Gene editing can repair mutations that prematurely halt protein synthesis, resulting in incomplete peptides that cause various diseases. However, other approaches achieve the same effect without altering the genome. Startup Alltrna Inc. has developed a strategy based on transfer RNA (tRNA) to bypass the premature stop codons that end early protein translation. The company already has a first clinical candidate that could treat metabolic diseases such as methylmalonemia (MMA) or phenylketonuria (PKU). Read More

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive degeneration of upper and lower motor neurons, resulting in paralysis and death typically within 3-5 years of symptom onset. Historically, treatment options have been extremely limited. However, the identification of genetic contributors to ALS pathogenesis has enabled the application of antisense oligonucleotides (ASOs) to selectively modify or reduce the expression of disease-associated genes at the RNA level. Read More

Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration. Read More

Taigen Biotechnology Co. Ltd. and its Taigen Biopharmaceuticals subsidiary have entered into an exclusive in-licensing agreement with Insilico Medicine Inc. for ISM-4808, a prolyl hydroxylase domain (PHD) inhibitor. Read More

Shenzhen Targetrx Inc. has disclosed proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase-binding moiety covalently linked to a Bcr-Abl (Bcr-Abl1) kinase and its mutant targeting moiety through a linker reported to be useful for the treatment of cancer and immunological disorders. Read More

Antibodies targeting CD269 and GPRC5D have shown unprecedented clinical efficacy in the treatment of multiple myeloma (MM), but many patients still develop progressive disease. It was hypothesized that dual-targeting T-cell immunotherapies might improve the efficacy by addressing the difficulty of heterogenous target expression and preventing resistance development due to antigen escape. Read More

Superoxide dismutase 1 (SOD1) mutations were among the first genetic causes identified in familial amyotrophic lateral sclerosis (ALS) and confer a toxic gain-of-function that drives motor neuron degeneration via protein misfolding, oxidative stress, mitochondrial dysfunction and neuroinflammation. Read More

Shanghai Aryl Pharmtech Co. Ltd. and Zhejiang Hisun Pharmaceutical Co. Ltd. have synthesized aza-tricyclic derivatives acting as Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer. Read More

Nanjing Minova Pharmaceutical Co. Ltd. and Precision Brain Science Biotechnology (Suzhou) Co. Ltd. have identified salts of deuterated dextromethorphan reported to be useful for the treatment of cough, pain, epilepsy, cancer, rheumatoid arthritis, dermatitis, cardiovascular disorders and glaucoma. Read More

Pancreatic cancer is a leading cause of cancer-related deaths worldwide and presents a 5-year survival rate of under 12%. Most patients are diagnosed at an advanced stage, with over half of them presenting with metastatic disease at diagnosis. Read More

Inventisbio Co. Ltd. and Inventisbio LLC have divulged kinesin-like protein KIF18A inhibitors reported to be useful for the treatment of cancer. Read More

Apollo AP45 Ltd. has described eukaryotic translation initiation factor 2-α kinase 3 (PERK) inhibitors reported to be useful for the treatment of cancer. Read More

Yes-associated protein (YAP), encoded by Yes1-associated transcriptional regulator (YAP1) and transcriptional coactivator with PDZ-binding motif (TAZ), encoded by WW domain containing transcription regulator 1 (WWTR1), are two crucial paralog transcriptional regulators of the Hippo pathway regulating organ size, tissue homeostasis and disease progression. Read More