WELCOME TO FIERCE LIFE SCIENCES WEEKLY DIGEST Intellia Therapeutics aims to show the world that in vivo gene editing could bring a watershed moment in the treatment of genetic diseases. With its partner Regeneron’s CRISPR-based candidate, patients with ATTR amyloidosis saw reduced serum levels of a key biomarker for the rare disease, by up to 87% with high doses. The first-in-human results could also pave the way to further advancements in the field, as the FDA sees more data that gene editing trials can work in living cells. That story, plus our top reads of the week, follow below. | |
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Featured Story | By Noah Higgins-Dunn Novartis claims it accidentally overpaid Roche's Genentech nearly $210 million in antibody patent licensing agreements that eventually led to the discovery of its best-seller Cosentyx. But the company says Genentech isn't returning the money. read more |
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Top Stories Of The Week By Annalee Armstrong Interim results are in for Intellia and partner Regeneron’s in vivo CRISPR/Cas9 genome editing candidate, NTLA-2001, in patients with transthyretin (ATTR) amyloidosis: and the numbers look good. This is the first time gene editing has been proven to work in humans, which “opens up a whole new area of therapies for patients that wasn't there.” read more By Noah Higgins-Dunn Pfizer has predicted that a COVID-19 vaccine booster could be needed as soon as antibody blood concentration wanes to ensure the broad population can't carry the virus, Bernstein analysts wrote in a recent note. However, a key CDC panel doesn't appear to agree. read more By Amirah Al Idrus Chalk another one up for Eli Lilly’s next-generation diabetes drug. All three doses of tirzepatide beat placebo at lowering blood sugar and body weight in adults with early-stage Type 2 diabetes, more than half of whom had never tried a diabetes-specific treatment. read more By Fraiser Kansteiner Novo Nordisk has data showing 2-mg Ozempic topped its lower-dose predecessor on blood sugar and weight reduction. Side effects were on par with the lower dose, too, which could quiet analyst concerns about the potential popularity of stronger GLP-1 meds. read more By Arlene Weintraub Researchers at Massachusetts General Hospital have spent the last two decades building up data to support their idea of repurposing the old tuberculosis vaccine BCG in Type 1 diabetes. Now they have new data backing up their finding that the vaccine restores the ability of regulatory T cells (Tregs) to prevent the destruction of insulin-producing pancreatic islets. read more By Conor Hale The final results from a Grail study of its cancer-tracing blood test found it was accurate at detecting a range of different tumors at early stages—especially those more likely to be lethal and have fewer screening options. read more By Beth Snyder Bulik Moderna has officially earned European Medicine Association approval for Spikevax as its COVID-19 vaccine brand name. Now, it joins Pfizer and BioNTech’s Comirnaty and AstraZeneca’s Vaxzevria, all with European name approvals, in awaiting a U.S. nod. read more By Amirah Al Idrus Anavex’s Parkinson’s disease treatment beat placebo at slowing the progression of motor and non-motor symptoms, a benefit that correlated with an increase in SIGMAR1, the drug’s target and the biomarker the company will use to forecast its efficacy. Anavex will now submit the phase 2 data to the FDA to figure out the drug’s path forward. read more By Fraiser Kansteiner Counting the latest investment, Fujifilm has blueprinted nearly $3 billion in manufacturing expansions since January, primarily in the U.S. The company will divvy up the latest $850 million infusion between operations in the U.K. and the U.S., where the company’s Texas and North Carolina plants are tackling manufacturing of Novavax’s late-stage pandemic vaccine, NVX-CoV2373. read more Resources Sponsored by: Patheon by Thermo Fisher Scientific Choose the right AAV platform for your viral vector-based therapy Sponsored by: Patheon by Thermo Fisher Scientific Take a closer look at your supply chain to ensure it delivers with speed, efficiency, and quality — all within budget. Sponsored by: Blue Matter Consulting E-book to get newcomers “up to speed” quickly on digital health and digital therapeutics while also addressing key issues important to those already familiar with the topic. Sponsored by: WCG Engage Independent Expert Endpoint Adjudication and Data Monitoring Committees in a strategic manner to identify methods for retrospective and prospective evaluations that may improve competitive advantage and bolster regulatory submissions. Sponsored by: PA Consulting What does the future of Cell & Gene Therapy look like? Sponsored by: Clinical Ink For faster deployment, improved patient engagement, and better-quality data — go where your patients are. GO BYOD with Lunexis™ from Clinical Ink. Sponsored by: BBK Worldwide Create a more diverse and inclusive clinical trial enrollment and engagement environment by learning the barriers to participation and the strategies to overcome them. Sponsored by: Thermo Fisher Scientific Autologous cell and gene therapy workflows involve isolating cells from an individual, engineering the cells, expanding and concentrating them, and infusing them back into the patient. Optimized automation of certain steps of the workflow may decrease hands-on time and the cost of the cell manufacturing process. Sponsored By: Veeva Learn how to develop a standards program that has your full team’s buy-in and improves the efficiency and quality of your clinical trials. Sponsored By: Oracle Health Sciences New research indicates an accelerated adoption of decentralized clinical trials due to COVID-19. Sponsored by: Cytiva A one-stop hub for Cytiva resources to support process intesification Sponsored by: Cytiva A one-stop hub for Cytiva resources to support process development and manufacturing of your novel molecule Sponsored By: Remarque Systems Remarque Systems consolidates data, then layers on visualization and automated metrics to generate consistent, actionable data insights in real-time. Click here for more info. Sponsored By: Within3 We enable virtual work — offering over-time sessions where participants are more focused, insights are more diverse and business goals are met. Sponsored by: Catalent Hear industry experts discuss the recent advances, challenges and considerations in pediatric drug development. Real-life examples presented by the experts illustrate the impact of age-appropriate dose forms for pediatrics and highlight insights into the caregiver perspectives. Sponsored by: Catalent Autologous cell therapies have seen propelled growth since 2017, with the spotlight on the first FDA approval of a chimeric antigen receptor (CAR) T cell immunotherapy, Kymriah® (tisagenlecleucel), for the treatment of relapsed/refractory B-cell acute lymphoblastic leukemia. Sponsored by: Catalent Learn about proven strategies for a successful product launch, including cross-functional team collaboration, risk management and using the right project management tools. | 
