#Didyouknow On May 29, 2024, the FDA notified selected participants of their acceptance into the Support for #clinicaltrials Advancing #Rarediseases Therapeutics #START Pilot Program. Read more: https://lnkd.in/eFNrXnCV 𝐁𝐚𝐜𝐤𝐠𝐫𝐨𝐮𝐧𝐝 The FDA launched the START program in Sep 2023 to [...] 𝘩𝘦𝘭𝘱 𝘧𝘶𝘳𝘵𝘩𝘦𝘳 𝘢𝘤𝘤𝘦𝘭𝘦𝘳𝘢𝘵𝘦 𝘵𝘩𝘦 𝘥𝘦𝘷𝘦𝘭𝘰𝘱𝘮𝘦𝘯𝘵 𝘰𝘧 𝘯𝘰𝘷𝘦𝘭 𝘥𝘳𝘶𝘨 𝘢𝘯𝘥 𝘣𝘪𝘰𝘭𝘰𝘨𝘪𝘤𝘢𝘭 𝘱𝘳𝘰𝘥𝘶𝘤𝘵𝘴 𝘧𝘰𝘳 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵 𝘰𝘧 𝘳𝘢𝘳𝘦 𝘥𝘪𝘴𝘦𝘢𝘴𝘦𝘴.[...] The participants of the program will benefit from increased communication with FDA officials to receive timely advice for addressing their #clinicaldevelopment program-specific needs, including #clinicalstudydesign, choice of control group, fine-tuning the choice of #patientpopulation, leveraging nonclinical information, and product characterization. The eligibility criteria are the following: 👉 𝐉𝐨𝐢𝐧𝐭 𝐂𝐁𝐄𝐑 𝐚𝐧𝐝 𝐂𝐃𝐄𝐑 𝐄𝐥𝐢𝐠𝐢𝐛𝐢𝐥𝐢𝐭𝐲 𝐂𝐫𝐢𝐭𝐞𝐫𝐢𝐚 The program is open to sponsors 📌of products currently in #clinicaltrials under an active investigational new drug application #IND submitted in or converted to Electronic #CommonTechnicalDocument #eCTD format 📌who have demonstrated substantial effort to ensure that Chemistry, Manufacturing, and Controls #CMC development aligns with clinical development. 👉 𝐂𝐁𝐄𝐑-𝐒𝐩𝐞𝐜𝐢𝐟𝐢𝐜 𝐄𝐥𝐢𝐠𝐢𝐛𝐢𝐥𝐢𝐭𝐲 𝐂𝐫𝐢𝐭𝐞𝐫𝐢𝐚: Eligible products must be a gene or #cellulartherapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first decade of life. 👉 𝐂𝐃𝐄𝐑-𝐒𝐩𝐞𝐜𝐢𝐟𝐢𝐜 𝐄𝐥𝐢𝐠𝐢𝐛𝐢𝐥𝐢𝐭𝐲 𝐂𝐫𝐢𝐭𝐞𝐫𝐢𝐚: Products must be intended to treat rare #neurodegenerative conditions, including those of rare genetic #metabolic etiology. #pharma #pharmaindustry #biotech #fdacompliance #regulatoryaffairs
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⚠️ WEBINAR ALERT⚠️ 🧬 GlobalData Plc Presents: Cell & Gene Therapy Intended Audience: VPs Commercial Strategy Market Access Pricing Competitive Intelligence Brand Managers Manufacturing Supply chain Sectors Covered: Service providers (CMOs, CDMOs, CROs) Biotechs Drug manufacturers and developers Clinical trial operators Analytics providers MoHs Market access consultancies Financial capital markets Delivery suppliers WHEN: UK - Thursday 9th Nov 4pm Korea - Friday 10th Nov 1am India - Thursday 9th Nov 9:30pm *If these times are not suitable for you, please still register as you can watch the playback at any time post the event finishing. This virtual conference will provide an all-encompassing view of regenerative medicine from leaders in GlobalData’s pharmaceutical team, alongside an exclusive Q&A session. Key industry challenges across the pharmaceutical value chain will be addressed, from drug development and clinical trials, to manufacturing and supply chain management. Split into three insight sections on therapeutic areas, contract service providers, and pricing and market access, and followed by a Q&A, this event is not to be missed. Register Here: https://lnkd.in/gEGmE5u8 Looking forward to hearing the insightful speakers: Jasminemay Barcelon Barbora Salcman Amanda Murphy Fiona Barry Milena Izmirlieva #pharmanews, #drugdevelopment #pharmaceuticals #celltherapy #cellandgenetherapy #cellandgene #cellandgenetherapies
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The week in pharma: action, reaction and insight – week to December 15, 2023 - The Pharma Letter >>> lqventures.com #strategy #competitiveintelligence #marketing #biotech #pharma #healthcare #competitivemarketing #pharmaceutical
