Scientists from the Icahn School of Medicine at Mount Sinai have found a sexual dimorphism of depression based on the different expression of a molecule that could be developed as a therapeutic strategy. “There is a big sex difference in depression. Women are much more likely to have depression than men. They tend to have different subsets of symptoms. They tend to respond better to different antidepressants, and the depression tends to be more severe,” Orna Issler, the first author of the study and a postdoctoral researcher at the Nash Family Department of Neuroscience, Friedman Brain Institute, Icahn School of Medicine at Mount Sinai, told BioWorld. Their project, directed by Eric Nestler, a professor of neuroscience and director of the Friedman Brain Institute at the Icahn School of Medicine at Mount Sinai, had the aim to understand the biology of these sex differences of depression and to find therapeutic targets for it.

Rznomics Inc. has received IND approval from the FDA for a phase I/IIa trial of RZ-001 for hepatocellular carcinoma (HCC).

Arizona State University scientists have discovered an unprecedented pathway in a fungus to produce telomerase RNA (TER) from a protein-encoding messenger RNA (mRNA). Unlike in animals and other fungi, this fungal TER is transcribed by RNA polymerase III, lacks a protective 5′ cap and it is processed from the 3′-untranslated region of an mRNA transcript. This telomerase has two essential structural domains that keep it active. For now, scientists have only observed this process in the fungus Ustilago maydis, or Mexican truffle. “In animals, and even in Ascomycota, which is another fungal phylum, the telomerase RNA is transcribed by RNA polymerase II as an independent gene. This is the only case among all different kingdoms in eukaryotes that the telomerase RNA is processed from the mRNA molecule. It is a very unusual biogenesis pathway.” Julian Chen told BioWorld.

Wave Life Sciences Ltd. has highlighted progress with WVE-006, its preclinical Aimer (A-to-I RNA base editing) oligonucleotide candidate for the treatment of alpha-1 antitrypsin deficiency (AATD).

A first-of-its-kind blood test that can detect whether cancer is absent, imminent, or present in different stages is on its way to the market. This is a claim made by brothers Ashish Tripathi and Anish Tripathi, who lead Singapore-based molecular diagnostic company Tzar Labs Pte. Ltd. and Mumbai-based Epigeneres Biotechnology Pvt. Ltd., respectively.

The rapid development of mRNA vaccines to combat COVID-19 has highlighted the importance of this single-stranded molecule. Although there have been some costly developmental bumps along the way, interest in RNA-targeted therapies is once again surging.

RNA has “huge potential” as a therapeutic modality and is beginning to deliver on that potential. But “manufacturing RNA has issues in production, delivery and performance,” Thomas Barnes told BioWorld. Barnes is the CEO of startup Orna Therapeutics LLC, which has the goal of addressing those issues with oRNA, an engineered form of circular RNA.

Pointing to his company’s work on “the next frontier in small molecules,” Ribometrix Inc. CEO Mike Solomon said the potential $1 billion-plus deal with Roche Holding AG offers evidence of big pharma’s mounting interest. Expertise in RNA folds helped Ribometrix nail down an agreement with Basel, Switzerland-based Roche’s Genentech arm that brings $25 million up front and potential milestone payments beyond $1 billion, as the pair discovers and develops RNA-targeted small-molecule therapeutics. Genentech gains exclusive rights to several predefined targets, including an exclusive global license for the development and commercialization of molecules.

Remix Therapeutics Inc., a Cambridge, Mass.-based startup developing small-molecule RNA modulators, said it has raised $65 million in series A financing led by Foresite Capital.