Gene editing holds promise for treating neuromuscular disorders such as limb-girdle muscular dystrophy, but its clinical translation remains challenging due to a lack of complementary delivery tools for the extensive network of skeletal muscles in the human body. A team at University of Massachusetts Chan Medical School compared editing outcomes mediated by either Cas9 mRNA and RNP delivery to skeletal muscle via local injection in the context of the previously described selective organ targeting (SORT) lipid nanoparticles (LNPs) platform.

Discoveric Bio Alpha Ltd. and collaborators have presented data regarding the rationale and design of NIDB-3101, a third-generation, human IgG1 anti-tau biparatopic antibody for the treatment of Alzheimer’s disease (AD).

Insilico Medicine Cayman Topco has established a strategic research collaboration with Aska Pharmaceutical Co. Ltd. to identify novel therapeutic targets for challenging gynecological conditions, including endometriosis, uterine fibroids and adenomyosis.

Rybodyn Inc. has announced the initial close of a $10 million seed financing to support its work decoding the dark proteome using an AI-powered novel sequencing and discovery platform. The financing will accelerate the company’s transition from foundational discovery into scaled platform execution and progress early-stage programs into IND-enabling studies.

Epifrontier Therapeutics Inc. has been awarded a grant of up to $32 million in nondilutive funding from the Japan Agency for Medical Research and Development (AMED) to advance the clinical development of EPF-001 (RK-701), a first-in-class G9a inhibitor being developed for sickle cell disease and β-thalassemia.

PerturbAI has emerged from stealth mode with the release of the world’s largest in vivo CRISPR atlas as described in a preprint on Biorxiv. The study profiled over 7.7 million cells from the brains of 74 mice with different cellular knockouts of 1,947 disease-associated genes. The San Francisco-based company’s Perturb-seq platform combines CRISPR perturbations with single nucleus RNA sequencing to look at gene expression.