It's week 6 of 2020 and these are the biggest news stories from the life science industry. In this edition we bring news of a new gene correction therapy for Duchenne muscular dystrophy, the FDA approves the first drug for peanut allergies, the results and analysis are in from the first US human safety trial with CRISPR, and a newly discovered receptor in T-cells may lead to cell-based immunotherapies that are more universal than those currently in existence. You’ll find these stories and more are below.
First Peanut Allergy Drug Gets FDA Thumbs Up
Peanut allergies are one of the most common causes of severe allergy attacks and to date, there is no cure for this allergy. An estimated 1.2 million children and teens in the U.S. have peanut allergies. The drug that the FDA have approved is the first drug for the treatment of peanut allergies in children aged four and older. Although patients must continue to avoid peanuts while taking this medication, in the case of accidental peanut exposure, the drug may mitigate the harmful allergic reactions.
First treatment for pain using human stem cells a success
Researchers at the University of Sydney have used human stem cells to make pain-killing neurons that provide lasting relief in mice, without side effects, in a single treatment. The research now moves on and if it gets to the stage of human testing, and if the tests are successful in humans, it could be a major breakthrough in the development of new non-opioid, non-addictive pain management strategies for patients.
Newly Discovered T-cell Receptor May Lead to More ‘Universal’ Cancer Therapy
A newly discovered receptor in T-cells makes these cells able to recognize and eliminate several cancer types, without harming healthy cells, through an unconventional immunological mechanism that isn’t fully understood yet. This finding may pave the way for cell-based immunotherapies that are more universal than those currently in existence but more research will be necessary before this finding is translated into use in people.
New gene correction therapy for Duchenne muscular dystrophy
Duchenne type muscular dystrophy (DMD) is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of 12 and reducing life expectancy. Researchers have developed a gene therapy that may provide permanent relief for those suffering from DMD.
French researchers develop a “yarn” of human skin
A team of researchers at the French National Institute of Health and Medical Research in Bordeaux have grown yarn from human skin cells that they call a "human textile". Further research is needed but they hope to develop the material into a viable product used by medical professionals. This material could be used as a simple suture to close a wound or could be assembled into fully biological, human, tissue-engineered vascular grafts that have high mechanical strength and are implantable.
Crispr'd Cells Show Promise in First US Human Safety Trial
Researchers from the University of Pennsylvania and Stanford have revealed the first published report describing the first US human safety trial of CRISPR which was carried out on a cancer patient population. The take home point is that using genetically-edited cells to supercharge the immune system caused no adverse effects in cancer patients. It's too soon to tell if it can be a cure.
New platform for composing genetic programs in mammalian cells
A new synthetic biology toolkit developed at Northwestern University will help researchers design mammalian cells with new functionalities. The toolkit, called the Composable Mammalian Elements of Transcription (COMET), includes an ensemble of synthetic transcription factors and promoters that enable the design and tuning of gene expression programs in a way not previously possible. The result could be new therapies for difficult-to-treat diseases, like cancer.