Biopharma just wouldn’t be biopharma without continuing innovation. Even in a year rife with economic and regulatory turmoil, the industry still achieved major advancements set to change the health care landscape going forward. Standouts for 2022 include cell therapy and gene editing approaches making significant gains, while industry celebrated a new checkpoint inhibitor added to the oncology armamentarium.

Repeat expansions of two or more base pairs cause dozens of neurological disorders – Huntington’s disease, which is caused by an expansion of the triplet CAG in the coding sequence for huntingtin, is perhaps the most famous one. Now, investigators at Stanford University have shown that cancer genomes, too, frequently feature repeat expansions.

CRISPR gene editing has been one of the important advances of the last decade, in biotechnology and increasingly in medicine. First applied to human cells in 2013, and honored with the 2020 Nobel Prize in Physiology or Medicine, its meteoric rise can make CRISPR look like the molecular equivalent of a miracle healer. But in the research and clinical trenches, CRISPR-based approaches, like any others, need to find applications where their desired effects outweigh their side effects. And finding those applications necessitates ways to identify off-target effects.

Chimeric antigen receptor (CAR) T cells are astounding. In B-cell cancers, they have been transformative. Yet engineering-wise, CAR T cells are in the equivalent of the Model T era. CAR T-cell engineering has already evolved, with the addition of costimulatory domains, which affect cell expansion and signaling. But once the cells are injected into a patient, there is really no way to affect their behavior.

Repeat expansions of two or more base pairs cause dozens of neurological disorders – Huntington’s disease, which is caused by an expansion of the triplet CAG in the coding sequence for huntingtin, is perhaps the most famous one. Now, investigators at Stanford University have shown that cancer genomes, too, frequently feature repeat expansions.

Unlike amphibians, mammals do not regenerate appendages. Except when they do. “If you amputate one of the branches off of the antler [of a reindeer], it will also regenerate,” Jeff Biernaskie told BioWorld. Even without amputation, the antlers of both male and female reindeer regenerate annually, including their skin. That regeneration is “the only large mammal model of true skin regeneration,” he said.

The positively charged nanoparticle polyamidoamine generation 3 (P-G3) can be specifically targeted to either visceral or subcutaneous fat, and affects both types of fat in different ways, researchers from Columbia University reported in two papers recently published.

The positively charged nanoparticle polyamidoamine generation 3 (P-G3) can be specifically targeted to either visceral or subcutaneous fat, and affects both types of fat in different ways, researchers from Columbia University reported in two papers recently published. The studies, published online in Nature Nanotechnology on Dec. 1, 2022, and in Biomaterials on Nov. 28, 2022, are both “a conceptual advance” and “quite amenable to translation,” co-corresponding author Kam Leong told BioWorld.

Researchers have identified a link between amyloid plaques and dysfunctional neuronal conduction in animal models of Alzheimer’s disease (AD). Their study, which was published in the Dec. 1, 2022, issue of Nature, suggests new ways to think about AD, as well as badly needed potential alternatives to plaque removal to fight the disease.