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.
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.
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.
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.
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.
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.
Peptidream Inc. has announced a preclinical development milestone in its collaboration with Alnylam Pharmaceuticals Inc. under their siRNA conjugate discovery collaboration.
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.
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).
Disco Pharmaceuticals GmbH announced the final close of its seed round at €36 million (US$42 million), as it shapes up novel targets it has discovered with its surfaceome platform technology for formal preclinical development.
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