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Rare disease development is complex, characterized by distinctive challenges due to small patient populations spread across many geographies, limited disease data, and narrow site pools. For sponsors, successful studies involve striking a balance among cost, quality, and innovative approaches that improve trial efficiency and patient engagement. Read our checklist to explore 9 best practices for optimizing the efficiency and quality of rare disease clinical trials.
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Today’s Big NewsApr 24, 2024 |
| By Annalee Armstrong To be a better neuroscience company, Biogen is going to diversify—outside of the therapeutic area that has in recent years defined the company. |
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By Nick Paul Taylor Roche has performed a little light spring cleaning, sweeping a pair of phase 1 solid tumor drugs and an early-stage psychiatric disorder prospect out of the door as part of its update for the first quarter. The culls are part of a broader refocusing that has removed 20% of new molecules from the pipeline in recent quarters. |
By Gabrielle Masson What does it feel like to be a woman CEO in biotech? An island, according to former Gossamer Bio CEO and co-founder Sheila Gujrathi, M.D. |
Sponsored by NSF NSF TraQtion software combines decades of quality expertise with real-time digital insight in one trusted, cloud-based digital solution. |
By Annalee Armstrong If you asked an AI to generate a brand-new drug discovery biotech with a team of some of the biggest names in the biz for you, it couldn’t dream up one like Xaira Therapeutics. The company emerged Tuesday with $1 billion in committed funding. |
By Annalee Armstrong Vertex’s cystic fibrosis triple combo is poised to become the top selling orphan drug in terms of net present value, amid shifting sentiments in the pharma industry for rare disease drugs, according to Evaluate. |
By Annalee Armstrong After Alpine Immune Sciences presented initial data for the kidney disease therapy povetacicept at a conference in November 2023, the biotech's confidential data room became the most exclusive club in biotech. |
By Max Bayer Endeavor has bagged another nine-digit financing to advance its IPF drug into a second phase 2 study. The biotech also plans to start a clinical trial for its HER-3 ADC. |
By Conor Hale Profluent has staked a claim at the intersection of some of the buzziest terms in biopharma. The protein-focused AI startup believes its large language models can help extend the reach of CRISPR gene editing tools. |
By Angus Liu Giovanni Caforio, M.D., is lining up a new gig soon after handing all his Bristol Myers Squibb leadership roles over in March. The recent Big Pharma CEO, known for steering the $74 billion acquisition of Celgene, has been proposed as the new chair at Novartis for 2025. |
By Ayla Ellison,Anastassia Gliadkovskaya This week on "Podnosis," we take a closer look at how a mobile clinic in Detroit is using free CT scans to learn more about long COVID. |
Fierce podcastsDon’t miss an episode |
| | This week on "The Top Line," we are joined by Annalee Armstrong, Senior Editor at Fierce Biotech, and Ben Adams, Senior Editor of Fierce Pharma Marketing, to discuss Fierce's take on March Madness. |
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| eBookTo make every rare disease trial count, we design patient-centric, relevant strategies for sponsors and caregivers to produce impactful results every time. Sponsored by: Emmes Company |
| WhitepaperDiscover the secrets behind successful patient engagement Sponsored by: ProofPilot |
| WhitepaperAntibody biomarkers are redefining precision medicine, from diagnosing diseases earlier to stratifying patients – download your FREE white paper today! Sponsored by: Sengenics Corporation LLC |
| WhitepaperThe use of prefilled syringes to administer biotherapeutics is beginning to gain traction because they offer a range of stability, efficacy, and patient safety benefits. Download this white paper to learn how Lonza has developed a novel workflow to ensure confidence regarding functionality in PFS and autoinjectors. Presented by Lonza |
| eBookUnlock the future of monoclonal antibody (mAb) manufacturing with a complete guide to enhancing productivity and quality for exceptional outcomes. Sponsored by: Thermo Fisher Scientific |
| Whitepaper This paper assesses the current state of the oncology market, reviewing key expectations for 2024. It addresses CAR-T therapies, KRAS, radiopharmaceuticals, & targeted protein degradation. Presented by Blue Matter, strategic consultants in the life sciences |
| WhitepaperChoosing the right contract lab can help drug developers accelerate their timelines and generate cost-efficiencies. For insights into what to look for when selecting a GMP/CMC lab partner read this white paper which delves into a range of factors that can support your needs in both the short-term and in planning for the future. Sponsored by: PPD®️ Laboratory Services |
On Helix Date: 4 July 2024 - Location: Babraham Research Campus, Cambridge UK |
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