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Overcoming Scale-Up Challenges in Gene Therapy Manufacturing

Gene therapies can scale economically, but not just with practices adapted from traditional biologics. According to Avantor, gene therapies pose unique material, workflow, and partnering challenges.

Liquid Biopsies Generate Ample Insights from Sparse Clues

New liquid biopsy tools such as target enrichment and duplex sequencing can help investigators derive meaning from the subtly variable DNA fragments shed in trace amounts by healthy and diseased tissues.

Monte Carlo Simulations to Democratize COVID-19 Policies*

The COVID-19 pandemic is fraught with questions. How should scarce resources be allocated? Should restrictions be loosened or tightened? To find the best answers, try new forms of pandemic modeling.

Cryo-EM Captures CRISPR-Cas9 Base Editor in Action

Researchers have obtained the first 3D structure of CRISPR-Cas9 base editor. The high resolution cryo-EM structure is of the newest version of adenine base editor, ABE8e, trapped in the catalytic conformation. The structure, together with kinetic data, explains how ABE8e edits DNA bases and could inform future base-editor design. This work provides a roadmap to make base editors more versatile and controllable for use in patients.

Vaccine Effectiveness Could be Boosted in the Elderly

Annual flu vaccinations tends to be less effective in the elderly than it does in young people, because as we age our immune systems get weaker, so the response to vaccination isn’t as robust. Scientists at Cincinnati Children's Hospital have found that this immune system dampening may be due to active suppression by a group of immune cells known as Thf10 cells, which produce interleukin-10. The team’s results in mice suggest that it may be possible to boost vaccine effectiveness in the elderly by transiently managing Tfh 10 cells in a way that avoids disrupting the useful roles the cells play in fending off other types of disease.

Malaria Transport Channel Identified Connecting Parasite to Red Blood Cell

Researchers at the National Institutes of Health and other institutions have discovered another set of pore-like holes, or channels, traversing the membrane-bound sac that encloses the deadliest malaria parasite as it infects red blood cells.

Tumors Instruct T Cells to Produce Steroids and Turn Themselves Off

Mouse tumor T cells produce immunosuppressive steroids, even though T cells from healthy mice do not. This observation prompted scientists to prevent the T cells from producing the steroids, either by removing a key steroid-producing gene, or by switching it off with a drug. Tumor growth was dramatically reduced, suggesting that steroidogenesis in T cells is a target for cancer immunotherapy.

Antioxidants Gone Bad: When the Microbiome Promotes Cancer

HU researchers found that certain cancer mutations are not necessarily bad actors, in and of themselves. In fact, in certain micro-environments like the gut, these mutations can actually help the body to fight cancer, not spread it. However, if the gut microbiome produces high levels of metabolites then it acts as a particularly hospitable environment to mutated genes and will accelerate the growth of bowel cancers.

ENCODE Project Generates Registry of More Than One Million Human and Mouse Gene Regulatory Elements

An online registry of over 1,200,000 human and mouse candidate functional elements that regulate genes has been generated as part of the third phase of the global ENCyclopedia of DNA Elements (ENCODE) project. With completion of its latest phase, the project has added millions of candidate DNA "switches" from the human and mouse genomes that appear to regulate when and where genes are turned on.

Wacker Invests in Its Amsterdam Biotech Site

New construction projects will contribute to preparing the plant to produce new classes of pDNA and mRNA-based vaccines.

Gene Therapy: What’s Up With AAV?

Join us on this GEN Live webinar, sponsored by PerkinElmer, as we discuss the state of gene therapy, the rise of AAV, and the safety issues that need to be addressed going forward.

Artificial Intelligence Classifies Cancer Types, Predicts Genetic Alterations

Artificial Intelligence can be used to quickly and accurately screen cancer patient biopsies for certain genetic alterations that may inform their treatment options and likelihood to respond to specific therapies. Researchers have created a deep learning algorithm that infers molecular alterations directly from routine histology images across multiple common tumor types. If this model was validated and deployed at scale, it could dramatically improve the speed of molecular diagnosis across many cancers.

SARS-CoV-2 Lurked for Decades Where Others Like It Lurk Still

SARS-CoV-2 studies suggest that investments in pathogen surveillance and resource management offer great returns: less human suffering and fewer material losses due to unrestrained pandemics. One study reconstructed the evolutionary history of SARS-CoV-2 and concluded that the virus had been lying in wait for decades, and that viruses of similar lineage may already circulate in bat populations, lending urgency to prevention efforts. Another study indicated that preventing a pandemic like COVID-19 could be 500 times less expensive that combatting it.

Stem Cells Plus Glaucoma Drug Promote Diabetic Wound Healing

Scientists at the University of California-Davis demonstrated how a drug commonly used to treat glaucoma improved healing and decreased inflammation in the wounds of diabetic mice by as much as 75 percent over the control groups.

Diabetes Drug Candidate Could Offer “Distinct and Innovative” Treatment For Type 1 and Type 2 Disease

Researchers at the University of Alabama at Birmingham, and Southern Research, identified a drug candidate that they claim could potentially treat both 1 and type 2 diabetes. The small molecule drug, designated SRI-37330, inhibits the expression of a protein known as TXNIP—which the team had previously identified as a top glucose-induced gene—in both mouse and human islets. Results from preclinical studies suggested that SRI-37330 acts on pancreatic islet cells that produce glucagon and insulin, and reduced excess glucose production by the liver.

Hair Regrowth Therapies May Reside with MicroRNAs

Investigators at North Carolina State University have been able to identify a microRNA (miRNA) that could promote hair regeneration. This miRNA—miR-218-5p—plays a vital role in regulating the pathway involved in follicle regeneration and could be a candidate for future drug development.

Pfizer, BioNTech Begin Phase II/III Trial of COVID-19 Vaccine, Switching Lead Candidate

The companies said preliminary Phase I/II data from nearly 120 patients showed a more favorable overall tolerability profile than BNT162b1, with no serious adverse events and generally mild to moderate and transient systemic events such as fever, fatigue, and chills.

Democratizing the Availability of Off-the-Shelf Natural Killer Cells

Artiva Biotherapeutics views as its critical and necessary mission making cancer cell therapies available to all.

Utilizing Machine Learning for Better Bioprocess Development

Novel technology could revolutionize production if biopharma industry can improve the quality of its manufacturing.

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