Remove Gene Remove Genomics 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

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
Insiders

Sign Up for our Newsletter

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

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

Vertex signs licence deal with CRISPR Therapeutics for diabetes therapies

Pharmaceutical Technology

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

article thumbnail

New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing

pharmaphorum

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. Other initiatives include a Scientific Advisory Board that will help the firm establish its technology in RNA interference.

article thumbnail

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. That said, a lot has changed in science and drug development since 1983.

RNA 111
article thumbnail

New CRISPR-Based Tool Called PASTE Gene Editing Inserts Large DNA Sequences at Desired Sites

XTalks

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.