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Silence Therapeutics gets grant for compound for rna interference therapy in treating liver disease

Pharmaceutical Technology

Discover the groundbreaking patent by Silence Therapeutics Plc for a compound revolutionizing RNA interference therapy. Learn how formula (II) is paving the way for targeted treatment of liver disease and genetic disorders.

RNA 130
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Exsilio debuts with $82M, pitching a ‘leap’ forward for genetic medicine

Bio Pharma Dive

The company claims it can address some of the field’s limitations with medicines delivered via messenger RNA and lipid nanoparticles.

Medicine 280
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Beyond Covid-19: the disruptive potential of RNA-based therapeutics

Pharmaceutical Technology

Further, the acceptance of new mRNA vaccines has rejuvenated activity within previously established categories of RNA therapeutics including lifesaving antisense technologies. These include antisense oligonucleotides (ASO), RNA interference (RNAi), and RNA aptamers.

RNA 147
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Lilly and ProQR to expand genetic medicine development agreement

Pharmaceutical Technology

Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. This alliance is utilising the Axiomer ribonucleic acid (RNA) editing platform of ProQR to address ailments affecting the liver and nervous system.

Genetics 328
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Opinion: Interest in RNA Editing Accelerates as Therapies Approach the Clinic

BioSpace

The next frontier in RNA therapies, RNA editing has the potential to treat both genetic and common disorders, and the technology is rapidly expanding beyond the liver.

RNA 105
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Silencing RNA causes moles to self-destruct in rare condition

Drug Discovery World

UK researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin condition. The team used a genetic therapy called silencing RNA, which blocks the action of the mutated NRAS in mole skin cells. The treatment could be used to reverse moles, and therefore prevent cancer.

RNA 52
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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

Last week, CAMP4 Therapeutics announced the close of a $100 million Series B round , which will be used to advance their regulatory RNA (regRNA)-focused programs. However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.

RNA 245