Eight years after Novartis AG gained U.S. FDA approval of the first CAR T therapy, Kymriah (tisagenlecleucel), for B-cell acute lymphoblastic leukemia, developers are advancing prospects that could significantly impact another disease space outside of cancer – autoimmunity. The efforts are getting a swirl of attention, with Bristol Myers Squibb Co. (BMS) announcing Oct. 10 that it would offer $1.5 billion in cash to buy three-year-old privately held Orbital Therapeutics Inc., including its lead, next-generation CAR T-cell therapy OTX-201, which is designed to reprogram cells in vivo for autoimmune diseases. Read More
Biopharma dealmaking was up 32% month over month in September, reaching $27.15 billion in total value, up from $20.52 billion in August. It was the fourth-highest month for deals of the year, which through the month average $23.6 billion a month. Year-to-date deal value (January through September) reached $212.44 billion, surpassing the nine-month totals of all previous years. Read More
A paper last month in the Journal of Clinical Oncology reported on the pooled analysis of data showing that the use of neoadjuvant immune checkpoint inhibitors work better than the frequently used FLOT regimen (fluorouracil, leucovorin, oxaliplatin and docetaxel) in certain gastroesophageal adenocarcinoma (GEA) cancers. But there’s plenty more coming down the pike, even as scientific knowledge about the disease advances. Read More
While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity. Read More
BioWorld Staff Writer Randy Osborne reflects on the transformative progress in gene therapy – from tools like CRISPR and AAV vectors to major breakthroughs in diseases like hemophilia, sickle cell and beta-thalassemia. Read More
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Adcendo, Ascendis, Cidara, OS therapies, Regeneron, Xspray, Zelluna. Read More
