In the largest genetic study of schizophrenia published to date, researchers have identified 50 new gene variants, and half a dozen functionally related groups of genes, that affected schizophrenia risk. They also showed that schizophrenia risk variants were more likely to be found in the relatively small fraction of the genome that is essential for normal development and function. Read More
Gene therapy occupies a "really excellent place" after "extraordinary" scientific advances, several approvals, a plump development pipeline and a flurry of partnering deals, observed Geoff McDonough, president and CEO of Generation Bio. But despite their "unprecedented opportunities to provide durable benefit," version 1.0/2.0 gene therapies also bear significant mechanistic limitations that confine their usefulness to small populations and relatively rare indications. Read More
Senti Biosciences Inc., a synthetic biology company developing an immuno-oncology drug, has landed a $53 million series A round led by New Enterprise Associates. The money is expected to help advance its cancer asset to the starting line for an investigational new drug filing and accelerate the scaling of what it calls a "genetic circuit programming platform." Read More
Quentis Therapeutics Inc.'s $48 million in series A money will help, among other things, put into the clinic next year a small-molecule IRE1 alpha inhibitor for proof-of-concept work. Going after cancer immunotherapies that modulate endoplasmic reticulum (ER) stress response pathways in the tumor microenvironment (TME), Quentis will use the funds to push the leading prospect through clinical proof of concept, develop a pipeline of preclinical programs, and beef up the company's team. Read More
The delay of the FDA's intended use rule was welcome news, but the comments to the docket suggest that the agency cannot simply turn back the hands of time, and must instead clarify a number of issues, including the circumstances under which knowledge of off-label use can be construed as intent. Read More
Russian biopharma Biocad was ahead of the curve when it concentrated its initial drug development efforts in then-nascent biosimilars. With a handful of biosimilars assets now launched in Russia and beyond and more in the pipeline, the St. Petersburg-based company increasingly is turning its attention to novel drugs, with an eye to expanding its market in a big way. Read More
Sterotherapeutics LLC, of Philadelphia, completed an initial financing, the amount of which was undisclosed, led by the current management team. The proceeds will be used for phase I/II testing as well as additional R&D activities, including funding incremental working capital. Read More
Biocryst Pharmaceuticals Inc., of Research Triangle Park, N.C., and Idera Pharmaceuticals Inc., of Exton, Pa., said they filed a joint preliminary proxy statement/prospectus with the SEC in connection with their proposed merger. Read More
Cynata Therapeutics Ltd., of Melbourne, Australia, reported data from the first cohort of eight patients in its phase I trial testing CYP-001, a Cymerus mesenchymal stem cell product, who received 1 million cells per kilogram of bodyweight, up to a maximum of 100 million cells per infusion, for the treatment of steroid-resistant graft-vs. host disease (GvHD). Read More
Exelixis Inc., of South San Francisco, said renal cell carcinoma drug Cabometyx (cabozantinib) generated net product sales of $90.4 million and $324 million for the fourth quarter and full year, respectively. Cometriq (cabozantinib), approved for progressive, metastatic medullary thyroid cancer, posted sales of $5.3 million and $25 million for the quarter and year, respectively. Read More
The U.S. House of Representatives passed several public health bills Monday that will prioritize prevention efforts, improve education for medical professionals, and establish or reauthorize grant programs. Read More