𝘁𝗼 𝗼𝗿𝗱𝗲𝗿. This requires a high degree of flexibility and agility in the supply chain. ❄️ 𝗧𝗲𝗺𝗽𝗲𝗿𝗮𝘁𝘂𝗿𝗲 𝗦𝗲𝗻𝘀𝗶𝘁𝗶𝘃𝗶𝘁𝘆: Cell and gene therapies must be stored and transported at a cold temperature, typically -𝟴𝟬°𝗖 or below. This adds complexity to the supply chain and requires specialized equipment and trained personnel. ⏳ 𝗦𝗵𝗼𝗿𝘁 𝗦𝗵𝗲𝗹𝗳 𝗟𝗶𝗳𝗲: Cell and gene therapies have a short shelf life, typically only a few days or weeks. This further complicates the supply chain and requires careful planning to ensure the therapies are delivered to patients on time. 📑 𝗥𝗲𝗴𝘂𝗹𝗮𝘁𝗼𝗿𝘆 𝗥𝗲𝗾𝘂𝗶𝗿𝗲𝗺𝗲𝗻𝘁𝘀: Cell and gene therapies are subject to strict regulatory requirements. This can add complexity to the supply chain and require careful documentation and tracking. These challenges can make managing the supply chain for cell and gene therapies difficult. However, several solutions can be implemented to address these challenges. 🏭 𝗖𝗲𝗻𝘁𝗿𝗮𝗹𝗶𝘇𝗲𝗱 𝗠𝗮𝗻𝘂𝗳𝗮𝗰𝘁𝘂𝗿𝗶𝗻𝗴: This can help to improve efficiency and reduce costs. ❄️ 𝗜𝗻𝗻𝗼𝘃𝗮𝘁𝗶𝘃𝗲 𝗖𝗼𝗹𝗱 𝗖𝗵𝗮𝗶𝗻 𝗦𝗼𝗹𝘂𝘁𝗶𝗼𝗻𝘀: This can help to ensure the safe and efficient transportation of cell and gene therapies. 🔗 𝗕𝗹𝗼𝗰𝗸𝗰𝗵𝗮𝗶𝗻 𝗧𝗲𝗰𝗵𝗻𝗼𝗹𝗼𝗴𝘆: This can help to improve traceability and security. 🚚 𝗧𝗵𝗶𝗿𝗱-𝗣𝗮𝗿𝘁𝘆 𝗟𝗼𝗴𝗶𝘀𝘁𝗶𝗰𝘀 (𝟯𝗣𝗟) 𝗣𝗿𝗼𝘃𝗶𝗱𝗲𝗿𝘀: This can help to share expertise and resources. 💰 The global market for these therapies is projected to skyrocket, growing from $6.02 billion in 2017 to $35.4 billion in 2026. 💡 Companies like Marken, World Courier, QuickSTAT , Cryoport, and others are leading the charge, offering specialized services and technologies to meet the unique demands of cell and gene therapy logistics. Their efforts contribute to the successful commercialization of advanced therapies and ensure patients benefit from groundbreaking treatments. #cellandgenetherapy #supplychain #healthcare #innovation

🌟 Transforming Lives Through Gene Therapy: The Journey from Challenges to Breakthroughs 🌟 🎉 Exciting news from the medical field has once again highlighted the profound impact of cell and gene therapies on individual lives. A recent breakthrough reported by the UK NIHR (National Institute for Health and Care Research) details how a young girl named Opal, born deaf, can now hear and communicate thanks to pioneering gene therapy developed by Regeneron. This remarkable achievement not only enhances Opal’s quality of life but also opens a whole new world of possibilities for her future. 🚧 However, the hope and potential for life-changing impact that cell and gene therapies can bring are not devoid of challenges. As outlined in a comprehensive paper published in Pharmaceutical Medicine (https://lnkd.in/e9wDnZgV), the road to developing these treatments is filled with scientific, regulatory, and ethical complexities. These include technical challenges in gene manipulation, stringent and lengthy approval processes, ethical dilemmas over genetic inheritance, scalability issues, and immune responses that can limit therapy effectiveness or safety. These therapies require not just ground-breaking research but also robust support systems to navigate these challenges. 🔍 Here at Inbeeo, we provide strategic insights to accelerate patient access to innovative cell and gene therapies in all markets. We support companies by focusing on: 👉 Identifying key opportunities where these advanced therapies can meet unmet medical needs 👉 Navigating complex market access and pricing landscapes 👉 Setting competitive and sustainable price points that reflect the value your therapy will bring to patients, healthcare systems, and society 👉 Outlining approaches to reach healthcare providers and patients effectively 💬 Let’s Chat! 💬 Reach out for an informal introduction to the Inbeeo team, and let’s explore how we can be your partner in devising access strategies for cell and gene therapies across your target markets. #GeneTherapy #HealthcareInnovation #MedicalResearch #PatientCare #Biotechnology

