Insulin for diabetes. Lactase for gastrointestinal tract disorders. Factor VIII and IX for hemophilia. There is no doubt about the therapeutic potential of human proteins.   With this in mind, two of Flagship Pioneering’s senior principals, Hozefa Bandukwala, PhD, and Theonie Anastassiadis, PhD, asked: Are there potential sources of novel and powerful therapeutics yet to be revealed in nonhuman organisms?  Anastassiadis picks up the story: “Immediately, viruses came to mind because they’re rapidly evolving to be able to drive and modulate complex processes. In doing so, they act ..read more

Brain metastases, as these secondary tumors are called, most commonly arise from solid tumors like breast, lung, and colon cancer and are often associated with a poor prognosis. When cancer breaches the brain, it can be difficult for treatment to follow, in part because of the blood-brain barrier, a near-impenetrable membrane that separates the brain from the rest of the body. Now, researchers at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine say they have developed a nanoparticle that can penetrate the blood-brain barrier. The findings are publi ..read more

REGISTER NOW Broadcast Date:  Friday, May 10, 2024 Time:  8:00 am PT, 11:00 am ET, 17:00 CEST Organ-on-a-chip (OOC) technologies are now being used across a range of drug discovery applications, through efficacy and into safety assessment. As the technology and applications evolve, new questions and opportunities are arising in terms of regulatory acceptance, cost efficiency, and model confidence through standardization. In this GEN Learning Lab, CN Bio’s CSO Tomasz Kostrzewski, PhD, and toxicology consultant Clive Roper, PhD, will discuss all things OOC, including cu ..read more

Researchers at the University of Birmingham and Ludwig Maximilian University of Munich have for the first time been able to locate an internal neural compass that the human brain uses to orientate itself in space and navigate through the environment. The research identifies finely tuned head direction signals within the brain. The results are comparable to neural codes identified in rodents and have implications for understanding diseases such as Parkinson’s and Alzheimer’s diseases, where navigation and orientation are often impaired. The findings are published in Nature Human Behavior in an ..read more

Wacker Biotech US, a San Diego-based subsidiary of Wacker Chemie, reports that it is expanding its mRNA and protein production. As a CDMO, the company offers several fermentation lines with a capacity of up to 650 L fermentation and subsequent downstream processing steps. Wacker Biotech US is now creating additional development and production capacities in the field of advanced therapy medicinal products to offer to the U.S. market. By putting new laboratories into operation, Wacker Biotech US is expanding its bioprocess development capabilities for pDNA, mRNA, and recombinant proteins to supp ..read more

Results from a small clinical study suggest that EDIT-101, a CRISPR-based gene editing therapy for an inherited form of Leber’s congenital amaurosis (LCA), is safe and efficacious. The therapy did not cause any serious treatment or procedure-related adverse events, and there were no dose-limiting toxicities. Moreover, the therapy resulted in several clinically meaningful improvements in vision. The study, a Phase I/II trial called BRILLIANCE, was led by principal investigator Eric Pierce, MD, PhD, of Mass Eye and Ear, a member of the Mass General Brigham healthcare system. It was sponsored by ..read more

Amgen chairman and CEO Robert A. Bradway Amgen (AMGN) signaled it intends to become the third powerhouse developer in the booming weight loss drug segment on Thursday, as two of its top executives delivered positive comments about the progress of its obesity candidate Maridebart Cafraglutide (MariTide, formerly AMG 133)—comments that propelled Amgen shares to their biggest gain in 15 years Friday. Addressing analysts on his company’s quarterly earnings call following the release of first-quarter results, chairman and CEO Robert A. Bradway said Amgen was “very encouraged with the results” glean ..read more

Working with human breast and lung cells, Johns Hopkins Medicine scientists say they have charted a molecular pathway that can lure cells down a hazardous path of duplicating their genome too many times, a hallmark of cancer cells. The findings reveal what goes wrong when a group of molecules and kinases are triggered to regulate the cell cycle, resulting in replication of chromosomes in cells that exit the cell cycle. The findings, the team suggest, could be used to develop therapies that interrupt these abnormal events in the cell cycle, and have the potential to stop the growth of cancers ..read more

REGISTER NOW Broadcast Date:  Thursday, June 13, 2024 Time:  8:00 am PDT, 11:00 am EDT, 17:00 CEST Studying the spatial organization of all the cells in the niche or neighborhood of a tumor is crucial to fully understand how cancers progress. Since proximity speaks to cellular activity, the spatial positioning of cells certainly plays a role in modulating clinical outcomes. However, most platforms used to reconstruct the tumor ecosystem (TE) fail to include spatial context in the three-dimensional (3D) space of a solid tumor with single-cell resolution, and thus lack informatio ..read more

BioSolution Designs (BSD) reported that it is collaborating with RoosterBio to simplify the development and manufacturing of engineered human mesenchymal stem/stromal cell- (MSC) derived therapies. The partnership leverages BSD’s multigenic design and assembly platform, Bird of Prey, and cell engineering expertise along with RoosterBio’s MSC and exosome bioprocessing products, manufacturing protocols, and analytical services, according to Thomas D. Reed, PhD, CEO of BSD, with the goal of delivering an end-to-end solution for the engineering and development of cell- and exosome-based therapeuti ..read more