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Programmable Oligonucleotide Delivery System (PODS), developed by Sixfold Bioscience, is a versatile system which delivers short interfering RNA (siRNA) genesilencing cargo to specific cancer cells.
It was previously expected to enter clinicaldevelopment last year, but no announcements were made. In addition to gene therapy, genesilencing, which can be done using ASOs, is also an interesting approach, says Barmada.
More Drugs for Dry Eye Disease Are on the Way Several other drugs have recently gained FDA approval or are currently in advanced stages of clinicaldevelopment for the treatment of dry eye disease. One such approved medication is Miebo , an ophthalmic solution developed by Bausch + Lomb and Novaliq.
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. If approved, it could become a $500 million-a-year product, according to the company.
With 28 pioneering speakers from large pharma, innovative biotech and KOLs of academia who are ready to discuss the full and comprehensive range of RNAi drugs from discovery to development and beyond, join us in Boston to hear how they address the major challenges facing the industry.
In summer, Alnylam’s genesilencing drug Oxlumo, the first treatment for primary hyperoxaluria type 1 (PH1), an ultra-rare and life-threatening genetic disorder Oxlumo was approved in the European Union and the US. Rare disease progress. The post 2020 review – Pharma’s progress outside of COVID-19 appeared first on.
While this narrative will undoubtedly evolve throughout the clinicaldevelopment process, the first draft should be developed as early as the IND-enabling stage. This will create an early shared understanding that will provide a framework for consistent communications throughout clinicaldevelopment and post launch.
The safety and efficacy of Oxlumo are also being evaluated in the ongoing ILLUMINATE-C Phase 3 clinical trial in patients of all ages with advanced PH1, including patients on dialysis. Visit OXLUMO.com for more information, including full Prescribing Information. Until today, there were no approved pharmaceutical therapies for PH1.
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