For 75 years, the standard tools for autoimmune disease have consisted of steroids, cytotoxics and broad biologics that tamp down the entire immune system. They can help, but they are rarely curative. “They’re blunt instruments,” Regcell Inc. CEO Mike McCullar told BioWorld. “They can’t distinguish good immune cells and bad immune cells,” which is why many carry black-box warnings and must be taken for years, sometimes for life.

With a modest $7 million up front but an ultimate payout potentially topping $1 billion on the line, Modex Therapeutics Inc. will collaborate with Regeneron Pharmaceuticals Inc. to find and develop multispecific antibodies for treating multiple indications.

Researchers at Monash University in Melbourne, Australia, have uncovered the hidden code governing how genetic mutations affect RNA splicing and result in disease. The researchers were able to identify the specific mutations that cause changes in RNA splicing, Sureshkumar Balasubramanian, the lead researcher at Monash University’s School of Biological Sciences, told BioWorld.

A technology that combines transcriptomic data and AI enables a novel approach to drug discovery based on the state of cells, how they behave and which genes they express. The Drugreflector model, developed by scientists at Cellarity Inc., learns from gene expression profiles and predicts which compounds could induce beneficial changes in that cellular state to develop a treatment.

Ventus Therapeutics Inc. has entered into a multi-year collaboration with Genentech Inc., a member of the Roche Group, to discover and optimize novel small-molecule candidates for challenging targets in major disease areas using Ventus’ Resolve drug discovery platform.

Durable reprogramming of human T cells may now be possible thanks to a new technique based on the CRISPRoff and CRISPRon methodology. Researchers from the Arc Institute, Gladstone Institutes, and the University of California San Francisco (UCSF) have stably silenced or activated genes in this type of immune cell without cutting or altering its DNA, making T cells more resistant, active, and effective against tumors.

Judo Bio Inc. has announced preclinical data demonstrating that its megalin-STRIKER oligonucleotide therapeutics achieved robust and sustained, kidney cell-selective gene silencing in rodents and nonhuman primates (NHPs).

In vaccine development, one might think that targeting multiple epitopes increases the likelihood of improving outcomes. However, when several immunogens are administered together, the immune system does not always generate antibodies against all of them. Two parallel studies have overcome this challenge by using multiple simultaneous immunogens against HIV, effectively triggering various types of broadly neutralizing antibody (bnAb) precursors in two different preclinical animal models.

A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.

Nabla Bio Inc. has established a new multiyear research collaboration with Takeda Pharmaceutical Co. Ltd. to advance AI-driven design of protein therapeutics.