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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. CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year.

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RNA-based immunotherapy eradicates melanoma tumours

Drug Discovery World

Investigators at the Icahn School of Medicine at Mount Sinai have designed an RNA-based strategy to activate dendritic cells that eradicated tumours and prevented their recurrence in mouse models of melanoma. However, these have not been as successful in clinical trials as hoped.

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Investment fuels AI-driven development of breakthrough genomic medicines

pharmaphorum

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

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Verge Genomics takes AI-sourced drug for ALS into clinic

pharmaphorum

Verge Genomics has joined a select group of biotechs who have taken a drug discovered and developed using artificial intelligence into human testing. The post Verge Genomics takes AI-sourced drug for ALS into clinic appeared first on.

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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. . Using RNA interference, researchers restored the normal biological function of FKTN in patient-derived cells, offering hope for a new therapy. . Modifying RNA function.

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Opinion: As it turns 40, the Orphan Drug Act for rare diseases needs a refresh

STAT News

The human genome was sequenced in 2003. It is now possible to treat diseases with gene therapy, antisense oligonucleotides, messenger RNA (mRNA), noncoding RNA (known as small interfering RNA, or siRNA), and other gene-based modalities. New ways of conducting clinical trials have also emerged.

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CAMP4 raises $100m to take lead RNA drugs into clinic

pharmaphorum

Armed with a $100 million second-round financing, CAMP4 Therapeutics is preparing to start the first clinical trial of a drug targeting regulatory RNA (regRNA) molecules that can be used to fine-tune the expression of genes. ” The post CAMP4 raises $100m to take lead RNA drugs into clinic appeared first on.

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