Advanced Therapy, Cell Therapy and Gene Therapy News
FDA Places Partial Clinical Hold on Some Trials of Novartis Gene Therapy
Oct. 29, 2019 Novartis acknowledged today that the FDA has imposed a partial hold on clinical trials for intrathecal administration of AVXS-101, the gene therapy that won the FDA’s first approval for treating some forms of spinal muscular atrophy (SMA)...MorphoSys, Galapagos Halt Development of Atopic Dermatitis Candidate Licensed to Novartis
Oct. 29, 2019 MorphoSys and Galapagos said they are halting development of MOR106—an atopic dermatitis candidate the companies licensed to Novartis last year for potentially more than $1 billion...Biomarkers Linked to Specific Behaviors in Depression Identified for Precision Medicine
Oct. 29, 2019 Researchers in the United States and Switzerland have identified neural subcircuits and genetic biomarkers in mice that are linked...Peripheral Vision: Cygnal Looks Beyond the CNS to Develop Drugs
Oct. 29, 2019 A spinout of Flagship Pioneering has emerged from stealth mode to develop drugs based on pathways and targets the company...Protein-Scaffolding Discovery Shines Light on DNA Repair Mechanisms
Oct. 29, 2019 Scientists at the University of Copenhagen have discovered how some types of proteins stabilize damaged DNA and thereby preserve DNA function and integrity...Microprotein Could Point to New Therapeutic Targets for Diverse Human Diseases
Oct. 28, 2019 The microprotein PIGBOS (magenta) shown sitting on the outer membranes of mitochondria (green), where it is poised to make contact with other organelles in the cell. [Salk Institute/Waitt Advanced Biophotonics Core Facility]...Deleting a Liver Enzyme Lowers the Health Risk of Sweet Treats (at Least in Mice)
Oct. 24, 2019 Hepatic insulin resistance, caused by diets high in sugar and fat, can lead to type 2 diabetes. Researchers found that the Elovl6 gene plays a key...Novel Inexpensive Technique Can Identify Aggressive Tumors
Oct. 21, 2019 Researchers at the Karolinska Institutet in Sweden say they have developed a new cheap method that can identify highly heterogeneous tumors that tend to be aggressive, and therefore need to be treated more aggressively...Hyper-Long Telomeres Give Non-Genetically Modified Mice Longer, Healthier Lives
Oct. 18, 2019 Scientists at the Spanish National Cancer Research Centre (CNIO) generated the first mice that have super-long telomeres in every one of their body cells. Studies with the animals showed...Synthesis of Ginkgo Biloba Compound Opens Door to Drug Design
Oct. 18, 2019 The Ginkgo tree’s metabolite, bilobalide, is widely ingested by humans despite its effect on the mammalian central nervous system not being well understood. Although there are many hypotheses...Male and Female Mouse Brains Have Different Neurons
Oct. 18, 2019 The differences between the male and female minds may actually have its roots in neurobiology, at least for mice, as a new study from investigators at Caltech has discovered rare brain cell types that are unique to male mice and other types that are unique to female mice...Metabolic Mitochondria Dysfunction May Be Primary Cause of Alzheimer’s
Oct. 18, 2019 A team of researchers led by Yale-NUS College says it has found evidence that metabolic dysfunction is a primary cause of Alzheimer’s disease...Danaher Selling Off Some Businesses to Sartorius for $750M, Tying the Deal to GE Biopharma Acquisition
Oct. 21, 2019 The 8-channel Octet system (pictured) of Danaher’s FortéBio label-free biomolecular characterization business performs quantitation of 96 samples in 32 minutes and kinetic screening of 64 samples in 1.5 hours. Danaher has agreed to sell FortéBio, as well as its chromatography hardware and resins....When Added to Gene Therapy, Plant-Based Compound May Enable Faster, More Effective Treatments
Oct. 17, 2019 Today's standard process for administering gene therapy is expensive and time-consuming -- a result of the many steps required to deliver the healthy....Five Prime Eliminates 70 Jobs in Restructuring
Oct. 11, 2019 Five Prime Therapeutics will eliminate approximately 70 jobs—about 40% of its workforce—and shrink its facilities as part of a restructuring by Chairman and interim CEO William Ringo that is intended to conserve cash without impacting or delaying its clinical programs....In-Office Gene Therapy for Wet Age-Related Macular Degeneration Is Coming
