Adverum shares halved after trial patient goes blind in one eye after experimental gene therapy In a first, FDA issues a serious rebuke for trial data delay as it looks to get tough Brii's COVID antibody combo cleared for phase 3 as it targets high-risk patients to head off hospitalization Sponsored: Look before you leap: How a joined-up development strategy pays off for early-stage biotechs Boundless Bio corrals $105M financing round to prep aggressive cancer programs for clinic Arrowhead, Takeda’s RNA-silencing med cuts scarring in rare liver disease Flagship's Repertoire allies with Yale to identify drivers of multiple sclerosis Adcendo raises €51M series A round to pursue 'ideal' ADC target With $140M in backing and partnership with AbbVie, Capsida ready to make its gene therapy play Roche extends blood test reach with sweeping new use claims in Type 2 diabetes, heart attack risk and more Gene therapy 'development engine' scores $120M to beef up contract manufacturing Featured Story | By Ben Adams Adverum saw its shares down more than 51% after-hours Wednesday night after a patient it was testing a gene therapy on for a common eye condition went blind in the treated eye. read more |
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Top Stories By Ben Adams The FDA is on the verge of slapping Acceleron Pharma with up to a $10,000 fine for failing to publish a summary of its trial data results on a cancer combo using its old med dalantercept. read more By Annalee Armstrong Brii Biosciences has been given the green light to proceed with a phase 3 trial for a COVID-19 combination therapy in ambulatory care patients after the National Institutes of Health shut down a study of hospitalized patients in March. read more Sponsored By: Parexel Biotech A well-thought-out strategic development plan that brings together nonclinical, clinical, regulatory and commercial experts can improve efficiencies, cut costs, shorten timelines, and increase the chances of success for a new drug program. read more By Annalee Armstrong Boundless Bio has wrapped a $105 million series B financing to launch the company's extrachromosomal DNA programs for aggressive cancers into the clinic. The financing was co-led by RA Capital Management and Nextech Invest. read more By Amirah Al Idrus Takeda bet $300 million upfront and promised more than twice that in milestones to work on Arrowhead Pharmaceuticals’ RNA-silencing treatment. Six months later, the program is delivering results, reducing scarring and slashing levels of a disease-causing protein in patients with a rare, genetic liver disease linked to alpha-1 antitrypsin deficiency. read more By Nick Paul Taylor Repertoire Immune Medicines has teamed up with Yale University to understand the immunological causes of multiple sclerosis (MS). The partners hope to identify pathogenic T cells that drive MS and the antigens that activate them. read more By Nick Paul Taylor Adcendo has raised a €51 million ($62 million) series A round to develop antibody-drug conjugates (ADCs). The Novo Seeds-Ysios Capital-led financing positions Adcendo to take an anti-uPARAP ADC to clinical proof of concept in cancer patients. read more By Kevin Dunleavy As an endocrinologist, Bob Cuddihy, M.D., felt stymied with the lack of advances in his field. read more By Conor Hale Roche aims to show you can still teach old biomarkers new tricks. The Big Pharma announced five additional uses for two of its mainstay heart tests, which could extend the diagnostics’ reach to hundreds of millions of new patients. read more By Fraiser Kansteiner Gene therapies are on the rise, but does the industry have the capacity to actually make them? Biotech-CDMO hybrid Forge Biologics thinks it could get there, thanks to $120 million in new funding to boost its viral vector manufacturing services—and advance a few therapies of its own. read more Resources Sponsored by: Datacubed Health The average clinical trial spends $40,000 per patient. Yet despite these high costs, most trials struggle to retain patients. Sponsored By: AmerisourceBergen Developing a quality product and getting it approved for distribution is not enough to guarantee commercial success. Sponsored By: WCG Trifecta The new standards in study training help sponsors and CROs reduce costs by 60%, cut training time by 50%, and start enrollment 55 days faster – learn more today. 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: August Bioservices How has COVID-19 impacted CRO/CDMO customer (dis)satisfaction and outsourcing decisions for pharma and biotech companies in 2021 and beyond? Sponsored by: Cytiva A one-stop hub for Cytiva resources to support process development and manufacturing of your novel molecule Sponsored by: PerkinElmer Informatics Discover the benefits of a cloud-native ELN that delivers better performance, security, connectivity, total cost of ownership PLUS sub-second search results through even the largest data volumes. 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: TAC Roche has inked a $1.8 billion, all-cash deal for GenMark Diagnostics, to acquire its molecular tests designed to screen patient samples for multiple infections simultaneously. 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: Culture Biosciences Find out how upstream bioprocess scientists can use Culture Biosciences’ bioreactor system and real-time data visualizations to get faster insights and get their product to market more quickly. Sponsored By: BC Platforms Those wishing to unlock RWD’s true value must have access to a variety of data sources/types and the technological capabilities to use the data to uncover meaningful insights. Sponsored By: Oracle Health Sciences New research indicates an accelerated adoption of decentralized clinical trials due to COVID-19. 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 drug product process development challenges and risks, the difference between innovator and biosimilar drug product process development, and more during the drug product process development phase. Sponsored by: Catalent Get insights on key considerations for a successful tech transfer process for manufacturing a biologic drug product, including a case study on overcoming challenges in a process transfer for a sterile diluent. Sponsored by: Catalent In a BioPharma Dive industry survey, planning and forecasting were identified as top concerns for trial sponsors. In response to this need, the CSM team at Catalent has developed a methodology for identifying, evaluating, and proactively managing the inherent risks involved in clinical trial supply chain management. Sponsored by: Catalent Learn how to drive excellence within the forecasting process and utilize forecasting throughout the clinical study to better plan clinical supply budgets and project timelines. Explore how to identify potential supply-related issues before they can negatively impact your study. Drug Development Boot Camp® 2021 Onsite and VIRTUAL in real time November 17-18, 2021 | Register now! Pre-Boot Camp preparation is now available. Medical Affairs Strategic Summit (MASS) Virtual May 5-7, 2021 | Virtual Event TMF Virtual Summit May 18-20, 2021 | Virtual Event Vaccines, cell and gene therapy, therapeutic antibodies and RNA drugs May 20-21, 2021 | 12 p.m. EST / 9 a.m. PST Vaccines, cell and gene therapy, antibodies, and RNA-based drugs June 10-11, 2021 | 9 a.m. EST / 6 a.m. PST Biopharma Supply Chain June 29-30, 2021 | Virtual Event | 