Hartmann Young’s Post

TLDR Biotech for June 18 - 20 𝐀𝐩𝐩𝐫𝐨𝐯𝐚𝐥𝐬 & 𝐋𝐚𝐛𝐞𝐥𝐬: Sarepta’s DMD gene therapy leaves Pfizer in the dust, lands key FDA label expansion 🧬 + 5 more stories 𝐁𝐮𝐬𝐢𝐧𝐞𝐬𝐬 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭: Biophytis and Blanver sign €108M licensing deal 💼 + 2 more stories 𝐂𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐓𝐫𝐢𝐚𝐥𝐬: Gilead's twice-yearly HIV prevention med scores 100% efficacy in Ph3 🧪 + 6 more stories 𝐂𝐨𝐦𝐩𝐚𝐧𝐲 𝐋𝐚𝐮𝐧𝐜𝐡𝐞𝐬: Yellowstone Biosciences erupts out of stealth with £16.5M 🚀 𝐅𝐮𝐧𝐝𝐫𝐚𝐢𝐬𝐞𝐬: iOnctura raises $86M Series B 💰 + 4 more stories 𝐈𝐧𝐯𝐞𝐬𝐭𝐦𝐞𝐧𝐭𝐬: Takeda plans $230M plasma-derived facility expansion 🏗️ + 1 more story 𝐌𝐞𝐫𝐠𝐞𝐫𝐬 & 𝐀𝐜𝐪𝐮𝐢𝐬𝐢𝐭𝐢𝐨𝐧𝐬: Bora Therapeutics acquires sterile manufacturing site from Emergent BioSolutions 🏭 + 2 more stories 𝐏𝐚𝐫𝐭𝐧𝐞𝐫𝐬𝐡𝐢𝐩𝐬: Samsung Biologics signs $223M+ manufacturing partnership with Baxter 🤝 + 1 more story 𝐑𝐞𝐬𝐞𝐚𝐫𝐜𝐡: AI-powered blood test could spot Parkinson's disease years in advance 🧠 + 2 more stories All this and more in today's TLDR Biotech newsletter - link to the latest issue: https://lnkd.in/gJQ4eFNb Or subscribe to get it straight to your inbox: https://lnkd.in/gh68uhTQ #tldrbiotech #biotechnews #pharmanews

Senator Richard Burr's OpEd in Stat provides some insightful perspective and highlights the crucial role the FDA plays in the future of these revolutionary treatments. While he is a proponent of the FDA's mission, he remans a strong critic of the time it takes for the agency to review cutting edge therapies. 🚀 Accelerated Approval Pathway: The FDA's adoption of an accelerated approval pathway, similar to what has been successful in cancer and HIV/AIDS treatments, could be a game-changer. It provides a balance between rapid access to novel treatments and maintaining safety and efficacy standards. However... 🔍 FDA’s Pivotal Role in Innovation: Despite the progress, the FDA's cautious approach may slow down the pace of innovation in genetic medicine. With over 2,500 investigational new drug applications filed, the agency has approved just a handful of therapies this year. This calls for a more adaptive and collaborative regulatory approach, especially for rare diseases with significant unmet needs. 🌐 A Call for Modernization: The biotech sector faces a critical juncture – the need for regulatory frameworks that keep pace with scientific advancements. It's time for regulatory bodies to align with the rapid advancements in cell and gene therapies, ensuring patients receive timely access to these life-altering treatments. #CellAndGeneTherapy #FDA #Biotech #Innovation #Healthcare

PRESS RELEASE - For Immediate Release Galway, November 10, 2023 Afortiori Development and HiTech Health Forge an Important Collaboration to Support the Clinical Research and Product Development of ground breaking cell and gene therapies. We are delighted to announce this collaboration with HiTech Health. Brian and his team provide vital services when it comes to the development, manufacture and supply of multiple product types including cell and gene therapies for clinical trials and also the supply of approved medicines to patients. Read more below and we will post further details (press release available here also) in future posts and on our website.   Afortiori Development and HiTech Health invite clients and industry professionals to get in touch to discuss their current and future needs. Engaging with their dedicated teams of experts will unlock new possibilities and propel clients towards success on this exhilarating path. #cmc #biomanufacturing #celltherapy #cellandgenetherapy #celltherapymanufacturing #clinicaltrials #clinicalresearch HiTech Health Brian Harrison Aoife Duffy Elizabeth Ralph Afortiori Development and HiTech Health Forge an Important Collaboration to Support the Clinical Research and Product Development of ground breaking cell and gene therapies.   Afortiori Development, a premier full-service Clinical Research Organisation, is delighted to announce its latest partnership with HiTech Health, a leading Contract Development and Manufacturing Organisation (CDMO) for Advanced Therapy Medicinal Products (ATMP) including Cell and Gene Therapies (CGT) (Europe/UK/Ireland/US). This strategic alliance aims to provide an integrated suite of services to companies conducting research in the ATMP and CGT areas.  The two companies believe that by seamlessly integrating clinical trial expertise with cutting-edge product development and manufacturing capabilities, this will provide the calibre of service required by companies conducting research, ensuring that their clinical research journey is enhanced overall.   Afortiori Development and HiTech Health represent a combined team of seasoned experts who work closely with clients, offering flexible, scalable, and tailored solutions to each trial’s specific needs. Throughout the entire process, as industry leaders we remain dedicated advocates for our clients, ensuring they benefit from the optimal clinical trial delivery services that generate actionable insights for safety, efficacy and regulatory compliance. Contact us on info@aftdev.com for more information.  

