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. Read More
Cutaneous squamous cell carcinoma (cSCC) is the second most common skin cancer, arising from abnormal proliferation of epidermal keratinocytes due to chronic UV exposure. While highly curable (>90%) with surgical excision when detected early, it carries a significant risk of local invasion and metastasis if left untreated. Read More
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. Read More
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. Read More
Arvinas Operations Inc. has disclosed proteolysis targeting chimera (PROTACs) compounds comprising a cereblon (CRBN) E3 ubiquitin ligase-binding moiety covalently linked to a B-cell lymphoma 6 protein (BCL-6)-targeting moiety through a linker with potential for use in the treatment of cancer. Read More
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. Read More
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). Read More
The Institute of Materia Medica Chinese Academy of Medical Sciences and the Peking Union Medical College have reported magnolol/honokiol nitrone derivatives acting as nitric oxide (NO) production inhibitors for the potential treatment of amyotrophic lateral sclerosis, traumatic brain injury, cerebral infarction, autoimmune disease, and respiratory tract and skin inflammation, among others. Read More
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. Read More
TYK Medicines Inc. has synthesized cyclin-dependent kinase 4 (CDK4) inhibitors designed for use in the treatment of cancer, infections, and metabolic, inflammatory cardiovascular and inflammatory disorders. Read More
Yuhan Corp. has discovered glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of obesity, diabetes, hypertension, hyperlipidemia, hypoglycemia, coronary artery disease, chronic kidney disease and cardiovascular disorders, as well as hepatic steatosis and alcoholic fatty liver disease. Read More
To overcome the limitations regarding conventional immunotherapy for treating tauopathies, researchers from Sanofi SA aimed to improve brain exposure and targeting pathological tau species by optimizing antibody design. Read More