Cell and gene therapies are the best - and often only - hope for a cure for many rare disease patients. But, they still take far too much time and resources to develop. Greater standardization - of chemistry manufacturing and controls (CMC), regulatory approaches, as well as key terminology - will be vital to the future of cell and #genetherapies. What an insightful day 1 of the Gene Therapy Product Activity & Comparability & the Evaluation of T-Cells Workshop! Some of the key take-aways from today's workshop: - The need for manufacturing changes is common - e.g., to scale up or out, improve quality, reduce cost, or address supply-chain challenges; - Not all manufacturing changes are created equal and some will pose a much larger risk to patients than others. Risk assessment approaches can help prioritize which characteristics to evaluate (and how). - Plan today for future manufacturing changes; consider what data, meta-data, and samples you may need to collect today to be ready when manufacturing changes are needed. - Engage statisticians from the start to ensure your comparability study is appropriately powered and actually proves what you hope to prove. - Where you are the in product development program matters; larger manufacturing changes are easier to implement early in development; and the evidentiary standards pre- to post-approval are distinct. - Greater standardization is vitally important, but not all areas of CMC are equally suited for standardization (today); prioritizing where to standardize (first) will be key. - And a variety of different standards already exist today, and not all standards are created equal. FDA -CBER's new Standards Recognition Program for Regenerative Medicine Therapies will help create some clarity. - The field of cell and gene therapies is rapidly-developing and it can be tough to catch up with the status of science and technology. Thank you to FDA, US Pharmacopeia and Standards Coordinating Body (SCB) for hosting such an inspiring conversation!

How much AAV do we need? The attached infographic summarizes the challenges in AAV production for several of the currently approved gene therapies. It is quite helpful to review the challenges accompanied with a relatively high dose (1.0 x 10E13 - 1.0 x 10E14 vg/kg) translating to high cost of goods, and issues with regards to toxicity and immunogenicity for these treatments. When reviewing these data, we should be aware that a 500 liter bioreactor process under GMP conditions might cost approx. 1.0 - 1.5 mio USD per run (fully loaded costs). Taking into consideration the relatively high cost of goods (COGS) and the required dose per therapy for all therapies on the market or in clinical trials, one will see immediately the most pressing needs for AAV therapies: 1. Better selectivity of various serotypes for certain cell-types  (hopefully translating in lower dose, lower COGS, less toxicity) 2. Better infectivity of various serotyps for certain cell-types  (hopefully translating in lower dose, lower COGS, less toxicity) 3.  Better production processes for AAV production upstream/downstream (hopefully translating to much lower COGS per dose) A big thank you to Mischa Schwendy (Cytiva, Germany) for generating and sharing that highly valuable information and very fruitful discussions on future avenues for solving these problems. Please contact me when you are working on these challenges for widespread use of AAV in gene therapy. I am happy to connect you to other people working in the field. This is urgently needed for the benefit of patients. .

