Radically different
A deeply phenotyped healthy Hong Kong cohort reveals age- and sex-dependent immune, clinical, and lifestyle variation, highlighting population-specific differences and the need to extend systems immunology frameworks beyond European ancestry.
Hang et al. identify an out-of-frame CBX3::ALK fusion in metastatic melanoma that generates functional ALK isoforms through alternative translation start sites. This study demonstrates that rare noncanonical out-of-frame fusions can be oncogenic and therapeutically actionable, highlighting an analytic blind spot in current gene fusion detection pipelines.
SAN DIEGO — Revolution Medicines is already cooking up the next iteration of RAS inhibiting drugs.
At the American Association of Cancer Research annual meeting here, the company is the talk of the town for the clinical trial success of daraxonrasib, its next generation targeted therapy, in advanced pancreatic cancer. And while the company presented more data on that drug Tuesday, showing promising first line and combination data on daraxonrasib, scientists also showed in another session intriguing preclinical data on a completely new compound that may represent what comes after the current lineup.
That drug, currently called RM-055, is what RevMed CEO Mark Goldsmith is calling an entirely “novel class of catalytic inhibitors.” These are targeted therapies that not only block the RAS signaling that drives cancer, but molecularly turn the cancer protein off.
A one-time, personalized cell therapy from Kyverna Therapeutics improved mobility and reduced disabilities in patients with stiff person syndrome, a rare, neurological autoimmune disorder, according to study results presented Tuesday.
Kyverna intends to submit the treatment to the Food and Drug Administration by the middle of the year. If approved, it would become the first treatment for stiff person syndrome and the first personalized CAR-T therapy for an autoimmune disease of any kind to reach the market.
Currently, CAR-T treatments are approved only for blood cancers, but using engineered T cells to deplete B cells — essentially performing an immune system reset inside a patient — has pushed a growing number of biotech companies to shift their CAR-T focus to autoimmune diseases.