NPR Health - Shots

FEBRUARY 20, 2023

The bottles of Starbucks' vanilla Frappuccinos may contain pieces of glass, the Food and Drug Administration said. Image credit: Gene J. Puskar/AP)

Containment 144

Containment Gene Drugs 144

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

Gene Therapy 97

Gene Therapy Gene Manufacturing Clinical Trials 97

Medical Xpress

MAY 9, 2023

Leucine-rich repeats containing 4 (LRRC4)—a gene abundantly found in the brain and located on human chromosome 7q31–32—plays a pivotal role in memory formation, autism, spinal cord injury, as well as in determining the malignant potential, development, and progression of glioblastoma (GB), an aggressive cancer involving the brain and/or spinal cord. (..)

Gene 100

Gene Containment Development Genetics 100

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

XTalks

APRIL 24, 2023

Awareness of rare diseases is growing, and with a better understanding of the pathophysiology of many rare diseases, innovative treatment options are emerging, like gene therapies that can treat the root cause of rare genetic diseases and potentially provide long-term symptom relief, or even a definitive cure.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

Pharmaceutical Technology

AUGUST 1, 2022

The mRNA-1273.222 vaccine containing the BA.4/5 Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Last month, the company announced the advancement of two bivalent candidates based on various population health security approaches in different countries.

Vaccine 289

Vaccine Vaccination Gene Therapy RNA 289

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). The FDA approval came after several delays and a narrow advisory committee vote of eight to six in favor of the gene therapy’s risk-benefit profile.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

Medical Xpress

MAY 16, 2023

Diet drinks often contain a mix of non-nutritive sweeteners that also enter the bloodstream after consumption. As a new pilot study shows, even dietary intake levels of saccharin, acesulfame-K and cyclamate are enough to modulate the copy rate of various genes in white blood cells.

Containment 117

Containment Gene Research 117

Pharmaceutical Technology

SEPTEMBER 8, 2022

The information contained within the download document is designed for pharmaceutical executives, developers, research scientists and associates, regulatory solutions consultants, and any other individual involved in API biologics production in the pharmaceutical industry.

Gene 100

Gene Marketing Vaccination Vaccine 100

Scienmag

MAY 28, 2021

Genetics toolkit targets less researched Culex mosquitoes, which transmit West Nile virus and avian malaria Credit: Gantz Lab, UC San Diego Since the onset of the CRISPR genetic editing revolution, scientists have been working to leverage the technology in the development of gene drives that target pathogen-spreading mosquitoes such as Anopheles and (..)

Containment 69

Containment Genetics Research Gene 69

Pharmaceutical Technology

DECEMBER 12, 2022

It leverages the new D-Domain binder of Arcellx and contains autologous T cells genetically modified for targeting multiple myeloma. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Editorial content is independently produced and follows the highest standards of journalistic integrity.

Development 264

Development Gene Therapy Clinical Trials Clinical Trials 264

Scienmag

APRIL 8, 2021

This full assembly may contain clues to ape and human divergence; certain immune, brain and heart disorders; and other biomedical questions Credit: Kendra Hoekzema The full assembly of human chromosome 8 is reported this week in Nature.

Gene 90

Gene DNA Containment Genetics 90

Scienmag

FEBRUARY 8, 2022

Defective mitochondria – the ‘batteries’ that power the cells of our bodies – could in future be repaired using gene-editing techniques. Our cells contain mitochondria, […].

Gene Editing 67

Gene Editing Gene Genome Genomics 67

Pharmaceutical Technology

AUGUST 15, 2022

The updated, Omicron-containing bivalent vaccine that acts on two coronavirus variants is indicated as a booster dose for active immunisation for the prevention of Covid-19 in people of this age group. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. 1 Omicron variant of concern.

Vaccine 214

Vaccine Vaccination Immune Response Clinical Trials 214

Medical Xpress

FEBRUARY 28, 2025

Dana-Farber investigators found that normally defunct viral genes that lie dormant in the human genome can be activated in the most common form of kidney cancer (clear cell renal cell carcinoma) and can end up triggering an immune response against the cancer.

Genome 89

Genome Genomics Immune Response DNA 89

Camargo

NOVEMBER 29, 2021

Essentially, a biologic is a product that is produced from living organisms or that contains components of living organisms. Gene and cellular therapies. These products can be derived from humans, animals, or microorganisms with biotechnology. Biologics include a wide range of products , including: Vaccines. Blood and blood components.

Development 180

Development Protein Production Hormones 180

Pharmaceutical Technology

SEPTEMBER 14, 2022

Successful TIL therapy depends on the infusion product containing tumour-reactive T cells that, on infusion, generate an anti-tumour immune response that causes disease regression. Synthetic gene libraries would then be used to generate reporter cells expressing neoantigens, and reporter T cells expressing potential neoantigen reactive TCRs.

