Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

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Gene Editing Gene In-Vivo Gene Therapy 147

Medical Xpress

MAY 2, 2023

A team of gene therapists, oncologists, genetic sequencing experts and neurosurgeons affiliated with a host of institutions in the U.S. and one in Sweden has uncovered gene variants that appear to be responsible for passing on familial glioma from parent to offspring.

Genome 86

Genome Genomics Gene Genetics 86

pharmaphorum

SEPTEMBER 12, 2022

Alongside drugs and therapeutics, data collection and technology enhancements have redefined the traditional healthcare experience, especially within oncology, and genomic profiling has become a significant factor in allowing for personalised care. Tailored oncology. In other words, every person’s cancer has its own genetic construct.

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STAT News

AUGUST 17, 2022

When the Smithsonian National Museum of Natural History opened its genomics exhibit in 2013, the field was just celebrating the 10th anniversary of the completed Human Genome Project. Sequencing that first genome cost over $500 million. The genomes since cost $10,000. The development of CRISPR-Cas9 landed a Nobel Prize.

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Genome Genomics Genome Project DNA 98

Medical Xpress

NOVEMBER 7, 2022

Scientists have discovered the cause of a rare condition within a part of the genome that has been largely unexplored in medical genetics.

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Pharmaceutical Technology

OCTOBER 26, 2022

The biotherapeutics market is rapidly growing, with 2021 seeing the highest-ever cell and gene therapy approval number. Gene therapy uses DNA to manipulate cells and correct defective genes, whereas cell therapy is the infusion or transplantation of cells into a patient. The future of cell and gene therapies.

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Medical Xpress

NOVEMBER 10, 2022

Researchers from The Hospital for Sick Children (SickKids) have uncovered new genes and genetic changes associated with autism spectrum disorder (ASD) in the largest autism whole genome sequencing analysis to date, providing better understanding into the 'genomic architecture' that underlies this disorder.

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Genome Genomics Gene Genetics 71

XTalks

SEPTEMBER 9, 2021

The US Food and Drug Administration (FDA) has placed a clinical hold on BioMarin Pharmaceutical’s investigational gene therapy BMN 307 for the rare inherited disease phenylketonuria (PKU) over safety concerns found during preclinical testing. The mice developed the tumors one year after being given BMN 307.

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Gene Therapy Gene Genome Genomics 105

STAT News

MARCH 3, 2023

Next week, hundreds of scientists from around the world will convene in London for an international summit on genome editing. That technology, which enables scientists to easily excise, alter, or replace specific sections of DNA, was awarded the 2020 Nobel Prize for Chemistry. Continue to STAT+ to read the full story…

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Genome Genomics Gene Editing Genetic Disease 111

BioPharma Reporter

APRIL 6, 2022

The FDA has released two draft guidance documents: for human gene therapy products incorporating human genome editing, and for CAR T cell products.

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Gene Therapy Gene Genome Genomics 98

pharmaphorum

NOVEMBER 27, 2022

A group representing pharma companies selling precision therapies for cancer has called for a change to the way genomic testing is done in Scotland, to make sure patients get access to targeted drugs. The post Genomic testing ‘should be offered to all cancer patients in Scotland’ appeared first on. Why not lead?”

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Genome Genomics Genetics Medicine 104

Pharmaceutical Technology

AUGUST 11, 2022

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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Medical Xpress

FEBRUARY 28, 2025

Dana-Farber investigators found that normally defunct viral genes that lie dormant in the human genome can be activated in the most common form of kidney cancer (clear cell renal cell carcinoma) and can end up triggering an immune response against the cancer.

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Genome Genomics Immune Response DNA 76

Pharmaceutical Technology

APRIL 12, 2023

FoundationOne CDx is a sequencing-based in vitro diagnostic device that can identify alterations in 324 genes from tumour samples. The ROS1 gene is altered in approximately in 1-2% of lung cancer patients. Exposure to environmental factors can cause gene fusion which leads to upregulation of the ROS-1 enzyme.

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Genome Genomics In-Vitro Gene 130

Pharmaceutical Technology

DECEMBER 21, 2023

Monitor and assess gene editing and CAR-T cell therapies for advancements in immunotherapy. Detect the integration of a CAR-encoding nucleic acid into the TRAC locus with a patented method and kit.

