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. Shares in bluebird ticked up following the announcement.

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The Pharma Data

JULY 11, 2021

On the one hand, they can boost the metabolism of T cells by inducing central regulators of energy metabolism. On the other hand, they can inhibit specific enzymes that regulate the accessibility to the genetic material and thus the gene expression in the T cells. In doing so, they induce epigenetic changes.

Bacteria 52

Bacteria Genetics Scientist Regulation 52

XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy. Secondly, other funded research is exploring differential gene expression regulated by DNA methylation.

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Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation.

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Marketing Gene Drugs Protein 52

Pharmaceutical Technology

JUNE 4, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

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Genetics Gene Expression DNA Antibody 130

The Pharma Data

NOVEMBER 9, 2020

This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. Furthermore, Amgen’s research, testing, pricing, marketing and other operations are subject to extensive regulation by domestic and foreign government regulatory authorities.

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XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. This control plays a critical role in development and cellular function.

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Regulation Genetics Scientist RNA 52

pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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Gene Editing Gene Gene Therapy In-Vivo 145

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.”.

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DNA Gene Gene Silencing Genome 98

Roots Analysis

AUGUST 31, 2023

It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

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Gene Gene Therapy Gene Expression Regulation 40

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

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RNA Protein Genetics Gene Expression 40

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 52

DNA Gene Gene Silencing Genome 52

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. They are different from small molecules in terms of their size and complexity.

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Contract Manufacturing Protein Manufacturing Production 40

The Pharma Data

MAY 15, 2021

All too often, it is lifestyle factors – the things we do day to day – that determines whether a particular gene switches on to create actual illness. The scientific study of how to undo genetic illnesses – like HCT – is called epigenetics. It’s because certain genes act one way on some people… and another way on other people.

Bacteria 52

Bacteria Gene Gene Therapy Gene Expression 52

XTalks

DECEMBER 24, 2020

However, for virologists like Lori Frappier, PhD, professor in the department of molecular genetics at the University of Toronto, the outbreak of SARS-CoV-2 was less of a surprise and more of an inevitability. CRISPR Gene Editing Inventors Win Nobel Prize. Vaccine Considerations. But nevertheless, 2021 into 2022 will be the time.”.

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The Pharma Data

MAY 13, 2021

The trans-fat ban–food regulation and long-term health. Regulation of glucose metabolism from a liver-centric perspective. Neural and Genetic Correlates of the Social Sharing of Happiness. Adipose tissue regulates insulin sensitivity: role of adipogenesis, de novo lipogenesis and novel lipids. Biochem Cell Biol.

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