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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. .png)
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Today's Big NewsJan 31, 2023 |
| By Nick Paul Taylor Pfizer, amid a rethink of its rare disease R&D strategy, has punted a pair of orphan drug candidates from its clinical development pipeline in a cull that also narrowed the opportunities open to the near-approval JAK3/TEC inhibitor ritlecitinib. |
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By James Waldron You might have assumed that given the $2 billion launch of Altos Labs—and all the buzz the startup garnered by poaching GSK veteran Hal Barron to become CEO—that funding in longevity research would be sky high in 2022. But a new report suggests otherwise. |
By Gabrielle Masson Quince Therapeutics is laying off nearly half of its staff and looking to out-license a preclinical asset almost immediately after selling a portion of its pipeline to Lighthouse Pharmaceuticals. |
By James Waldron Six months after laying off 18% of its workforce, Inovio is sending out more pink slips. This round of redundancies will see 11% of remaining employees heading for the exits as the biotech unveils yet another restructuring. |
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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 Nick Paul Taylor Gilead has pulled out of an oral COVID-19 antiviral project, telling Matinas BioPharma that it is focusing on its own approach at the expense of its collaborator’s lipid nanocrystal (LNC) technology platform. |
By Max Bayer Three months after scoring FDA approval for a hemophilia B therapy, uniQure is sealing a deal for its next big thing, with $10 million upfront being offered to Apic Bio for an amyotrophic lateral sclerosis gene therapy. |
By Kevin Dunleavy Despite a plan to significantly jack up the price of Comirnaty when the U.S. goes to a commercial model this year for COVID-19 vaccines, Pfizer expects revenue from the shot to plummet by 64% in 2023. And COVID oral antiviral Paxlvoid will be in no better situation, Pfizer said Tuesday. |
By Heather Landi The White House plans to end the COVID-19 national emergency and public health emergency on May 11, the Biden administration said Monday. |
By Andrea Park Just a few months after it began a recall of more than 1 million hemodialysis catheters that were found to be at risk of leakages, Medtronic has spotted the same issue in yet another model of its catheters. |
By Nick Paul Taylor Sensorion has partnered to improve the delivery of its hearing loss gene therapy OTOF-GT. Working with Eveon, the French biotech is aiming to develop an injection system for getting its therapies to the inner ear. |
By Ben Adams New York-based nonprofit Black Health is running a new campaign for Black History Month in February. |
By Robert King A new analysis from the Commonwealth Fund discovered the U.S. spends the most on healthcare but has a lower life expectancy rate compared to other wealthy countries. |
Fierce podcasts Don't miss an episode |
| This week on "The Top Line," we hear from Stacie Dusetzina, Ph.D., a professor at Vanderbilt University's Department of Health Policy, about what the IRA means for the pharma industry. We'll also discuss what we can expect from the biotech industry in 2023. |
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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! .png)
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Whitepaper Exploring the biology of G Protein-Coupled Receptors (GPCRs) and outlining a novel approach for developing therapeutics against previously undruggable GPCRs Sponsored by: Orion Biotechnology |
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On-Demand Webinar See how high-throughput DNA synthesis combined with antibody engineering expertise provides end-to-end antibody discovery solutions. Sponsored by: Twist Bioscience |
Whitepaper Download this paper to learn considerations on how to move a biologic through clinical trials faster, advantages of Parallel Processing, and more. Sponsored by: Catalent |
Whitepaper What are key considerations for intranasal delivery in the treatment of acute and chronic conditions? Sponsored By: Catalent |
Whitepaper Learn how Catalent’s extensive network across APAC can meet a wide range of clinical supply needs for sponsors conducting trials within the region. Sponsored by: Catalent |
Whitepaper Explore Catalent’s newest clinical supply facility in Japan, including the comprehensive services & solutions it offers to help support trials both in-country & around the world. Sponsored by: Catalent |
Case Study Learn more on an approach for process characterization for the production of a monoclonal antibody, which will improve the efficiency and effectiveness of the process by targeting the right design and number of experiments. Sponsored by: Catalent |
| May 1-3, 2023 | Jersey City, NJ |
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