article thumbnail

Gene expression delivery tool ‘slides’ instructions into cells

Drug Discovery World

If further studies verify the results, this could be a new method for targeting specific cell types for a variety of disorders that could be treated with gene therapies. Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

article thumbnail

bluebird seeks gene therapy trial restart after cancer scare

pharmaphorum

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Insiders

Sign Up for our Newsletter

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

article thumbnail

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood. This gene therapy will give the instructions to produce the enzyme arginine so that it can be hydrolysed to ornithine.

RNA 245
article thumbnail

This week in drug discovery (2-6 October)  

Drug Discovery World

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52
article thumbnail

Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

article thumbnail

Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. JMB: Rick Young’s publication shows that RNA binding TFs fine-tunes gene expression.

article thumbnail

Right on target: The shift to precision pharmaceuticals

Drug Discovery World

Artificial intelligence (AI), gene editing, synthetic biology and more, are beginning to prove their usefulness in drug design and development, taking therapies away from the one-size-fits-all approach, towards more definable targets that operate more effectively. There are myriad ways that this is being enabled.