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.

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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.

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Gene Therapy Gene Protein FDA Approval 98

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)

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Containment Gene Drugs 144

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, […].

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Gene Editing Gene Genome Genomics 67

Drug Discovery World

DECEMBER 5, 2022

Some have even been successfully harnessed as gene therapy vectors for the treatment of genetic diseases. . In the study, researchers used ‘fossilised’ virus DNA sequences contained within animal genomes to reconstruct the long-term evolutionary history of parvoviruses. Parvovirus gene therapy is a cutting-edge biomedical technology.

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Gene Therapy Gene Genetic Disease Genetics 52

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 (..)

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Containment Genetics Research Gene 69

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.

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Containment Gene Research 119

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.

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Gene Bacteria Genome Genomics 40

Drug Discovery World

JANUARY 23, 2024

Scientists at the King Abdullah University of Science and Technology (KAUST) in Thuwal, Saudi Arabia used the KAUST Metagenomic Analysis Platform (KMAP) to analyse massive amounts of sequencing data to release Global Ocean Gene Catalog 1.0.

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DNA Drugs Gene Scientist 103

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.

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Gene DNA Containment Genetics 90

Drug Discovery World

JANUARY 27, 2023

Exclusive CGT Guide This issue contains an exclusive Cell & Gene Therapy Guide covering the latest innovations and challenges within the sector.

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Gene Therapy Gene Containment Protein 98

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).

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Gene Therapy Gene Manufacturing Production 52

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

Drug Discovery World

OCTOBER 25, 2023

bit.bio has launched the first offering from its new product range ioCRISPR-Ready Cells, which allow research and drug discovery scientists to knockout any gene of interest in glutamatergic neurons. ioCells products are reprogrammed from human induced pluripotent stem cells (iPSCs) by opti-ox, bit.bio’s precision cell programming technology.

Genome 52

Genome Genomics Gene Scientist 52

Drug Discovery World

OCTOBER 19, 2022

A new blood donor centre in Glasgow, Scotland has been opened to support cell and gene therapy research. . The donor center will be used primarily for producing LEUKOMAX leukopaks, which contain concentrated white blood cells and are used for cell and gene therapy research. .

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Gene Therapy Life Science Gene Containment 52

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.

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Vaccination Vaccine Gene Therapy RNA 289

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 […]. (..)

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DNA Regulation Genome Genomics 62

Drug Discovery World

MARCH 21, 2023

UK researchers, from the Wellcome Sanger Institute and collaborators, have used human stem cells and neurons to investigate what features influence how well CRISPR activation works for different sets of genes. CRISPR activation (CRISPRa) is a type of CRISPR gene editing that is used to overexpress certain genes.

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Research Gene Gene Editing Genome 52

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.

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Development Gene Therapy Clinical Trials Clinical Trials 264

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.

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Vaccination Vaccine Immune Response Clinical Trials 214

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.

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Vaccination Vaccine In-Vivo In-Vitro 130

Scienmag

SEPTEMBER 14, 2021

The term ‘DNA’ immediately calls to mind the double-stranded helix that contains all our genetic information. But the individual units of its two strands are pairs of molecules bonded with each other in a selective, complementary fashion.

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DNA Gene Containment Genetics 40

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.

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Drug Discovery World

MAY 5, 2023

The technology underlying Exacis’ platform uses mRNA cell reprogramming and mRNA gene editing to create engineered iPSC-derived cells for use in the development of cancer therapies that can target nearly any cancer antigen. The post Eterna acquires Exacis’ allogeneic immuno-oncology platform appeared first on Drug Discovery World (DDW).

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Gene Editing Engineer DNA Life Science 52

The Pharma Data

OCTOBER 26, 2020

DALLAS–( BUSINESS WIRE )– Taysha Gene Therapies Inc. Food and Drug Administration (FDA) for TSHA-104, an AAV9-based gene therapy in development for SURF1-associated Leigh syndrome. Obtaining these key designations highlights our commitment to developing a gene therapy for the treatment of SURF1 deficiency.”.

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Gene Therapy Gene Drugs Production 52

Pharmaceutical Technology

FEBRUARY 8, 2023

On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.

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Gene Therapy Gene Trials Clinical Trials 264

Drug Discovery World

JANUARY 11, 2024

Successful industry/academia collaboration Fewer than 5% of rare diseases have approved therapies and most of these treatments use gene therapy. Until recently, gene therapy employed modified viruses to bring the therapeutic gene to the disease cells, but these viral systems can cause severe adverse effects.

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Research Gene Therapy RNA Gene 59

Drug Discovery World

JANUARY 9, 2023

Using gene engineering, we are repurposing cancer cells to develop a therapeutic that kills tumour cells and stimulates the immune system to both destroy primary tumors and prevent cancer.” . CRISPR-Cas9 gene editing . Living tumour cells will travel long distances across the brain to return to the site of their fellow tumour cells.

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Scientist Gene Editing Gene Engineer 52

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.

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In-Vivo Genome Genomics Gene Editing 162

BioTech 365

FEBRUARY 8, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Complete characterization of the full mitochondrial kinome.The human cellular kinome contains over 500 kinases, accounting for almost 2% of all our genes.

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Containment Gene Research 40

The Pharma Data

JANUARY 18, 2021

SLC13A5 is a form of infantile epilepsy caused by mutations in the SLC13A5 gene. The disorder is an autosomal recessive disorder, so two copies of the mutated gene must be inherited to affect an infant. About Taysha Gene Therapies. Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease.

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Gene Therapy Gene Drugs Production 52

Scienmag

MAY 18, 2021

(Boston)–Researchers from Boston University School of Medicine (BUSM) report the formation of human cells containing a green fluorescent protein or GFP (one of the most important proteins in biology and fluorescence imaging) genetically fused with two interferon stimulated genes (ISGs), namely Viperin and ISG15.

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Immune Response Reagent Research Protein 41

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. IPs containing engineered genetic materials may pose risks potentially not anticipated by the clinic’s standard infection control policies.

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Engineer Genetics Clinical Research Research 52

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.

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Vaccination Vaccine Gene Therapy Containment 130

Drug Discovery World

AUGUST 14, 2023

Lung-I Cheng, Vice President and Head of Cell & Gene Therapy Service Line at AmerisourceBergen, and Cori Gorman, Senior Director of CMC and Regulatory Affairs at Biopharma Excellence, offer advice on navigating the different and sometimes contradictory regulatory requirements in the US and EU. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

Drug Discovery World

OCTOBER 19, 2022

Yapan Bio has expanded its capabilities with a new process development facility at Genome Valley, Hyderabad, India, which has enhanced its ability to support end-to-end development and manufacturing of RNA, DNA and gene therapy products starting from plasmids. .

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Development Gene Therapy RNA DNA 52

Pfizer

SEPTEMBER 25, 2022

Pfizer and Sangamo Therapeutics Announce Phase 3 Trial of Investigational Gene Therapy for Hemophilia A Has Re-Opened Recruitment. Hospira Issues A Voluntary Nationwide Recall For One Lot of Propofol Injectable Emulsion (containing benzyl alcohol), Due To The Potential Presence of Visible Particulates. Leadership. 09.22.2022.

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