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Cell and gene therapies have revealed great promise for the treatment of rare diseases. But development of these advanced therapeutics is complex - requiring careful attention to study design and patient safety.
Read our guide to understand the current landscape of cell and gene therapy (CGT) and explore expert insights on the key challenges and questions to address when designing trials in rare diseases. Premier Research. Built for Biotech℠ 
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Today’s Big NewsApr 11, 2024 |
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Monday, April 22, 2024 | 10am ET / 7am PT Explore cutting-edge NASH research with a novel NAFLD/NASH animal model mirroring patient experiences. Witness Resmetirom's impact on fat accumulation using human cell-based in vitro models. Expect clinical relevance, quick insights in 12-16 weeks, and cost savings. Register now! 
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| By Max Bayer Chinese biotechs have never had so much success inking cross-region out-licensing deals. But could an increase in national security dampen the scorching-hot space? |
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By Nick Paul Taylor Novartis is adding another weapon to its arsenal of oncology therapies, paying Arvinas $150 million upfront for rights to a phase 3-ready protein degrader that could treat a wide range of prostate cancer patients. |
By Max Bayer Vertex is buying Alpine Immune Sciences for $4.9 billion, the companies announced Wednesday. At the center of the deal is Alpine's midstage IgA nephropathy treatment. |
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 James Waldron It’s been a busy morning for business development in the autoimmune space, with Century Therapeutics snapping up Clade Therapeutics and Eliem Therapeutics striking a deal for Tenet Medicines. |
By Gabrielle Masson On the heels of Relyvrio’s market withdrawal in amyotrophic lateral sclerosis, Amylyx Pharmaceuticals has shared early data from a phase 2 trial in a separate rare disease that the biotech is touting as “clinically meaningful.” |
By Gabrielle Masson A few weeks ago, Johnson & Johnson nabbed FDA fast-track status for an investigational therapy in a rare blood disorder called fetal and neonatal alloimmune thrombocytopenia. Now, the Big Pharma is joining hands with Rallybio, paying the biotech to promote the companies’ complementary programs designed to reduce the risk of the condition. |
By Helen Floersh Verastem Oncology, Quanta Therapeutics, Frontier Medicines and more shared preclinical updates at the American Association for Cancer Research’s annual meeting in San Diego in early April, adding to the ever-growing list of companies vying to bring forth therapies against the once-”undruggable” target. |
By Helen Floersh MoonLake Immunotherapeutics has signed a three-year deal with health analytics software maker Komodo Health for tech to help with clinical trial selection and a range of other data needs. |
By Conor Hale The process, being developed by First Ascent Biomedical, showed promise in a small study published this week in Nature Medicine. |
By Fraiser Kansteiner The American Society of Health-System Pharmacists has released new data showing there were 323 drugs in shortage in the U.S. as of 2024’s first quarter—the highest number recorded since ASHP began tracking shortage data back in 2001. |
Fierce podcasts Don’t miss an episode |
| This week on “The Top Line,” join Editor-in-Chief of Fierce Life Sciences and Healthcare Ayla Ellison as she sits down with Kumar Madassery, M.D., Director of Peripheral Vascular Intervention & CLI Program at Rush University Medical Center, to explore peripheral arterial disease—a serious health issue that is often overlooked and undermanaged. |
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Tuesday, April 30, 2024 | 2pm ET / 11am PT Liquid biopsies have shown tremendous progress for the early detection of cancer and for treatment monitoring, but still have limitations. The inclusion of protein glycosylation can help. Join usto learn more about the latest solutions for leveraging AI and machine learning to accelerate data processing, overcome current barriers, and provide crucial information. Register now. 
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eBook Unlock the future of mAb manufacturing with our complete guide to integrating the revolutionary Gibco™ Efficient-Pro™ Medium and Feeds System, 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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