Oct. 11, 2019 Gene therapy is showing promise for one of the most common causes of ...Schizophrenia Memory Deficits Reversed in Adult Mice
Oct. 10, 2019 An international research team headed by scientists at Columbia University restored normal working memory in a mouse model of schizophrenia (SCZ). The achievement, demonstrated using both genetic, and pharmacological approaches, effectively reversed in adult mice a core symptom of the...New Method Visualizes a Population of Neurons in Awake, Behaving Mice
Oct. 10, 2019 Researchers at MIT and Boston University have teamed up to achieve the longstanding goal of imaging membrane voltage across a population of individual neurons in an awake, behaving mammal. Using a genetically encoded fluorescent voltage indicator, SomArchon, that lights...Let’s Throw Sand in the Tuberculosis Pathogen’s Nanogears
Oct. 10, 2019 Tuberculosis is an infernal machine, and it keeps spreading thanks to the operations of many tiny infernal machines—not the pathogens, mind you. Look more closely. Each tiny Mycobacterium tuberculosis pathogen is studded with even tinier machines, secretion machines...Breast Cell Differentiation Mapped to Help Understand How Cancer Develops
Oct. 9, 2019 Breast cancer is one of the most common forms of cancer, and some subtypes are among the most difficult of all cancers to treat. The involvement of many different cells makes targeting such tumors challenging...Mitochondria Shown to Regulate Brain’s CA2 Subregion, Highlighting a Potential Target for Neuro Disorders
Oct. 9, 2019 A team of researchers has published evidence showing in mice that mitochondria-associated pathways in cell bodies and dendrites within...Cell Death Blocker Could Lead to New Ways of Minimizing Tissue Damage and Preserving Transplant Organs
Oct. 8, 2019 Scientists in Australia have developed a small molecule compound that can prevent a form of cell death known as apoptosis, and which could ultimately lead to new treatments that minimize cellular...Hornless Genome-Edited Bull Passes Trait to Offspring
Oct. 8, 2019 Three years ago, a genome edit (POLLED) was made in two male dairy bulls using transcription activator-like effector nucleases (TALENs) to introduce a hornless phenotype. A new study...Pfizer to Develop Akcea/Ionis Antisense Candidate for Up to $1.5B
Oct. 7, 2019 Pfizer has licensed exclusive global rights to Akcea Therapeutics’ Phase II cardiovascular and metabolic disease candidate AKCEA-ANGPTL3-LRx, through an agreement that could generate...IBD and Infection Control Linked with Disruption to Gut Immune Cell Clock Genes
Oct. 7, 2019 Two independent sets of research published in Science Immunology have demonstrated how a subset of immune cells in the gut, known as group 3 innate lymphoid cells (ILC3), are tuned to the body’s daily...Brain Tumors Modeled Using Tunable Extracellular Matrix
Oct. 7, 2019 The progression of brain tumors cannot be staged properly without adequate stagecraft—stagecraft so thorough and technically adept that the distinction between players and props becomes...Researchers Create New Viral Vector for Improved Gene Therapy in Sickle Cell Disease
Oct. 2, 2019 Researchers have developed a new and improved viral vector -- a virus-based vehicle that delivers therapeutic genes -- for use in gene therapy for...Researchers Advance Search for Safer, Easier Way to Deliver Vision-Saving Gene Therapy
Sep. 30, 2019 In experiments with rats, pigs and monkeys, researchers have developed a way to deliver sight-saving gene therapy to the retina. If proved safe and...IsoPlexis and Miltenyi Report on Synergistic CAR-T Cell Technologies
Sep. 30, 2019 IsoPlexis, which recently received an NCI grant, reports that it has published a technology note with Miltenyi Biotec in a co-marketing agreement. The tech...Molecular Analysis of Elephantiasis Post-Treatment Adverse Events
Sep. 27, 2019 Close to a billion people worldwide remain threatened by lymphatic filariasis, a neglected tropical disease commonly referred to as elephantiasis. This parasitic infection, which is caused by a...Prostate Cancer Screens Might Turn to Biomarkers in Semen
