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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.

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Alltrna raises $109M for ‘transfer RNA’ drug vision

Bio Pharma Dive

The Flagship-backed company claims its technology can create genetic drugs that work broadly to treat diseases caused by “stop” mutations.

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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. In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs).

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14th Annual RNA Therapeutics Conference

pharmaphorum

14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases.

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FDA grants orphan drug designation to DTx Pharma’s CMT1A therapeutic

Pharmaceutical Technology

The US Food Drug Administration (FDA) has granted orphan drug designation to DTx Pharma’s investigational DTx-1252 for the treatment of Charcot-Marie-Tooth disease Type 1A (CMT1A). DTx-1252 targets the underlying genetic lesion of the disease and leverages our FALCON platform to unlock the promise of RNAi therapeutics. “We

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RNA interference method could treat muscular dystrophy

Drug Discovery World

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.

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