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UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into genesilencing and other areas. The post New CEO Mortazavi takes UK biotech e-therapeutics into genesilencing appeared first on.
Novo Nordisk’s chief scientific officer Marcus Schindler called it “a new and innovative way of collaborating”, with the current plan that it will generate between three and five research programmes within the first three years. The move follows Novo Nordisk’s $3.3
RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.
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. The research was published earlier this month in the journal Cell.
The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.
InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.
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. The research was published earlier this month in the journal Cell. pyogenes dCas9.
It has been a year dominated by the pandemic and many life sciences research projects were put on hold as big pharma turned its attention to vaccines and therapies. But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines. Rare disease progress.
Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease.
The present hATTR treatment market constitutes compounds that stabilize the TTR tetramers, a TTR-directed small interfering RNA, and a TTR-directed antisense oligonucleotide, along with supportive therapies. Gene-silencing Therapies. hATTR market : In The Pipeline.
Oxlumo works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate – the toxic metabolite responsible for the clinical manifestations of PH1. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1.
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