DISEASE CATEGORY: Musculoskeletal INDICATION: Musculoskeletal Transplantation with muscle-derived CLEC14A-positive stem cells could regenerate muscle following injury, despite lacking expression of known myogenic regulator PAX7. The myogenic cells were isolated from muscle fiber fragments that underwent...

While strong progress in CAR T therapies will again feature at this year’s ASCO meeting, bispecific antibodies are having a moment -- making more inroads into solid tumors than CAR Ts and branching out via...

TECHNOLOGY: Transgenics and knockouts Zebrafish expressing human PAX3 and FOXO1 could be used to screen therapies for rhabdomyosarcoma. Zebrafish harboring M214K-mutant p53, engineered to express human PAX3 and FOXO1 and injected with a ubiquitous promoter...

INDICATION: Sarcoma Cell culture and mouse studies suggest combining Mekinist trametinib with IGF1R inhibitors could help treat PAX3-PAX7 fusion-negative rhabdomyosarcoma. Screening of a panel of 1,912 approved or tool compound small molecule inhibitors in a...

Scientists from the Institute of Cancer Research (London, U.K.) identified coding and non-coding driver mutations involved in newly diagnosed multiple myeloma, suggesting the genes could be targeted to treat the disease. The research was published...

Stanford University researchers have provided preclinical proof of concept that adding polyadenine (polyA) tails to atypical sites on mRNAs could treat disease. Although the team applied the strategy to the transcript of the fibrosis target,...

INDICATION: Sarcoma Cell culture studies suggest inhibiting CHD4 could help treat PAX-FOX1 fusion gene -positive rhabdomyosarcomas. In two human rhabdomyosarcoma cell lines positive for the PAX-FOX1 fusion oncogene, shRNA targeting CHD4 decreased cell numbers and...

See Figure: Top indications See Table: Novel cancer targets at AACR 2016 See Figure: Top institutions and companies See Figure: Top targets Tables Novel cancer targets at AACR 2016 Breast cancer amplified sequence 1 (BCAS1)...

Amidst a flood of promising data for hot targets, indications and technologies at this year's American Society of Hematology meeting, one collection of presentations stands out not only for the wide-reaching implications of their data,...