Fabric Genomics Inc.’s Gem artificial intelligence algorithm plus whole genome and whole exome data detected more than 90% of disease-causing variants in infants with rare diseases, a study in Genome Medicine demonstrated. The full process from blood sample to shortlist of causative variants and likely diseases takes just a matter of hours and the time to interpret whole genomes is condensed to about 15 minutes.
Recent news that The University of Texas MD Anderson Cancer Center is teaming up with Schrödinger Inc. to work on the latter’s WEE1 inhibitor – along with data disclosed at the 2021 meeting of the European Society for Medical Oncology (ESMO) – shone a light on the gatekeeper checkpoint kinase, which a number of players are busy exploring, though nothing in the class has been approved so far.
Mitochondrial disease, where the tiny energy factories in cells aren’t functioning properly, can have devastating and sometimes fatal consequences. As there are no approved treatments, patients rely on strategies such as strict diets and conserving energy to try and control symptoms that can affect organs including the brain, kidneys, muscles, pancreas and heart. Netherlands-based biotech Khondrion BV is one of a group of companies that is making progress against this group of rare inherited conditions that affects around one in every 4,000 people.
Biogen Inc. opened the 37th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) virtual meeting with data showing those treated with its multiple sclerosis (MS) therapies had an effective antibody response to COVID-19 vaccination.
It looks like Shanghai Miracogen Inc.’s antibody-drug conjugate (ADC) partnership with Synaffix BV is paying off as MRG-004A, an ADC designed to treat solid tumors, has entered a U.S. phase I/II trial to treat solid tumors.
PERTH, Australia – After meeting with the FDA, Medlab Clinical Ltd. is reformulating its cannabinoid pain product into a synthetic formulation because the agency indicated it was more likely to register a synthetic compared to a botanic.
On the last day of this year’s Molecular Targets meeting, an annual joint conference of the American Association for Cancer Research, the National Cancer Institute and the European Organization for the Research and Treatment of Cancer, the final plenary went from molecular to macro in a lively discussion of the biggest roadblock in cancer drug development, and what can be done to improve it.
DUBLIN – Shares in Idorsia Ltd. were off almost 4% Oct. 11 on news that lucerastat missed the primary endpoint of the Modify phase III trial in Fabry disease. The double-blinded, placebo-controlled study, which recruited 118 adult patients, was designed to evaluate the effect of lucerastat on neuropathic pain. Patients were randomized to drug or placebo in a 2-to-1 ratio.