Worldwide Clinical Trials

JUNE 15, 2022

At the end of May, we hosted a webinar titled “ Changing Times, Changing Therapies: Keeping Up with Advancements in Cell and Gene Therapies ” to provide a quick update on the latest advancements and ongoing in development of these advanced therapeutics. Clinical holds are becoming more common, especially in gene therapy programs.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

Drug Discovery World

JULY 15, 2022

The first complete, gapless sequence of a human genome was published 1 April 2022 in a special issue of the journal Science 1. While The Human Genome Project mapped about 92% of the human genome two decades ago, sequencing the last 8% of the genome proved highly challenging.

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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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Gene Therapy Gene Trials Clinical Trials 95

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.

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Drug Discovery World

MARCH 27, 2023

Genomics is driving a revolution in drug development and, in the long run, the state of public healthcare. The first draft human genome took a decade to create, and 13 years to complete. A robust environment will contain secure access controls that can prevent and detect misuse of data.

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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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XTalks

NOVEMBER 10, 2020

For more information on tackling this “genomic analysis bottleneck,” watch this on-demand webinar. How many genes need to be scanned in order to determine the presence of a genetic disease? It also slows the progress of identifying new disease gene associations in very rare conditions.

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Genome Genomics Medicine Genetic Disease 98

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry CRISPR nuclease is a key innovation area in pharmaceutical development CRISPR, which refers to clustered regularly interspaced short palindromic repeats, are bacteriophage-derived DNA sequences that had previously infected the prokaryote and are found in the genomes of bacteria and archaea.

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In-Vivo Genome Genomics Gene Editing 162

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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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

Drug Discovery World

MAY 2, 2023

The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system. In an effort to defend themselves against viral infection, bacterial cells capture and copy DNA fragments of bacteriophages into their genome.

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Genome Genomics Gene DNA 52

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

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Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

JULY 29, 2022

Scientists modelling the cancer-suppressing p53 gene identify how the 20 different molecules unique to elephants get activated for increased sensitivity and response against carcinogenic conditions – with implications for cancer treatments in humans. The post Could elephant genes hold the key to avoiding cancers?

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Gene DNA Protein Genetics 52

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

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Production Gene Protein Vaccination 52

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.

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DNA Gene Gene Silencing Genome 98

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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Roots Analysis

AUGUST 31, 2023

According to a study, around 20,000 genes are present in the human body, all of which interact with the nutrients in the food, either directly or indirectly. Nutrigenomics is the science studying the relationship between human genome, nutrition and health.

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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

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 without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

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DNA Gene Gene Silencing Genome 52

XTalks

DECEMBER 1, 2021

To ensure the new tests can identify Omicron based on its mutations, the companies assessed their tests against sequences in public databases of genomic data, such as GISAID and GenBank. Cue is also currently working with Google Cloud and genomics company Helix to study variants and share mutation sequencing data.

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Gene Genome Genomics Genetics 105

Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. Further, these can be used to activate both innate and adaptive immune system responses by silencing or upregulating immune-relevant genes.

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XTalks

APRIL 15, 2021

New advances in heart failure genomics are helping to address this challenge. Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. Watch this on-demand webinar to hear from these experts.

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Development Drugs Genome Genomics 98

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Everything the 100,000 Genomes Project does has to be rubber-stamped by the patients”. For some people, a job is more than just a job.

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XTalks

JANUARY 2, 2024

Filsuvez topical gel is a sterile botanical drug product designed for topical use, containing birch triterpenes within an oil base. For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

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XTalks

OCTOBER 21, 2020

The answer may lie in the genetic code of the virus, which scientists at Duke University have found contains several silent mutations that affect protein folding. Previous research found fingerprints of positive selection within a gene that encodes the spike proteins. The study was published on October 16 in the journal PeerJ.

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RNA Protein Genome Genomics 98

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. This innovative method now brings technology closer to developing genome-wide PPI networks.

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Protein Gene Bioassay DNA 122

Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. In addition to STC-15 and our METTL1, our pipeline contains a number of preclinical programs that are advancing rapidly for the treatment of viruses, neurodegeneration, cancer, and immuno-inflammatory conditions.

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RNA In-Vivo In-Vitro DNA 64

Drug Discovery World

AUGUST 10, 2022

For example, one clinical trial examined the safety and efficacy of a CAR T cell that includes a suicide gene, which produces a toxic protein that causes the CAR T cells to die upon exposure to a drug 10. If the gene does not integrate into the genome, the cell will not become a CAR T cell.

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Manufacturing Gene Genome Genomics 98

Drug Discovery World

NOVEMBER 14, 2022

AAV vectors for diabetes gene therapy. A collaboration between PerkinElmer’s SIRION Biotech business and the Centre for Genomic Regulation (CRG) agreed to jointly develop new generation adeno-associated virus (AAV) vectors for type 1 and type 2 diabetes gene therapy in the pancreas. .

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Development Gene Therapy Drugs Gene 52

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Everything the 100,000 Genomes Project does has to be rubber-stamped by the patients”. For some people, a job is more than just a job.

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Genome Genomics Genetics Genome Project 66

Pharmaceutical Technology

DECEMBER 9, 2022

In regards to the latter, notable examples include the acquisition of Cergentis, a genomics-focused biotechnology company based in the Netherlands. The deal allows the company to provide proprietary technology and knowledge steeped in fast-evolving genomic techniques that support effective decision-making and R&D program design.

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Scienmag

JUNE 22, 2021

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XTalks

FEBRUARY 11, 2021

The Human Genome Project recently marked 20 years since the publication of the first full sets of human genomic sequences, an endeavor that spanned well over a decade. Today, new next-generation sequencing technologies allow for the sequencing of complex genomes within just a day or two.

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Genetics DNA Genome Genomics 119

The Pharma Data

JANUARY 10, 2021

Nasdaq: EDIT), a leading genome editing company, today announced the U.S. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. CAMBRIDGE, Mass.,

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

The Pharma Data

NOVEMBER 24, 2020

BRCA 1/2 or ATM gene mutation must be confirmed before Lynparza treatment is initiated. 2 Germline and somatic mutations in HRR genes, which include BRCA1/2 and ATM, are associated with potentially more aggressive prostate cancers and poor prognosis, demonstrating the importance of biomarker testing. MISSISSAUGA, ON , Nov.

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Gene Hormones DNA Medicine 52

Drug Discovery World

JULY 3, 2023

PrecisionLife and the University of Oxford have signed a data access agreement to license the Oxford Endometriosis Gene (OXEGENE) dataset to develop new personalised treatments for endometriosis patients. Endometriosis affects 10% of women globally, but on average it takes over seven years for patients to receive a diagnosis.

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