Immune Response 246

Immune Response In-Vivo Production Manufacturing 246

Pharmaceutical Technology

JANUARY 31, 2023

It contains a single mRNA sequence encoding for a stabilised prefusion F glycoprotein. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The vaccine candidate is indicated to prevent RSV-associated lower respiratory tract disease (RSV-LRTD) in people aged 60 years or above.

Vaccine 182

Vaccine Vaccination Gene Therapy Gene 182

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 98

DNA Gene Gene Silencing Genome 98

XTalks

JANUARY 21, 2025

The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. The test collection kit includes a container for stool collection and a separate sampler for hemoglobin testing, requiring only a single stool sample to collect both.

FDA Approval 105

FDA Approval Manufacturing Development RNA 105

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

Gene Therapy 52

Gene Therapy Gene Research Genetics 52

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Scienmag

JUNE 7, 2021

Travellers abroad may pick up bacteria and other vectors containing genes conferring antimicrobial resistance which remain in the gut when returning to their home country, according to a study published in Genome Medicine.

Gene 40

Gene Bacteria Genome Genomics 40

Pharmaceutical Technology

FEBRUARY 15, 2023

The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells. Developing both in vivo and ex vivo gene editing medicines, Editas Medicine is primarily involved in the haematology and oncology therapy areas.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

Scienmag

MAY 11, 2022

NEW YORK CITY, May 11, 2022 – Credit: Memorial Sloan Kettering Cancer Center NEW YORK CITY, May 11, 2022 – Many cancers contain similar genetic “hotspots” — areas of DNA that are likely to be mutated. For example, more than 50% of cancers contain a mutation in a gene called TP53, often within a narrow DNA […].

Genetics 62

Genetics DNA Containment Gene 62

pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

Biobanking 52

Biobanking Gene Gene Silencing Drugs 52

Pharmaceutical Technology

JUNE 14, 2023

metres of supercoiled DNA contained within its nucleus. All that DNA is organised into hereditary units called genes, with humans having about 25,000 genes collectively known as the genome. It is these characteristics that make us uniquely human. Each human cell has 1.8

Genome Project 130

Genome Project Medicine Genome Genomics 130

Pharmaceutical Technology

MAY 12, 2023

The in vivo study, conducted in mice, involved a phosphatidylserine-containing nano-formulation of mRNA supplied by BioNTech, distinct from traditional lipid nanocrystals (LNCs). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. Matinas announced the news in a May 10 statement.

Vaccination 130

Vaccination Vaccine In-Vivo In-Vitro 130

The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris).

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

pharmaphorum

SEPTEMBER 14, 2020

While there may be just under 20,000 confirmed protein coding genes, it turns out that much of the genome outside of these genes is also important in regulating how the genome is controlled. For example, we know that not all genes are expressed in all tissues and that not all genes are expressed during all developmental stages.”.

Genome 119

Genome Genomics Medicine Gene 119

pharmaphorum

AUGUST 5, 2021

Whereas targets like receptors contain an obvious ligand-binding site for drug molecules to interact with, PPIs comprise a more complex structure. The interface is highly hydrophobic, hence any compounds containing water would be repelled. Current challenges and conventional approaches in targeting PPIs.

Protein 126

Protein Gene Bioassay DNA 126

Scienmag

MAY 7, 2021

The two meters of -stretched- DNA contained in human cells are continuously twisting and untwisting to give access to genetic information: when a gene is expressed to generate a protein, the two strands of DNA are separated to give access to all the machinery necessary for this expression, resulting in an excessive accumulation of coiling […]. (..)

DNA 62

DNA Regulation Genomics Genome 62

Roots Analysis

OCTOBER 16, 2023

DNA and Gene Cloning involves the isolation of a DNA sequence of any species ( often a gene ) and its insertion into a vector to enable growth without any alteration in the original DNA sequence. There are some motifs, such as hairpin loops which hinder the usability of a gene.

DNA 40

DNA Gene Protein In-Vitro 40

Pharmaceutical Technology

AUGUST 31, 2022

ILC Dover, specialists in the innovative design of biopharmaceutical manufacturing solutions, continuously monitors the market in order to provide in-demand equipment such as flexible containment solutions that reduce bottlenecks by providing closed, Grade-A, aseptic environments for fill and finish activities.

Packaging 130

Packaging Packaging Production Containment 130

Pharmaceutical Technology

OCTOBER 18, 2022

Gavi CEO Dr Seth Berkley said: “This agreement with Moderna represents a critical step for equitable access, helping COVAX adjust its portfolio to current demand and ensuring lower-income countries have access to variant-containing vaccines to use where appropriate.”. 5 Omicron-targeting bivalent booster vaccine for Covid-19.

Vaccine 130

Vaccine Vaccination Gene Therapy Containment 130

Pharmaceutical Technology

SEPTEMBER 15, 2022

The increase in the production of biological drugs such as vaccines, gene and cell therapies, and monoclonal antibodies, is increasing the volume of injectable drug products in the global healthcare market. Factors driving the demand for injectables.

Marketing 100

Marketing Containment Production Sales 100