BioCentury | Jan 31, 2020
Distillery Therapeutics

CLEC14A-positive stem cell transplants for muscle regeneration

...CLEC14A-positive stem cells could regenerate muscle following injury, despite lacking expression of known myogenic regulator PAX7...
...alone or followed by reinjury with the myotoxic agent cardiotoxin, transplantation with human muscle-derived CLEC14A-positive, PAX7-negative... regeneration of muscle fibers. TARGET/MARKER/PATHWAY: C-type lectin domain family 14 member A (CLEC14A; TNFR5); paired box gene 7 (PAX7)...
BioCentury | Jun 1, 2019
Product Development

ASCO 2019 abstracts show solid tumor race heating up among bispecifics and CAR Ts

...marker of high-risk cancers Translational Genomics Research Institute (TGen) e21516 PAX7-FOXO1 oncogenic fusion protein Rhabdomyosarcoma Paired box gene 7 (PAX7)...
BioCentury | Jul 25, 2018
Distillery Techniques

Disease models

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...
BioCentury | Jul 10, 2018
Distillery Therapeutics


...Cell culture and mouse studies suggest combining Mekinist trametinib with IGF1R inhibitors could help treat PAX3-PAX7... the combination that most potently inhibited cell viability. In a xenograft mouse model of PAX3-PAX7...
...compared with vehicle. Next steps include testing Mekinist and BMS-754807 in additional mouse models of PAX3-PAX7...
BioCentury | Apr 10, 2018
Preclinical News

Researchers identify coding, non-coding driver mutations in MM

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...
BioCentury | Dec 22, 2016
Translation in Brief

Pin the tail

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,...
BioCentury | Dec 6, 2016
Distillery Therapeutics


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...
BioCentury | Jun 16, 2016
Emerging Company Profile

No cells left behind

Although transplantation of human islets from donated pancreatic tissue is well known to restore insulin production in Type I diabetes patients, there is a severe shortage of donor tissue. Islexa Ltd. has developed a way...
BioCentury | Apr 14, 2016
Product R&D

AACR by the numbers

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)...
BioCentury | Dec 14, 2015
Product Development

Following CoMMpass

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,...
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