Sep. 27, 2019 Semen could be considered a liquid biopsy of the organs of male reproductive system—specifically, of the prostate...GSK Completes $120M Manufacturing Expansion in Upper Merion, PA
Sep. 24, 2019 GlaxoSmithKline (GSK) announced that it has completed a $120 million expansion of its manufacturing site in the Philadelphia suburb of Upper Merion, PA, adding that the upgrade will enable it to accelerate the development...Critical Cellular Player Controlling HIV Reproduction in Immune Cells Identified
Sep. 26, 2019 Using genetic sequencing, scientists at the University of California, San Diego (UCSD), School of Medicine say they have identified a principal cellular player controlling HIV reproduction...Wacky Worms Thrive in Extreme Environment
Sep. 27, 2019 Mono Lake, located in the Eastern Sierras of California, is three times as salty as the ocean and has an alkaline...Non-Viral Gene Therapy to Speed Up Cancer Research
Sep. 24, 2019 A new treatment method promises to speed up gene therapy research and could bring new, patient friendly cancer treatments to market...World's First Gene Therapy for Glycogen Storage Disease Produces Remarkable Results
September 20, 2019 The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels...Circular RNA Plays Key Role in Tissue Repair after a Heart Attack
Sep. 17, 2019 Scientists at the Lewis Katz School of Medicine at Temple University (LKSOM) and colleagues describe, reportedly for the first time, a circular RNA that fills a critical role in tissue repair after a heart attack, due to its ability to bind harmful molecules, including microRNAs and proteins. Their study (“Circular RNA CircFndc3b modulates cardiac repair after myocardial infarction via FUS/VEGF-A axis”) appears online in Nature Communications....HIV Drug Delivery Made Easier with Long-Acting Implant
September 23, 2019 Adherence to a strict HIV regimen is an essential part of effective HIV treatment. Medication should be taken every day, at specific times of the day, and with or without certain kinds of food. Making this process easier would, undoubtedly, increase medication adherence and improve the efficacy of current HIV treatment plans....Anticancer siRNAs Delivered to Cells Using MOF Nanoparticles
Sep. 17, 2019 Scientists from the University of Cambridge have developed a highly porous type of nanoparticle known as a metal-organic framework (MOF) that can deliver disease gene targeting small interfering RNAs (siRNAs)...IL-6 and IL-2 Interplay Shown to Affect T Follicular Helper Cell Development
Sep. 17, 2019 Researchers at the University of Alabama at Birmingham (UAB) department of medicine published a study (“Inhibition of IL-2 responsiveness by IL-6 is required for the generation of GC-TFH cells”) ...Common Cold Foiled If It Cannot Exploit Host Protein
Sep. 17, 2019 Temporarily disabling a host protein can effectively deny the common cold a foothold it needs to replicate inside host cells.ADCK3/COQ8A: the choice target of the UbiB protein kinase-like family
Sep. 16, 2019 UbiB kinase-like proteins exist in archaea, bacteria and eukaryotes, and comprise ~25% of all microbial protein kinase-like (PKL) enzymes. The founding member of the UbiB family, UbiB from Escherichia coli...A probe for every protein
Sep. 11, 2019 On a summer day in 2018, 54 industry and academic researchers gathered at Bayer’s headquarters in Germany to discuss an old and bold idea: what would it take to...Fight-or-Flight Response Shown to be Driven by Bone Hormone, Not Adrenaline
Sep. 13, 2019 When faced with situations of real or perceived sudden danger the body sets in motion a series of physiological changes in preparation either for a fight or to take flight. This acute stress response (ASR) includes ...Gene Therapy Repairs Stroke Damage and Improves Motor Function
Sep. 13, 2019 A new gene therapy turns glial cells into neurons, repairing damage that results from stroke and significantly improving motor function in mice, according to ...BioNTech Plans Pipeline, Platform Expansion with IPO Proceeds
Sep. 13, 2019 BioNTech, the German developer of individualized treatments for cancer and other diseases that has raised more than $1 billion in capital as a privately-held company, has disclosed plans to go public by raising $100 million through an initial public offering.AI Improves Diagnosis of Head and Neck Cancers Using DNA Methylation
