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Successful drug development requires a deep understanding of the disease of interest. In rare diseases, however, much of the information may be
unknown. Patient populations are small and historical data are collected
inconsistently. Patient registries and natural history studies are valuable sources of rare disease information for sponsors seeking to design reliable clinical trials - read our blog to explore their distinctions.
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Today's Big NewsFeb 3, 2023 |
| By Max Bayer,Fraiser Kansteiner Johnson and Johnson's pharmaceutical unit Janssen is undergoing a seismic overhaul, folding its infectious disease team with the vaccines unit and discontinuing numerous programs in the process. As a result, the company expects to lay off employees in several countries. |
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By Nick Paul Taylor The fight for the myasthenia gravis market has become too hot for Sanofi to handle. After reviewing the competitive landscape, the Big Pharma has decided to stop a phase 3 clinical trial of a drug candidate it acquired in its $3.7 billion takeover of Principia Biopharma. |
By Annalee Armstrong A week after pausing a leukemia trial due to a patient death, Magenta Therapeutics is halting development of all programs and exploring a potential sale or other measures to wind down operations. |
By Annalee Armstrong Small drug discovery biotech Vyant Bio is taking steps to wind down operations, including reducing its workforce, pursuing strategic alternatives and saying goodbye to CEO John Roberts. |
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April 3 - 4, 2023 | San Diego, CA Connect with leading communications professionals and have honest, open discussions about how to develop your career in the industry. Early Bird Rate Ends February 10th.
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By Gabrielle Masson With so many Big Pharmas reporting their full-year earnings yesterday, it was no surprise that drugmakers used the occasion as an excuse to slip out some pipeline changes. Gilead was no exception, removing a solid tumor candidate from its early-stage lineup along with two of the three indications for its IRAK4 inhibitor. |
By Nick Paul Taylor MaaT Pharma’s 18-month struggle to get out from under an FDA clinical hold may be entering its endgame. In its latest feedback, the agency reportedly agreed to a list of conditions that could enable clinical evaluation of its pooled fecal therapy MaaT013 in the U.S. |
By Helen Floersh Scientists have integrated human brain organoids into the brains of rats with injuries to their visual cortices and gotten the cells to respond to stimulation. The findings are a step toward using stem-cell-derived brain tissue to restore some functions of sight. |
By Fraiser Kansteiner Eisai is laying off 91 employees effective April 30, according to a Worker Adjustment and Retraining Notification Act (WARN) alert from New Jersey. The public disclosure lists the location of the layoffs as Nutley, New Jersey, where Eisai recently relocated its U.S. headquarters. |
By Conor Hale It’s been a year since the White House revived the Cancer Moonshot program, one of President Joe Biden’s signature initiatives during his time as vice president in the Obama administration. Now the ambitious effort has announced a new series of actions to help commemorate its anniversary. |
By Robert King CMS outlined the initial steps it will take to create quality measures aligned across all of the agency's programs, with the first measures released. |
By Teresa Carey This week on "The Top Line," we discuss what we can expect from the obesity market in 2023. We also recap the drugs approved last year. |
By Angus Liu Johnson & Johnson and Legend Biotech's Carvykti hit its phase 3 goal in earlier multiple myeloma. Sanofi is cutting hundreds of jobs at two facilities in India. The FDA has yet to conduct long-delayed pre-approval inspections in China for Novartis and BeiGene's PD-1 inhibitor tislelizumab. And more. |
By Gabrielle Masson,Annalee Armstrong Ginkgo BioWorks CEO and co-founder Jason Kelly, Ph.D., has been selected to lead a 12-member group assessing emerging biotech innovation for the U.S. Department of Defense. |
Fierce podcastsDon't miss an episode |
| | This week on "The Top Line," we discuss what we can expect from the obesity market in 2023. We also recap the drugs approved last year. |
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May 1 - 3, 2023 | Jersey City, NJ Connect on current challenges and opportunities found within your medical affairs role to become better equipped in developing a unified scientific voice. Take advantage of the Early Bird Rates, Register Today!
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