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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