Drug Discovery World

MAY 8, 2024

Join DDW for this exclusive free event, ‘ rAAV genome sequencing with Oxford Nanopore uncovers transgene integrity and packaging impurities ’. The event will be supported by Oxford Nanopore Technologies. The event will take place on 13 May, 4PM BST.

Gene Therapy 69

Gene Therapy Gene DNA Genome 69

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. Rosalind Franklin.

Genetics 119

Genetics DNA Genome Genomics 119

Scienmag

JUNE 16, 2022

In 2014, Professor Anton Henssen discovered something unusual in the cells of pediatric cancer patients: small rings of DNA, which meant that part of the genetic information was no longer packaged in the chromosomes as normal.

DNA 83

DNA Packaging Packaging Genome 83

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. Advances in sequencing are leading to scientific breakthroughs in preventative care, faster diagnoses, and proactive medical interventions.

Genome 52

Genome Genomics Research Scientist 52

Drug Discovery World

FEBRUARY 27, 2024

DDW’s Megan Thomas speaks with Dr Blythe Sather, Vice President and Head of Research at Tune Therapeutics, about how genetic tuning will transform the reach for CAR-T therapies, the different diseases that can benefit, and the disruptive technology making this happen. MT: What is genetic tuning?

Gene Expression 59

Gene Expression DNA Genetics Gene 59

World of DTC Marketing

DECEMBER 29, 2020

Both the Pfizer and Moderna vaccines copied RNA sequence from the virus genome and found a way to manufacture it at scale with high-level processes and quality control. These vaccines were developed using different “platform technologies” that involve slotting genetic material from the virus into a tried and tested delivery package.

Vaccination 307

Vaccination Vaccine Development Clinical Trials 307

Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

Gene Expression 52

Gene Expression Gene Packaging Packaging 52

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. Expand representation across global populations.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

FDA Law Blog

MARCH 3, 2024

Biotechnology equipment includes genetic sequencers, mass spectrometers, polymerase chain reaction machines or any other equipment, components or accessories designed for the research, development, product, or analysis of biological materials as well as any software, firmware, or other digital components. China, Russia, Iran or North Korea).

Genome 102

Genome Genomics Manufacturing Production 102

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. . — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 92

In-Vivo Gene Editing Trials Drugs 92

Drug Discovery World

OCTOBER 25, 2023

There are two main concepts underlying this impact; we’ve known for a few years now that people can have variable responses to the same medication and that these responses are largely based on an individual’s genetic makeup. NGS is becoming invaluable for treating individual cancer patients based on their tumour’s genetic makeup.

Bioinformatics 115

Bioinformatics Genetics RNA Drugs 115

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. This system allows for precisely installing various genetic modifications, including insertions, deletions, and all 12 possible base-to-base conversions – without creating DSBs.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

JANUARY 23, 2024

The platform was recently updated to support FDA 21 CFR Part 11 compliance and include a full Installation Qualification / Operation Qualification (IQ/OQ) package for pharmaceutical quality assurance, extending the system’s applications into drug manufacturing operations. The consortium has been awarded €3.4

Compliance 52

Compliance Production Development Packaging 52

The Pharma Data

JANUARY 17, 2021

It received FDA guidance January 5 th and is in the midst of preparing the first package of investigational new drug (IND) applications – usually with an n-of-1, for individual patients. n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We

Genetics 52

Genetics Clinical Trials Clinical Trials Drugs 52

Drug Discovery World

FEBRUARY 8, 2023

The threat of empty capsids Capsids are the protein shell that would usually enclose the genetic material of a virus. In AAV therapeutics, empty capsids are those that are not packaged with the therapeutic DNA. Partially-filled capsids may also be present, and contain packaged process-related impurities or truncated genetic material.

Gene Therapy 52

Gene Therapy Gene Antibody Production 52

Roots Analysis

OCTOBER 30, 2023

Self-amplifying RNA (saRNA): Self amplifying RNA is an engineered form of RNA, combining the amplifying genome (native to certain viruses) to the therapeutic gene of interest. The primary target indications for tRNA therapeutics include genetic and oncological disorders.

RNA 40

RNA Vaccination Vaccine Development 40

Drug Discovery World

FEBRUARY 6, 2023

A case in point is the emerging omics and other high-throughput laboratory technologies that allow simultaneous profiling of genetic alterations in combination with profiling thousands of molecules in plasma, cell populations, or single cells. Figure 2: Advanced data visualisation examples for transcriptomics data.

Big Data 52

Big Data RNA Bioinformatics Vaccination 52

Drug Discovery World

MARCH 13, 2023

They can manufacture the drug substance and then pass it to a specialised fill-finish facility that will load it into its final sterile packaging.   Conversely, cell therapy manufacturing starts with a highly heterogeneous source material: human blood. Genetic Engineering & Biotechnology News. August 2020. April 4, 2022.

Gene Therapy 52

Gene Therapy Gene Manufacturing Development 52