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Laverock Therapeutics, the gene editing-induced genesilencing platform for human therapeutic applications, has expanded its seed funding round to Â13.5 million.
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 post Novo Nordisk’s genesilencing alliance with Dicerna bears first fruit appeared first on.
and colleagues have described a novel form of generegulation that is altered in bladder cancer, leading to the boosting of a gene pathway that helps the cancer cells survive during rapid growth. BIRMINGHAM, Ala. Anindya Dutta, MBBS, Ph.D., Credit: UAB BIRMINGHAM, Ala. Anindya Dutta, MBBS, Ph.D.,
Under the terms of the deal, uniQure will obtain the worldwide rights to develop and market the clinical stage gene therapy, APB-102. uniQure stated that the license of APB-102 further strengthens its gene therapies pipeline developed for the treatment of neurological disorders as well as miRNA-based genesilencing programmes.
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.
But with Novartis failing to convince European regulators about the merits of Xiidra last year, and Allergan’s ageing Restasis potentially facing generic competition, there’s a lot to play for and two biotechs are aiming to disrupt the market. Novartis paid Takeda $3.4
InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulategene expression in cells.
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 billion in cash.
The regulator said it wanted more long-term safety and efficacy data from the REGENERATE study before making a final decision on the drug, which had been expected to be the first entrant into a likely multi-billion dollar market niche. The post High hopes as Inventiva takes NASH contender into phase 3 appeared first on.
The FDA has approved Alnylam’s genesilencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder. The Swiss pharma’s bet looks to have paid off as inclisiran is likely to be approved in Europe in the coming weeks after backing from CHMP regulators last month.
The UK medicines regulator has awarded Alnylam’s RNAi-based therapy zilebesiran for hypertension an ‘innovation passport ‘, a designation designed to speed up NHS access to promising new medicines.
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. These modifications regulategene expression without altering the sequence or structure of DNA.
RNA interference (RNAi) Therapeutics : Fundamentally, RNAi is a natural process of post-transcriptional genesilencing, involving short strands of nucleic acids. Cells use this process to silence and / or inhibit gene expression, via targeted degradation of specific (unwanted) mRNA molecules.
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