With the recent approvals of expensive gene/cell therapies for Sickle Cell Disease, Jason Mast asks what is the status of small molecule drug discovery around this disease area? Existing oral meds exist for SCD, but does not effectively treat the disease for a multitude of reasons. Will new advances in medicinal chemistry and molecular glues hold the answer to creating effective SCD drugs for the wider population afflicted with this disease? One popular method is to find drugs that upregulate naturally occuring fetal hemoglobin within cells to displace the dominance of the adult hemoglobin that carries the mutation. As this is still preclinical work, there are still long term studies to perform, and clinical trials to overcome. From STAT: " When Novartis screened a library of molecular glues, some increased fetal hemoglobin without neutering the cells, including one that grabbed onto a protein called WIZ....Novartis chemists then designed a better version of the molecule, called d-WIZ2. When they gave it to special lab mice endowed with a human blood system, it led to around 15% to 20% fetal hemoglobin expression." Disclaimer - Views expressed here are of the Author only. https://lnkd.in/dmAy-tRF

Thoughts on this? >> NANITE AWARDED $1.8M TO DEVELOP AI-DRIVEN GENE THERAPY FOR HIV - PR Newswire >> Comment below! >>> lqventures.com #strategy #competitiveintelligence #marketing #healthcare #biotech #competitivemarketing #pharma #pharmaceutical

💊💡Meet AbBind, a pioneering antibody-compatible, virus-like particle platform that holds transformative potential in vaccinology and tissue-specific gene therapy. Developed by our world-class researchers, AbBind has effectively addressed previous limitations in VLP self-assembly, broadening the range of antigens that can be displayed. What sets AbBind apart? Its unique plug-and-play design and full compatibility with established antibody manufacturing technologies. Furthermore, in vivo data supports its ability to induce both humoral and cell-mediated immune responses and target antigens or payloads to specific cell types or tissues. With this groundbreaking technology, we are furthering our mission of creating positive social, economic, and environmental impacts, particularly in the realm of health. 💪🔬 We are seeking partnerships with experts in vaccinology or tissue-targeted drug delivery for potential licensing, co-development, or spinout investment opportunities, if this could be something of interest, please get in touch with Elliot Jennings. Discover more about this innovative platform 👉 https://bit.ly/3OfPbYC #innovation #research #mcruk #health #healthandwellness #science #vaccines #biotechnology #pharma #UoM

𝐍𝐒𝐖’𝐬 𝐕𝐢𝐫𝐚𝐥 𝐕𝐞𝐜𝐭𝐨𝐫 𝐌𝐚𝐧𝐮𝐟𝐚𝐜𝐭𝐮𝐫𝐢𝐧𝐠 𝐅𝐚𝐜𝐢𝐥𝐢𝐭𝐲, 𝐀𝐮𝐬𝐭𝐫𝐚𝐥𝐢𝐚 🔬 The New South Wales Government, the governing body of NSW, Australia, is expanding a state-of-the-art good manufacturing practice (GMP)-grade viral vector manufacturing facility within the Westmead Health and Innovation District in western Sydney. The NSW Government established a new company to operate the viral vector manufacturing facility based at Westmead in February 2024. The facility manufactures viral vector products for research and clinical trials, supporting the current absence of GMP-licensed viral vector manufacturers in Australia. It will meet the increasing demand for clinical-grade viral vectors essential for gene therapy and cell therapy research, as well as clinical trials both domestically and internationally. The NSW Government has invested approximately A$134.5m ($88.2m) to establish the facility, which is being constructed in stages. Stage 1 of the facility is operational for gene therapy research, with the first clinical trial expected to begin in 2025 using vectors produced on site. Stage 2 of the facility is currently under construction and scheduled to be operational by the end of 2024. The facility will provide NSW with the capability to effectively address the increasing need for viral vectors. The facility is a collaborative effort involving the NSW Treasury, Investment NSW, and various NSW Health entities, including Health Infrastructure, Office of Health and Medical Research, and Sydney Children’s Hospitals Network, with support from the Children’s Medical Research Institute and Western Sydney Local Health District. #NewSouthWales #Australia #ClinicalTrials #Clinicalresearch

