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Gatehouse Bio and AstraZeneca to develop RNA therapeutics for heart failure

Pharmaceutical Technology

US-based biotech company Gatehouse Bio is extending its collaboration with AstraZeneca for developing RNA therapeutics to treat heart failure with preserved ejection fraction (HFpEF). It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored.

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Ochre Bio raises funds to develop RNA therapies for liver diseases

Pharmaceutical Technology

Biotechnology company Ochre Bio has raised $30m in a Series A financing round to develop RNA therapies for chronic liver diseases. It also intends to turn the insights from its research into RNA-based drug candidates, and they will be tested in human livers at the company’s recently opened ‘Liver ICUs’ in the US.

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?5 Things You Need to Know from Our Recent Gene Therapy Webinar

Worldwide Clinical Trials

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Around 40% of clinical holds are for gene therapy programs.

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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. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.

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

STAT News

The act also created an industry that didn’t exist in the United States before its enactment, enabling the formation of companies to develop and commercialize therapies for rare diseases. The human genome was sequenced in 2003. That said, a lot has changed in science and drug development since 1983.

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Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics

The Pharma Data

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.