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Gene Editing Gene Genome Genomics 100

NPR Health - Shots

MARCH 8, 2023

The Third International Summit on Genome Editing concluded Monday with ethicists warning scientists to slow down efforts to use gene-editing to enhance the health of embryos. Image credit: Mark Schiefelbein/AP)

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Gene Editing Gene Genome Genomics 98

Drug Discovery Today

OCTOBER 27, 2022

In the field of life sciences, Lithuania is best known for the discovery of gene scissors by Prof. The country now hosts the EMBL Partnership Institute for Genome Editing Technologies at the Vilnius University Life Sciences Center aiming to advance gene editing technologies. Virginijus Šikšnys. One of them is the Jones!Lab,

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Genome Genomics Gene Editing Life Science 85

BioPharma Reporter

FEBRUARY 21, 2022

PerkinElmerâs Sirion Biotech business is teaming up with the Centre for Genomic Regulation (CGR) 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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Gene Therapy Gene Development Genome 98

STAT News

MARCH 6, 2023

LONDON — The first gene-edited children were born in China five years ago , but it’s unlikely to happen again there anytime soon. That was the message Chinese scientists delivered Monday on the opening day of the Third International Summit on Human Genome Editing in London.

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Scienmag

JANUARY 19, 2021

publishers New Rochelle, NY, January 19, 2021–Gene editing therapies, including CRISPR-Cas systems, offer the potential to correct mutations causing inherited retinal degenerations, a leading cause of blindness. Credit: Mary Ann Liebert, Inc.,

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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

Pharmaceutical Technology

SEPTEMBER 9, 2022

It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored. The company discovered mutations in small RNA genes that are connected to microvascular dysfunction in a previous study.

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RNA Development Gene Therapy Gene 246

Scienmag

JANUARY 5, 2022

SAN FRANCISCO, CA—January 5, 2021—Over the past decade, the CRISPR genome-editing system has revolutionized molecular biology, giving scientists the ability to alter genes inside living cells for research or medical applications. Credit: Photo: Michael Short/Gladstone Institutes […].

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Gene Gene Editing Research Genome 87

XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Medical Xpress

NOVEMBER 16, 2022

Researchers at the National Institutes of Health have successfully identified differences in gene activity in the brains of people with attention deficit hyperactivity disorder (ADHD).

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Pharmaceutical Technology

MARCH 28, 2023

Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.

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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

Scienmag

APRIL 8, 2022

UNSW researchers have used CRISPR gene editing – a type of ‘molecular scissors’ – to understand how deletions in one area of the genome can affect the expression of nearby genes.

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Gene Editing Gene Genome Genomics 84

STAT News

APRIL 17, 2023

But with the advent of genetic engineering, genes encoding favorable traits in one species (say, that hornless cow) could be spliced into the genomes of other animals. For millennia, farmers and ranchers had to encourage desirable traits by selective breeding, a notoriously painstaking and imprecise technique. Read the rest…

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Genome Genomics Genetic Engineering Production 98

Medical Xpress

MAY 11, 2023

Genomic studies of cancer patients have revealed thousands of mutations linked to tumor development. However, for the vast majority of those mutations, researchers are unsure of how they contribute to cancer because there's no easy way to study them in animal models.

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Gene Editing Gene Genome Genomics 125

Scienmag

JUNE 17, 2022

The American College of Medical Genetics and Genomics (ACMG) has released an update to the recommended minimum gene list for the reporting of secondary findings (SF). In 2021, the ACMG Board of Directors and Secondary Findings Working Group (SFWG) declared that the College would update the list (SF v3.0) Today’s update (SF v3.1)

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Gene Genome Genomics Genetics 88

Medical Xpress

JANUARY 17, 2023

Technology that enables an unprecedented, high-resolution look for all structural variants in our genes that are known to cause cancer can outperform standard tests used today for common blood cancers like leukemia, researchers report.

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Gene Genome Genomics Research 90

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

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Drug Discovery Today

APRIL 24, 2023

Hussman Institute for Human Genomics at the University of Miami Miller School of Medicine have found that variations in chromatin accessibility, and thus gene expression, may explain why people of European descent with APOE4 gene variants have a greater risk of developing Alzheimer’s disease than people of African descent with similar genetics.

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Gene Gene Expression Genome Genomics 80

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. The connections between regulatory genes and their downstream targets have been mapped to some extent — they can be thought of as a subway or metro map, with connections existing between major “hubs.”

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Pharmaceutical Technology

JULY 29, 2022

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood.

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RNA Gene Expression Gene Gene Therapy 245

pharmaphorum

OCTOBER 13, 2020

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. The post New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing appeared first on.

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