Through 'serial de-risking,' Sage is getting closer to building an Alzheimer's contender CRISPR patent dispute not over yet as Emmanuelle Charpentier, universities appeal Phase 3 rare disease failure extends Theravance's losing streak Sponsored: Precision Recruitment at Scale: Rapid ID of High-Risk Participants Takeda's gene therapy hunger not yet quelled as Evozyne pact extended for $400M in biobucks As FDA funding debated, CDER chief makes a pitch for more scientists—not a reorganization Imara flunks phase 2b trials in sickle cell and thalassemia, wiping out clinical pipeline String of NIH departures continues as Ned Sharpless, head of the National Cancer Institute, steps down Ginkgo Bioworks tightens DNA ties with Twist Bioscience to fuel expansion plans Bluebird bio cuts 30% of its workforce as gene therapy dreams dashed in Europe, delayed in US New targets could mean better immunotherapy for colon cancer GlaxoSmithKline recruits PathAI to bring digital pathology AI to cancer, NASH drug development More lawmakers ratchet up pressure on CMS to overturn Aduhelm coverage proposal Ukraine war, China's COVID lockdown and more could weigh on J&J's early-year performance—and it may not be alone: analysts With newly discovered cell type, Penn scientists propose COPD drug development strategy Featured Story | By Annalee Armstrong Anytime an Alzheimer’s disease treatment generates data suggesting it can improve cognitive function, a murmur goes through the patient and caregiver communities. Could this be the one? Sage Therapeutics is presenting a study just like that this week at the American Academy of Neurology’s annual meeting, but the company’s chief development officer Jim Doherty, Ph.D., admits there’s still a lot to learn. read more |
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Top Stories By Annalee Armstrong Nobel Prize winner Emmanuelle Charpentier, Ph.D., and two universities have officially appealed in a dispute over certain patents for CRISPR gene editing, which could have wide implications for companies working on therapeutics based on the technology. read more By Nick Paul Taylor Theravance Biopharma’s losing streak is going on and on and on. After going 0 for 3 last year, the biotech added another failure to its portfolio late on Monday when it disclosed a phase 3 flop in a blood pressure disorder and pointed to a subgroup analysis as a potential path forward. read more Sponsored by: Evidation Health Evidation and BARDA researchers were able to directly recruit subjects for a COVID-19 study via mobile app, & create an AI risk model, while recruiting a representative cohort. read more By Annalee Armstrong It’s all about the proteins for Takeda’s latest pact with molecular engineering company Evozyne. Under a deal worth up to $400 million down the line, they'll work together to develop new proteins that can be incorporated into the next generation of gene therapy for rare diseases. read more By Max Bayer FDA's Patrizia Cavazonni, head of the agency's Center for Drug Evaluation and Research, called on more funding in its medical review office to account for new innovations, particularly for rare diseases. The calls come a week after the agency released its budget requests for fiscal year 2023. read more By Nick Paul Taylor Imara’s tovinontrine has reached the end of the line in sickle cell and beta thalassemia. With the phase 2b trials failing despite a midstudy endpoint change and the use of a higher dose, Imara has called time on its work to establish tovinontrine in the diseases, triggering more damage to its battered stock price. read more By Max Bayer Ned Sharpless, head of the National Cancer Institute, is stepping down at the end of the month. His decision to leave is the latest in a string of departures of top leadership at the National Institutes of Health. read more By Conor Hale After eating up about one billion base pairs to fuel its synthetic biology and cell programming efforts, Ginkgo Bioworks is going back for seconds, with another large order from the DNA weaver Twist Bioscience. read more By Angus Liu The multiple R&D, regulatory and commercialization setbacks have hit home for bluebird bio. Hoping to keep the business afloat, the gene therapy specialist has brought out the cost-cutting ax. read more By Sophia Sorensen Immunotherapies have taken cancer treatment by storm, but so far the approved therapies haven't made much progress in colon cancer. New research has zeroed in on two proteins as potential drug targets that can help the immune system attack this type of cancer. read more By Andrea Park After making its first foray into nonalcoholic steatohepatitis (NASH) drug development with a $1 billion RNAi therapy partnership last fall, GlaxoSmithKline is continuing to carve its own path in the race to develop the first FDA-approved NASH treatment. read more By Robert King More and more lawmakers are putting pressure on CMS to overturn its proposal to have Medicare only cover the Alzheimer's disease drug Aduhelm for beneficiaries in qualifying clinical trials. read more By Fraiser Kansteiner World events have prompted Cantor Fitzgerald analysts to dial back their first-quarter sales expectations for Johnson & Johnson—and it’s possible other big pharmas could be affected, too. read more By Angus Liu Scientists at the University of Pennsylvania’s Perelman School of Medicine have discovered a new type of cell in human lungs. The cells' regenerative ability points to a potential way to treat chronic obstructive pulmonary disease, the team said. read more Resources Presented By: Springer Nature The journey of drug development in 2021 delivered to your inbox: top 10 AdisInsight drug profiles viewed, new drug profiles released, overview of drug launches, key deals, research trends in 2021 and much more (free of charge). Sponsored by: Blue Matter, strategic consultants in the life sciences What’s in store for the oncology market in 2022 and beyond? This paper looks at 5 trends driving the market. Sponsored by: Thermo Fisher Scientific Learn how to position quality as a central component within your raw material sourcing strategies for the chemicals used throughout your workflows. Download the free whitepaper from Thermo Fisher Scientific. Sponsored by: Medidata, a Dassault Systèmes company The landscape of clinical trials has been permanently changed. 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Sponsored by: Catalent What is the most suitable dosage form technology for the Oral Delivery of Lipid-Based Formulations? Sponsored by: Catalent Biologics Ypsomed and Catalent Biologics experts discuss strategies for integrated PFS manufacturing and auto-injector assembly to accelerate timelines and more in this webinar. Sponsored by: Catalent Biologics Learn how formulation development for a PFS affects manufacturing to drive the industry toward optimizing delivery of injectable products and, ultimately, improving patient care. 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