“Dune”-inspired Arrakis Therapeutics has tested its spice—an RNA-targeting small molecule—in a mouse model of myotonic dystrophy type 1 (DM1), with the drug reversing the prolonged muscle contraction that is characteristic of the disease. Arrakis, based...
Cartesian Therapeutics reported longer-term results from a mid-stage study of its mRNA CAR-T therapy for myasthenia gravis, suggesting that the therapy is durable beyond the initial three-month study period. In 12 patients, the mean decrease at six months in a...
Antibiotic-driven modifications to ribosomal RNA (rRNA) have been suspected of giving rise to antibiotic resistance. For example, antibiotics can alter methylation patterns in rRNA, changing rRNA structures such that they bind antibiotics less effectively. To study...
SHANGHAI and CLINTON (NJ), November 4, 2024 – HuidaGene Therapeutics, a global clinical-stage biotechnology company focused on genome medicines, has achieved a groundbreaking milestone. The US FDA has cleared its investigational new drug (IND) application for HG202,...
We are witnessing a “golden age” for medicine, marked by breakthroughs in novel treatment modalities. While monoclonal antibodies (mAbs) have become routine, newer platforms like mRNA, antibody drug-conjugates (ADCs), and microbiome-based therapies are gaining...