FDA approves Gene Therapy for non-ambulatory Patients with Duchenne Muscular Dystrophy Jun 20, 2024 It is known that DMD is a rare disorder due to a defective gene. It results in abnormalities in, or absence of, dystrophin. This protein helps keep the body’s muscle cells intact. DMD worsens over time, leading to weakness and wasting away of the body’s muscles. Symptoms include trouble walking and running, falling frequently, fatigue and learning disabilities. Individuals with DMD may also experience heart / respiratory issues, due to weakening of muscles. Previously, Delandistrogene moxeparvovec-rokl (Elevidys; Sarepta) was approved for ambulatory individuals with DMD in June 2023. Now approved for non-ambulatory individuals with DMD. Elevidys is a recombinant gene therapy. It has been designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin. Micro-dystrophin is a shortened protein (138 kDa, compared to the 427 kDa dystrophin protein of normal muscle cells). It contains selected domains of the dystrophin protein present in normal muscle cells. Elvidys is administered as a single IV dose. #geneticdisorder #researchanddevelopment #innovation #healthcare #lifesicences #biotechnology #pharmaceuticals #genetherapy

If in a position to help your company's health benefit programs, it's crucial to recognize the transformative potential of gene therapy products. Here are three key takeaways: 1️⃣ Costly Challenges: The advent of gene therapy products offers unprecedented hope, but they can also present significant financial hurdles for employees and employers alike. A single employee receiving gene therapy treatment can substantially impact your company's costs. 2️⃣ Promise of Gene Therapy: These therapies have the power to cure diseases and enhance lives significantly. Consider Zynteglo, a gene therapy for Beta-Thalassemia, a genetic blood disorder. It's a game-changer, but it can come with a hefty price tag, ranging from $2.8 - $3.6 million. 3️⃣ The Urgency of Preparedness: As the FDA greenlights more gene therapy drugs, it's imperative that employers act proactively. Zolgensma, Spinraza, Luxturna, Skysona, and Zynteglo are just a few examples with costs between $800,000 to $3,000,000. More approvals are on the horizon, and readiness is key. 🚀 Take Action Today 🚀 Engage with your consultants now to strategize effective management within your benefits plan. Consider these vital questions: 🤔 How are gene therapy and specialty drugs currently covered in your plan? 🤔 Are they partially or completely covered, or perhaps excluded? 🤔 What cost containment strategies are being explored to address these life-changing medications? Remember, adaptation doesn't have to be immediate. A 3-5-year plan with your consultant can ensure a seamless transition that supports your workforce without disruption. #HealthBenefits #BusinessStrategy #EmployeeWellbeing

When Rubber Hits The Road Why Gene Therapy is Struggling to Become Profitable Though there are so many potentials like CRISPR, CAR-T,alloCAR, and AAV, Nonviral based.… for many deadly diseases yet the profit margins need a wider range 👉but challenges to make CGT profitable are the golden opportunity for India to fix the hindering 🔰Regulatory Framework: India's regulatory framework for cell and gene therapy was still evolving and might have lacked clarity, causing delays in approvals and uncertainty for developers. 🔰Infrastructure and Expertise: Establishing the necessary infrastructure and expertise for cell and gene therapy research and manufacturing could have been a significant challenge in India, leading to slower progress in this field. 🔰Funding and Investment: Adequate funding and investment in cell and gene therapy research might have been limited, which could have impacted the development and commercialization of treatments. 🔰Intellectual Property Issues: Intellectual property protection is crucial for attracting investment and fostering innovation. Challenges related to intellectual property rights might have discouraged companies from investing in cell and gene therapy development in India. 🔰Public Awareness: Low public awareness and understanding of cell and gene therapies could have led to skepticism or reluctance to participate in clinical trials or adopt these treatments. https://lnkd.in/dSb7YRdf