In several polls out over last couple of weeks, personalised medicine and Cell and Gene Therapy (#CGT) have scored highest as the most promising medicinal developments. It's a fantastic area of development generating treatments to so far untreatable conditions as well as new rapid response #vaccines. The pandemic of COVID-19 and the outbreak of MPox have shown how much we need a rapid vaccine rollout to new challenges. Yet did you know, that we still grow the annual flu vaccines in chicken eggs? That's a lot of eggs! We could make them faster and cheaper using recombinant methods. All in all it's great to see the new business network in Scotland launched by the Cell and Gene Therapy Catapult and Scottish Enterprise to boost the business engagement in Scotland for C&GT. It will be great to see this sector grow! https://lnkd.in/edDmYC8Q

mRNA in 1962... What occurred in 1962 in the mRNA field, you ask? Nothing (that I'm aware of)! But you can chat all things mRNA with DeciBio in Booth 1962 at #ASGCT24 next week in Baltimore! 😆 The field has a rich pipeline of early-stage assets in development. Gone are the days of "your body as a drug factory". Its all about low(er) dose gene therapy / editing applications using Crisper, prime, or base editing. Vaccines are obviously still a significant portion of the field, but I suspect we'll see that shift from infectious disease to oncology / personalized cancer vaccine programs, where the case for value could be more clear (assuming the science continues to mature). Too many investment and partnership dollars flowing into the space for that not to be the case. Come say hi next week in Booth 1962! #genetherapy #pharma #drugdiscovery

𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝘆 𝗠𝗮𝗿𝗸𝗲𝘁 𝟮𝟬𝟮𝟯-𝟮𝟬𝟯𝟬. 𝗚𝗹𝗼𝗯𝗮𝗹 𝗥𝗲𝘀𝗲𝗮𝗿𝗰𝗵 𝗥𝗲𝗽𝗼𝗿𝘁 Gene therapy is an evolving experimental treatment method aimed at combating or preventing various diseases, including severe combined immunodeficiencies, hemophilia, Parkinson's disease, cancer, and even HIV. This innovative approach involves introducing genetic material into a patient's cells, typically employing modified viruses called vectors for delivery. While efforts are made to enhance the safety of viral vectors, some inherent risks persist in gene therapy. The field is still in its early stages of development. This comprehensive analysis report delves into the global Gene Therapy market, offering both quantitative and qualitative insights categorized by company, region, type, and application. It examines the ever-changing market dynamics, competitive landscape, supply-demand trends, and key drivers influencing market demands across diverse sectors. Additionally, the report provides company profiles, product examples from select competitors, and market share estimates for leading players in 2023. 𝗧𝗼 𝗞𝗻𝗼𝘄 𝗚𝗹𝗼𝗯𝗮𝗹 𝗦𝗶𝘇𝗲 𝗮𝗻𝗱 𝗗𝗲𝗺𝗮𝗻𝗱 𝗼𝗳 𝗚𝗲𝗻𝗲 𝗧𝗵𝗲𝗿𝗮𝗽𝘆. 𝗥𝗲𝗾𝘂𝗲𝘀𝘁 𝗳𝗼𝗿 𝗦𝗮𝗺𝗽𝗹𝗲 𝗣𝗗𝗙: https://lnkd.in/dGxkydAB *𝗕𝘆 𝗧𝘆𝗽𝗲: Viral Gene Therapy, Non-Viral Gene Therapy *𝗕𝘆 𝗔𝗽𝗽𝗹𝗶𝗰𝗮𝘁𝗶𝗼𝗻: Neurological Diseases, Cancer, Other *𝗕𝘆 𝗥𝗲𝗴𝗶𝗼𝗻: North America, Europe, Asia-Pacific, South America, Middle East & Africa *𝗕𝘆 𝗞𝗲𝘆 𝗣𝗹𝗮𝘆𝗲𝗿𝘀: Biogen, Novartis, Gilead Sciences, Sarepta Therapeutics, Alnylam Pharmaceuticals, Amgen, Spark Therapeutics, Inc., AKCEA THERAPEUTICS UK LTD., bluebird bio, Sunway Biotech, Orchard Therapeutics, Human Stem Cells Institute #genetherapy #therapyservices #therapy #healthcaretherapy #healthcare #medical #viralgenetherapy #nonviralgenetherapy #neurologicaldiseases #cancer #marketdemand #globalresearch #marketsize #marketshare #gene