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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. +(002)+120w.png)
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Today’s Big NewsApr 22, 2024 |
| By James Waldron Boehringer Ingelheim is making yet another bet that RNA therapies hold the key to treating metabolic-associated steatohepatitis. |
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By Nick Paul Taylor Ipsen has swooped on a $1.8 billion R&D opportunity, joining with Skyhawk Therapeutics for the chance to option two candidates that could benefit from its “neuroscience expertise in movement disorders.” |
By Gabrielle Masson,Annalee Armstrong,Max Bayer Welcome to Fierce Biotech's Fundraising Tracker, 2024's version. |
Sponsored by NSF NSF TraQtion software combines decades of quality expertise with real-time digital insight in one trusted, cloud-based digital solution. |
By Gabrielle Masson,Max Bayer We really didn't want to have to create another Layoff Tracker this year. But here we are, launching the third annual iteration tracking biopharma layoffs. |
By Angus Liu As an all-important readout from Alnylam’s Amvuttra in the rare heart disease ATTR-CM is expected in a few weeks, experts suggested that the RNA silencer may have a hard time challenging Pfizer’s tafamidis franchise despite a last-minute trial design change. |
By Kevin Dunleavy Eight months after participating in a $255 million funding round in cell therapy contract manufacturer Cellares, Bristol Myers Squibb has expanded their partnership by signing a $380 million deal to reserve CAR-T manufacturing space. |
By Nick Paul Taylor Immunology is AbbVie’s world, everyone else just lives in it. That is the takeaway from ZoomRx’s analysis of the perceptions of healthcare professionals , who overwhelmingly see AbbVie as the top dog in a field targeted by rivals such as Pfizer and Johnson & Johnson. |
Fierce podcasts Don’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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eBook To 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 |
Whitepaper Discover the secrets behind successful patient engagement Sponsored by: ProofPilot |
Whitepaper Antibody biomarkers are redefining precision medicine, from diagnosing diseases earlier to stratifying patients – download your FREE white paper today! Sponsored by: Sengenics Corporation LLC |
Whitepaper The 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 |
eBook Unlock 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 |
Whitepaper Choosing 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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