The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing. Instead of snipping the genome, base editing lets for edits of individual letters in a genetic sequence.
Beam employs three approaches to deliver its genetic medicines to cells. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs).
GuideTx will aid Beam in developing LNP-based medicines. The startup spun out of Georgia Tech in 2018 and is based on James Dahlman’s research, a professor in the university’s department of biomedical engineering and school of medicine. The GuideTx technology screens for lipid nanoparticles inside the body that can be used to deliver RNA to cells throughout the body.
GRAIL and Quest collaborate for the cancer blood test
Cancer detection company GRAIL has announced a collaboration with Quest Diagnostics that sees Quest proffering phlebotomy services to bolster GRAIL’s multi-cancer early detection blood test Galleri™.
Quest runs 2,200 patient service centers and recruits 5,000 mobile phlebotomists who have experience and expertise in offering phlebotomy services for at-home visits. Through this pact with GRAIL, Quest’s phlebotomy network will benefit the Galleri test in blood specimen collection throughout the U.S. once Galleri becomes accessible in the second quarter of 2021. Earlier, the prescription-only blood test will only be available through partner health systems, medical practices, and self-insured employers.
Dr. Joshua Ofman, GRAIL’s chief medical officer and head of external affairs, said in a statement that the majority of cancers go undetected until too late when outcomes are often deadly, and they believe Galleri could offer a unique and potentially life-saving solution by finding multiple types of cancers earlier. Working with Quest will help them support convenient access to blood collection for patients and healthcare providers.
A study published in Cancer Epidemiology, Biomarkers & Prevention last year, December, estimated that the annual use of Galleri, among other multi-cancer early detection blood tests, could decrease late-stage cancer diagnoses by more than 50% in people aged 50 to 79 in the U.S. The study hints that this reduction could translate to a 39% reduction in five-year cancer deaths for patients who had their cancer detected earlier. Overall, this could result in a 26% overall reduction in all five-year cancer deaths.
Brain organoids grown in the lab mimic infant’s brains
A new study from UCLA and Stanford University researchers observes that three-dimensional human stem cell-derived brain organoids can mature in a strikingly similar way to human brain development.
The senior authors, Dr. Daniel Geschwind of UCLA and Dr. Sergiu Pasca of Stanford University, conducted an extensive genetic analysis of organoids grown for up to 20 months in a lab dish per the new study, which was published in Nature Neuroscience. They discovered that these 3D organoids follow an internal clock that controls their maturation in sync with the human development timeline.
Human brain organoids are developed using induced pluripotent stem cells, aka iPS cells derived from skin or blood cells, which have been reprogrammed back to an embryonic stem cell-like state, enabling scientists to develop any cell type.
These iPS cells are then subjected to a specialized mix of chemicals, which influences them to develop a certain brain region. With time and the appropriate conditions, the cells self-organize to develop 3D structures that faithfully replicate various human brain development aspects.
Human stem cell-derived organoids have the potential to transform the practice of medicine by conferring researchers unprecedented insights into how complex organs, including the brain, develop and respond to disease.
Increasing the immune system’s T cells to enhance cancer-destroying capabilities
Combining immuno-oncology treatments such as checkpoint inhibitors with other cancer-killing drugs is considered a remarkable strategy for enhancing the response rate to immunotherapy. A team of Canadian researchers is suggesting a new combination strategy for enhancing the response of the immune system’s T cells to immuno-oncology drugs, and it comprises epigenetics.
A team led by the Princess Margaret Cancer Centre in Toronto found that chemotherapy drugs known as DNA hypomethylating agents directly influenced the anti-tumor response of a specific type of T cell in samples from patients with several tumor types. Two genes were activated to improve the T cells’ ability to eradicate cancer cells, as reported in the journal Molecular Cell.
DNA hypomethylating agents are epigenetic drugs that eliminate specific chemical tags on DNA, hence turning specific genes on and off and changing the cells’ function.
The researchers had previously discovered that when mouse tumors were treated with epigenetic drugs, there was an increase in T cells infiltrating cancer. If they eliminated the T cells, the therapy stopped working.
They decided to evaluate the phenomenon further using samples from healthy people and patients with melanoma and breast, ovarian, or colon cancer. Again they discovered that DNA hypomethylating agents improved the ability of T cells to eliminate cancer.
