Remove Gene Remove Gene Therapy Remove Genome Remove RNA
article thumbnail

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.

RNA 246
article thumbnail

?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.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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.

RNA 130
article thumbnail

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. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

RNA 245
article thumbnail

Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

article thumbnail

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.

article thumbnail

The challenges and trends of cell & gene therapies 

Drug Discovery World

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.