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Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions. This is also one of the major challenges that slow the development of effective treatments for FTD, he adds. This protein promotes lysosomal function.
Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The nomination of a first candidate in the collaboration that will advance into development triggers a $2.5
The findings could yield important implications for development of drugs to treat cancer and other diseases. BOSTON – A long-running debate over how an important gene-silencingprotein identifies its targets has been resolved by researchers at Massachusetts General Hospital (MGH).
Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.
Alnylam’s gene-silencing drug Amvuttra has been recommended as a treatment for hereditary transthyretin-related (ATTR) amyloidosis by NICE, paving the way for the drug to be made available on the NHS in England and Wales.
(Nasdaq: ALNY ) announced today positive interim results from the ongoing single ascending dose part of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy (CAA). Yancopoulos, M.D.,
The bulk of Novo Nordisk’s revenues come from its diabetes products, although it also active in the development and sale of drugs for growth disorders, haemophilia and sickle cell disease. In ATTR amyloidosis, transthyretin (TTR) protein misfolds and accumulates as amyloid deposits throughout the body.
Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.
Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silencegene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.
Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. It also gets two preclinical-stage development projects.
Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. Epigenome Editing with CRISPR.
Together, the ILLUMINATE studies comprise a comprehensive clinical development program intended to demonstrate the safety and efficacy of Oxlumo across the full spectrum of patients diagnosed with PH1. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development.
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