𝐔𝐧𝐥𝐞𝐚𝐬𝐡𝐢𝐧𝐠 𝐭𝐡𝐞 𝐏𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥: 𝐓𝐡𝐞 𝐆𝐫𝐨𝐰𝐭𝐡 𝐓𝐫𝐚𝐣𝐞𝐜𝐭𝐨𝐫𝐲 𝐨𝐟 𝐭𝐡𝐞 𝐆𝐥𝐨𝐛𝐚𝐥 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐌𝐚𝐫𝐤𝐞𝐭 As we step into a new era of healthcare innovation, the landscape of medicine is being reshaped by groundbreaking advancements in gene therapy. The Global Gene Therapy Market, which soared to approximately 𝐔𝐒$ 𝟑.𝟒𝟒 𝐛𝐢𝐥𝐥𝐢𝐨𝐧 by 2022, is poised for a remarkable journey ahead, with forecasts indicating a 𝐜𝐨𝐦𝐩𝐨𝐮𝐧𝐝𝐞𝐝 𝐠𝐫𝐨𝐰𝐭𝐡 𝐫𝐚𝐭𝐞 𝐧𝐞𝐚𝐫𝐢𝐧𝐠 𝟐𝟒% over the period 2023-2030. Gene therapy, once a futuristic concept, has now emerged as a tangible reality, offering promising avenues for treating a myriad of genetic disorders and chronic diseases. 𝐑𝐞𝐪𝐮𝐞𝐬𝐭 𝐨𝐟 𝐭𝐡𝐢𝐬 𝐒𝐚𝐦𝐩𝐥𝐞 𝐑𝐞𝐩𝐨𝐫𝐭 𝐇𝐞𝐫𝐞 -https://lnkd.in/g-RA7icc This transformative approach involves the introduction, alteration, or deletion of genetic material within an individual's cells to correct or combat diseases at their root cause. From rare genetic disorders to complex ailments like cancer and cardiovascular diseases, gene therapy holds the potential to revolutionize treatment paradigms and enhance patient outcomes. 𝐓𝐨𝐩 𝐏𝐥𝐚𝐲𝐞𝐫𝐬 𝐢𝐧 𝐭𝐡𝐞 𝐆𝐞𝐧𝐞 𝐓𝐡𝐞𝐫𝐚𝐩𝐲 𝐌𝐚𝐫𝐤𝐞𝐭 Novartis Biogen GILEAD SCIENCES LTD Sarepta Therapeutics Amgen Neurophth Therapeutics / 纽福斯生物科技有限公司 As we navigate the dynamic landscape of gene therapy, it is imperative to address challenges such as scalability, affordability, and long-term safety and efficacy concerns. Collaborative efforts involving stakeholders across academia, industry, and regulatory bodies are essential to overcome these hurdles and realize the full potential of gene therapy in transforming healthcare. In conclusion, the exponential growth trajectory of the Global Gene Therapy Market heralds a new era of hope and possibilities in healthcare. With relentless innovation, strategic collaborations, and a steadfast commitment to patient-centric care, we can harness the power of gene therapy to conquer diseases, alleviate human suffering, and pave the way for a healthier future. #GeneTherapy #HealthcareInnovation #PrecisionMedicine #Biotechnology #CRISPR #FutureOfMedicine #HealthcareTransformation

Can genomic medicine create a trillion-dollar market opportunity?   Ø Since the approval of LUXTURNA for treating blindness due to Leber's congenital amaurosis (LCA) in 2017, the pace of development and approvals has accelerated quickly.   Ø With FDA’s broader definition of gene therapy, over twenty gene/cell-based gene therapies have been approved for cancer, viral infections, and inherited disorders, and over two thousand ongoing gene therapy clinical trials worldwide.   Ø Neuromuscular disorders represent a strong unmet medical need. Gene therapy holds great promise as a potential treatment for neuromuscular disease patients by delivering a functional gene to skeletal and cardiac muscles. However, several challenges need to be overcome.   Ø Gene therapy is an ideal approach for monogenic diseases, since restored gene expression and protein production in theory could potentially correct disease phenotype due to the loss of gene expression in a single gene.   Ø Continued enthusiasm by investors toward gene therapy is fuelled by strong unmet needs and the potential to bring significant benefits to a patient’s life by repairing a single gene. #genetherapy #genomeediting #genomicmedicine #raredisease #raredisorders #orphandrugs #geneticdisease #management #consulting #pharmaceuticals #marketintelligence #competitiveintelligence #marketresearch #marketing #leadership #eosintelligence #perspectives #lifesciences #healthcare ---------------------------------------------------------------------------------- Ready to leverage the power of intelligence to drive "Genomic Medicine" product development and commercialization strategy? At EOS Intelligence, we offer competitive intelligence monitoring services in key therapeutic areas to make you stay ahead of the curve. Let us know your perspectives & challenges and we will get you covered. Write us at bhabesh.panigrahi@eos-intelligence.com