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How can we avoid clinical stage failures and get drugs to patients faster? We share our thoughts on navigating R&D with evidence from large human populations in this latest Cell Press Trends in Biotechnology piece https://lnkd.in/e7q52qWP Reducing clinical failures is the biggest opportunity in drug development: 90% of drugs fail in clinical trials, with 50% failing for efficacy reasons. Stakeholders lack critical evidence on whether their drug target will work in humans before trialing for it, and they lack tools to use insights on target biology to optimize clinical positioning. Human evidence from large populations offers critical insights to navigate and de-risk R&D: ensure target safety and efficacy at the earliest stages and align target biology with the right clinical phenotypes benefitting from target modulation. Interested in learning more about how PheironGPS can benefit your program? We’d love to know https://lnkd.in/ey_B2gHj #clinicalsuccess #drugdevelopment #targetidentification #targetvalidation #humanevidence #geneticevidence #targets #biomarkers #populations
The GPS for drug development: navigating with evidence from human populations
cell.com
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Fellow professionals and leaders in #pharmaceuticals and #biopharma: We have a responsibility to pay attention to – and be part of – rapid whole genome sequencing (#rWGS) advancement. If you haven’t yet, make sure to give our FiercePharma article a read. We explore the great potential of rWGS in drug development and commercialization, spotlighting the cross-industry initiatives leading the charge. When we share expertise and resources, our impact on the lives of all newborns can be that much greater. Share this with someone you know to support the advancement of #RareDisease diagnosis and treatment. https://lnkd.in/g7xgMmb4 #SentynlSeeks #NewbornSequencing
Why Pharma Should Pay Attention to – and Be Part of – Rapid Whole Genome Sequencing Advancement
fiercepharma.com
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Exec Search Pro helping biotech, value-based care, digital health companies & hospitals hire transformational C-suite & Board leaders. Partner, Life Sciences, Healthcare, Diversity, Board Search | Board Member | Investor
The U.S. Food and Drug Center for Drug Evaluation and Research (CDER) 𝐚𝐩𝐩𝐫𝐨𝐯𝐞𝐝 𝟓𝟓 𝐧𝐨𝐯𝐞𝐥 𝐝𝐫𝐮𝐠𝐬 𝐢𝐧 𝟐𝟎𝟐𝟑, according to their annual New Drug Therapy Approvals report, 𝑛𝑜𝑡 𝑖𝑛𝑐𝑙𝑢𝑑𝑖𝑛𝑔 vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or other products that the Center for Biologics Evaluation and Research approved in 2023. This was ✅ 50% more than in 2022, ✅ the highest since 2018 and ✅ the second highest number of approvals in 30 years. So far, in the first 10 days of 2024, one therapy has been approved and there are several other novel therapies in the FDA’s pipeline for approval this year. Based on this information, ➕ the excitement coming out of #jpm2024 ➕ the Fed's cautious optimism about inflation and the likelihood of not having to raise rates again ➕ record high levels of employment ...I'd say that 2024 promises to be a good year for executive-level employment in life sciences and healthcare. The competition for top talent will be tough, but companies with solid talent strategies and strong corporate partners (hint, wink) will win. 🏆 There you go. You asked, I answered. Sincerely, Your Biotech Bestie Your Healthcare Homegirl Your Board Boo #biotech #healthcare #hospitals #executivesearch https://lnkd.in/eq-wTBM3 g͟r͟a͟p͟h͟i͟c͟s͟: NME = New Molecular Entities BLA = Biologics License Applications https://lnkd.in/eFeeCNmK.