Sep. 13, 2019 Diagnosing head and neck cancers is challenging. It is particularly difficult to distinguish pulmonary metastases of head and neck squamous cell carcinoma (HNSC) from primary lung squamous cell carcinomas (LUSCs). ...Nuclear Glycogen Linked to Non-Small Cell Lung Cancer
Sep. 13, 2019 Scientists at the University of Kentucky Markey Cancer Center say they have made a discovery that solves a mystery and identified a potentially novel avenue in preclinical models to treat non-small cell lung cancers.Tiny Bubbles in Our Body Could Fight Cancer Better Than Chemo
Sep. 13, 2019 Healthy cells in our body release nano-sized bubbles that transfer genetic material such as DNA and RNA to other cells. It's your DNA that stores the important information necessary for RNA to ...Pancreatic Cancer Drug Resistance Pathways Identified
Sep 10, 2019 | 8:47 am Scientists at Cold Spring Harbor Laboratory (CSHL) say they have found a way to tackle the problem of drug resistance and stop the growth of pancreatic ductal adenocarcinoma (PDA) tumors in mice. Their findings (“Identification of Resistance Pathways Specific to Malignancy Using Organoid Models of Pancreatic Cancer”) are published ...2019 Lasker Award Winners: B and T Cell Discoverers, Cancer Therapy Developers, and Gavi
Sep 10, 2019 | 8:47 am The Albert and Mary Lasker Foundation today named five researchers and an organization that promotes vaccination of children in underdeveloped countries as winners of its annual awards for work ...Potential MS Therapy Promotes Myelin Repair in Mice
Sep 9, 2019 | 8:47 am Scientists at Oregon Health & Science University (OHSU) have developed a compound that promotes repair of the nerve cell-protective myelin sheath that is damaged in diseases such as multiple sclerosis (MS)...Sumitomo Dainippon Commits $3B toward Alliance with Roivant
Sep 9, 2019 | 8:47 am Sumitomo Dainippon Pharma has agreed to acquire five of Roivant Sciences’ 20 companies or “vants”—as well as options to buy up to six more vants, and access to Roivant ...Success of Gene Therapy for a Form of Inherited Blindness Depends on Timing
Sep 9, 2019 | 8:47 am An FDA-approved gene therapy for Leber congenital amaurosis, an inherited vision disorder with a childhood onset and progressive nature, has improved patients' sight. But new research on the blinding ...Gene Therapy Reduces Obesity and Reverses Type 2 Diabetes in Mice
Aug. 29, 2019 | 8:47 am
The obesity epidemic affects nearly half a billion people worldwide, many of them children. Obesity-related diseases including heart disease, stroke, type 2 diabetes, and cancer are a leading cause ...
Cancer gene therapy:Dual-vector prodrug activator gene therapy displays synergistic cytocidal effects on cancer
Sep 9, 2019 | 9:29 am
Retroviral replicating vectors (RRVs) has been proved as an excellent gene therapy vector to achieve efficient gene transduction. To date, more than 499 clinical trials have been carried out using lentivirus vectors for gene delivery and promising gene therapy outcomes have been achieved in a variety of diseases, mostly in cancer. Today, a dual-vector prodrug activator gene therapy has been designed by one research team at the University of Miami to use two different RRVs carrying different prodrug activator genes, which displays synergistic cytocidal effects on different cancer models and indicates the potential utility of dual-vector gene therapy in the future.
Nature Communication: No immunogenicity! Can the spring of AAV mediated gene therapy be far behind?
Sep 9, 2019 | 9:29 am
Recombinant adeno-associated virus (AAV) vectors exhibit promising outcomes on gene therapy with its durable expression in the targeting tissues. Even so, in most cases, AAV immunogenicity still represents a major limitation for its clinical trials. Recently, a research team from Sorbonne Université and INSERM U974 (France) has employed an effective means to avoid AAV immunogenicity, which makes AAV safer and provide reference for its further clinical trial.
Targeting a Blood Stem Cell Subset Shows Lasting, Therapeutically Relevant Gene Editing
July 31, 2019 | 9:29 am
Researchers have used CRISPR-Cas9 to edit long-lived blood stem cells to reverse the clinical symptoms observed with several ...
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