Gene Expression, Gene Therapy, Genetics and Genome

Gene expression delivery tool ‘slides’ instructions into cells

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

Gene Expression Gene Packaging Packaging

This week in drug discovery (2-6 October)

Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs

Drugs Gene Editing Genome Genomics

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Typically, nitrogen in the blood is removed in the form of urea.

RNA

RNA Gene Expression Gene Gene Therapy

bluebird seeks gene therapy trial restart after cancer scare

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

Gene Therapy

Gene Therapy Gene Trials Gene Expression

Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: How easy was it to transfer theoretical research into a viable business?

Gene Expression

Gene Expression Gene Genetic Disease RNA

Gene editing: beyond the hype

pharmaphorum

JANUARY 29, 2021

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

Gene Editing

Gene Editing Gene Gene Therapy In-Vivo

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics

Genetics Medicine Gene Therapy Marketing

Decibel Therapeutics Announces $82M Series D Financing to Advance Gene Therapies to Restore Hearing and Balance

The Pharma Data

NOVEMBER 8, 2020

DB-OTO, Decibel’s investigational gene therapy to restore hearing in children with congenital deafness due to a deficiency in the otoferlin gene, is in preclinical studies, and Decibel expects to initiate clinical testing in 2022. Thompson, M.D., a partner at OrbiMed, to its board of directors.

Gene Therapy

Gene Therapy Gene Gene Expression Genome

Delivering on the promise of gene editing

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. In one example, Choi et al.

Gene Editing

Gene Editing Gene In-Vivo Genome

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

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.”.

DNA

DNA Gene Gene Silencing Genome

The future outlook for mRNA therapies

Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. However, these therapies can cause severe adverse effects.

RNA

RNA Protein Vaccination Vaccine

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

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.

DNA

DNA Gene Gene Silencing Genome

CRISPR breakthroughs: New solutions for common diseases

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. It also enables the downregulation of gene expression by directly knocking out the cytoplasmic mRNA transcripts.

DNA

DNA Genome Genomics RNA

The use of base editing in stem-cell based therapies

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. The success of base editing has led to rapid advances toward commercialisation and clinical application.

Gene Editing

Gene Editing Gene Engineer DNA

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

XTalks

DECEMBER 24, 2020

From isolating SARS-CoV-2 in early January to sequencing its genome shortly thereafter and having a prototype vaccine against it within days, scientific process and progress have held steadfast throughout the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize. The illness was later named coronavirus disease 2019 (COVID-19).

Life Science

Life Science Vaccination Vaccine Gene

Clinical Research Informer

Gene expression delivery tool ‘slides’ instructions into cells

This week in drug discovery (2-6 October)

Trending Sources

CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

bluebird seeks gene therapy trial restart after cancer scare

Turning science into business: Amplifying mRNA by targeting regRNAs

Gene editing: beyond the hype

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Decibel Therapeutics Announces $82M Series D Financing to Advance Gene Therapies to Restore Hearing and Balance

Delivering on the promise of gene editing

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

The future outlook for mRNA therapies

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

CRISPR breakthroughs: New solutions for common diseases

The use of base editing in stem-cell based therapies

2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences

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