Targets & Mechanisms

CPSF3 emerges as new synthetic lethality target in AML and sarcoma

A team led by the Novartis Institutes for BioMedical Research identified CPSF3, a pre-mRNA processing endonuclease, as a novel synthetic lethality target for a subset of AML and sarcomas. The premise of synthetic lethality is...

For $75M up front, Biogen will pursue Pfizer castoff for circadian rhythm in AD, PD

Biogen acquired a clinical asset from Pfizer and will investigate its potential to normalize circadian rhythm to treat “sundowning” in Alzheimer’s disease and irregular sleep associated with Parkinson’s disease. Monday’s deal is Biogen’s second in...

Colitis-associated somatic mutations yield new colorectal cancer target

Kyoto University team identifies colitis-associated somatic mutations that are lost in colitis-associated cancer to reveal a new colorectal cancer target. A Japanese team has identified novel colorectal cancer targets by contrasting somatic mutations in cancerous...

Cancer's trajectory, as viewed by targets at AACR, ASCO and ASH

Beyond the headliner presentations at 2019’s three major cancer conferences were over a hundred posters and presentations showcasing new or emerging targets. A look at the identity of those targets reveals the growing diversity of...

SIGLECs go from homing beacons to next-generation checkpoints

Propelled by growing understanding of their biology, and an expression pattern that goes well beyond T cells, SIGLEC proteins are rising as the next set of checkpoint targets to challenge PD-1. The sugar-binding proteins could...

Controlling T cells via epigenetics

An international academic team has discovered an epigenetic pathway that regulates CD4 + T cell differentiation and activity, and could yield new therapeutic targets for autoimmunity. As drug developers seek the next generation of targeted...

Bispecifics and allogeneics steal the spotlight from autologous CAR Ts

Standing out at this year's ASH meeting is a pair of growing threats to first-generation CAR T cell therapies: bispecific antibodies and allogeneic cell therapies. Gone are the days when autologous CAR Ts were the...

Emerging immuno-oncology mechanisms at ASH 2019

Researchers are finding new ways to monitor the immune system in hematologic diseases that could have implications for optimizing immuno-oncology therapies, according to BioCentury’s analysis of emerging targets at ASH 2019. In abstracts released ahead...

A view of immune reprogramming from ASH

ASH presentations on immune reprogramming point to new targets and mechanisms that control macrophage and T cell phenotype switching, and highlight the phenomenon's translational relevance in diseases beyond solid tumors. Strategies to convert immunosuppressive macrophage...

Genocea's cautionary tale: avoid inhibitory neoantigens in vaccines

Genocea’s SITC presentations bolstered its case for screening out inhibitory neoantigens from cancer vaccines by suggesting the peptides play a role in checkpoint inhibitor resistance and promote disease progression. One of the keys to designing...

New roles for HDACs in the cytoplasm and muscle disorders

While HDACs are known for their epigenetic function, NIBR researchers have discovered cytoplasmic HDAC4 substrates involved in muscle physiology. The findings suggest blocking HDAC4 deacetylation activity could treat muscle atrophy and other musculoskeletal disorders. HDACs...

Impaired wound healing: CD47’s latest safety hurdle?

Just as anti-CD47 antibodies are getting past their first safety hurdle of hematological toxicities, a new study has cautioned that the antibodies could have a dangerous effect on wound healing. In a paper published in...

New cereblon substrates linked to thalidomide teratogenicity

A Celgene-funded study from Tokyo Medical University and the University of Milan has identified two new thalidomide-dependent CRBN substrates that could be applied to screens for new, safer thalidomide derivatives. Thalidomide and its analogs, collectively...

How glycoRNA could go from surprise discovery to therapeutic target

The first evidence that sugars decorate RNA could open up new therapeutic opportunities in autoimmunity and cancer, but industry won't invest until structural data and disease associations emerge. Beyond the growing taxonomy of RNA subtypes...

CAR Ts can move well beyond cancer, but will need to go allogeneic first

Preclinical studies showing the utility of CAR T cells outside of cancer are ramping up, and developers have taken notice. But turning the modality into a general-purpose tool will require companies to break through safety...