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BioPharma & HealthTech Competitive Strategy & Insights | Digital & AI Solutions | Gene & Cell Therapy | Vaccines
The week in pharma: action, reaction and insight – week to December 15, 2023 - The Pharma Letter >>> lqventures.com #strategy #competitiveintelligence #marketing #pharma #pharmaceutical #biotech #healthcare #competitivemarketing
Google News
thepharmaletter.com
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"Healx pitches itself as a “drug discovery pipeline” that identifies hidden connections between existing chemical compounds (e.g., drugs and pharmaceuticals at various stages of the development cycle) and rare diseases. It does this by collating a myriad of public and proprietary data sources, like biomedical literature, disease and biochemical datasets, clinical trials and patents to create a “biomedical knowledge graph” of rare diseases." - https://lnkd.in/gxwgRNXC - #raredisease
Healx, an AI-enabled drug discovery platform for rare diseases, raises $47M | TechCrunch
https://techcrunch.com
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50% of clinical failures are attributed to a lack of efficacy. Reasons for this often go back to the earliest phases of R&D: decisions on the target and the clinical positioning do not only set the foundations for development but also largely determine success rates. Today, stakeholders lack tools to systematically assess target efficacy and safety with human evidence. Our mission at Pheiron is to change that. We use #AI and #populationgenetics to find routes through early R&D that have a higher likelihood of clinical success: de-risked targets, linked biomarkers and aligned clinical phenotypes. Read more about what we’re building here: https://lnkd.in/e6kubYvv #phenotyping #geneticevidence #AI #populationgenetics #predictiveutility
How can we avoid clinical stage failures and get drugs to patients faster? We share our thoughts on navigating R&D with evidence from large human populations in this latest Cell Press Trends in Biotechnology piece https://lnkd.in/e7q52qWP Reducing clinical failures is the biggest opportunity in drug development: 90% of drugs fail in clinical trials, with 50% failing for efficacy reasons. Stakeholders lack critical evidence on whether their drug target will work in humans before trialing for it, and they lack tools to use insights on target biology to optimize clinical positioning. Human evidence from large populations offers critical insights to navigate and de-risk R&D: ensure target safety and efficacy at the earliest stages and align target biology with the right clinical phenotypes benefitting from target modulation. Interested in learning more about how PheironGPS can benefit your program? We’d love to know https://lnkd.in/ey_B2gHj #clinicalsuccess #drugdevelopment #targetidentification #targetvalidation #humanevidence #geneticevidence #targets #biomarkers #populations
The GPS for drug development: navigating with evidence from human populations
cell.com
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Regulatory Professional @ Noven | Ex-Novartis (Regulatory/Quality) | MS in Regulatory Affairs (Clinical) & Quality Assurance for Drugs, Biologics & Medical Devices | SaMD |SAS| Pharmacovigilance
Excited to share insights from the online presentation attended: "Planning for a Successful Initial IND and Moving Beyond: Regulatory CMC Considerations for Biotherapeutics and Cell and Gene Therapies," hosted by Regulatory Affairs Professionals Society (RAPS) and Veristat. Interested in delving into comparability studies and assessments within therapeutic development or clinical research, this online presentation provided a comprehensive picture of comparability studies in a nutshell. One key takeaway was the importance of comparability studies in pharmaceutical manufacturing. These studies assess whether changes to the manufacturing process or product impact quality, safety, or efficacy. Evaluating Critical Quality Attributes (CQAs) is central to this process, ensuring consistency and performance of the investigational product. I learned about the analytical techniques and strategies for comparability studies, emphasizing their vital role in product development. From physicochemical properties to potency and stability, every aspect is meticulously evaluated to maintain product integrity. Looking forward to getting insights from all of you and applying these in my regulatory affairs work! #RegulatoryAffairs #Pharmaceuticals #CMC #Biotherapeutics #ClinicalResearch #TherapeuticDevelopment #ComparabilityStudies #ContinuousLearning #RAPS #Veristat #OnlinePresentation #northeasternuniversity #raps #certification
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Unlike traditional drugs, advanced therapeutic modalities (ATMs) are designed rather than discovered. They’re also about to become a standard modality. Writing for Harvard Business Review, L.E.K.’s Alex Vadas, Jeff Holder, Ph.D. and Adam Siebert explain how ATMs are different and what that means for life sciences companies as they confront profound changes in strategy, investment and risk management. https://bit.ly/48EK5NW #lifesciences #pharma #pharmaceuticals #pharmaceuticalindustry
A New Generation of Drug Therapies Requires New Business Strategies
hbr.org
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