pharmaphorum

SEPTEMBER 14, 2020

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his children’s rare neurological diseases. Here he gives us a deeper look at how genomic medicine is evolving and the barriers that are preventing it from reaching its full potential.

Genomics 119

Genomics Genome Medicine Gene 119

Worldwide Clinical Trials

DECEMBER 12, 2022

Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. What are the benefits and limitations to using genetic testing? It is important to consider the type of testing performed (e.g.,

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 130

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

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Gene Editing Gene Genetic Disease Research 126

Pharmaceutical Technology

MAY 17, 2023

The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

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.

Genomics 98

Genomics Genome Genetics Gene 98

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.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

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.

Genome 75

Genome Genomics Gene Genetics 75

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. In other words, every person’s cancer has its own genetic construct. Tailored oncology.

Genomics 105

Genomics Genome Clinical Trials Clinical Trials 105

pharmaphorum

OCTOBER 12, 2022

David Del Bourgo (CEO and co-founder, Whitelab Genomics) has always been passionate about introducing disruptive, innovative technologies to markets. We founded Whitelab Genomics after realising the potential to use data, data science, and AI in a more systematic way to develop genomic therapies,” Del Bourgo says.

Genomics 105

Genomics Genome Medicine Development 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…

Genomics 111

Genomics Genome Gene Editing Genetic Disease 111

Medical Xpress

JANUARY 5, 2023

A genotype-first approach to patient care involves selecting patients with specific genomic variants and then studying their traits and symptoms; this finding uncovered new relationships between genes and clinical conditions, broadened the traits and symptoms associated with known disorders, and offered insights into newly described disorders.

Genomics 75

Genomics Genome Genetics Genotype 75

Pharmaceutical Technology

FEBRUARY 15, 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: Genetically modified animal models. Regeneron Pharmaceuticals is one of the leading patent filers in the generation of genetically modified animal models.

Genetics 130

Genetics DNA Genome Genomics 130

Pharmaceutical Technology

FEBRUARY 15, 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: Gene splicing using nucleases. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins.

Gene Splicing 130

Gene Splicing Gene DNA Recombinant DNA Technology 130

XTalks

MAY 23, 2024

One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.

Genetics 114

Genetics Research Genomics Genome 114

Pharmaceutical Technology

JUNE 30, 2022

Analysing almost eight thousand tumours across 33 different cancers, researchers say this marks the first time that a framework was created to understand the role of internal factors in driving such genomic alterations. Genomic research have greatly expanded our understanding of disease pathophysiology over the years.

Genome Project 130

Genome Project Genomics Genome DNA 130

Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. AAV genomes are highly compact, with overlapping coding regions, alternate splicing schemes, and multiple transcription initiation codons.

Gene 262

Gene Gene Therapy Genetics DNA 262

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.

Genomics 130

Genomics Genome In-Vitro Gene 130

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. The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder.

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RNA Gene Expression Gene Gene Therapy 245

Scienmag

APRIL 15, 2021

Team of Japanese and European scientists identify a novel genetic mitochondrial disorder by analyzing DNA samples from three distinct families Credit: Fujita Health University DNA ligase proteins, which facilitate the formation of bonds between separate strands of DNA, play critical roles in the replication and maintenance of DNA.

Genetics 105

Genetics DNA Genomics Genome 105

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science 130

Life Science Genetic Disease Genome Genomics 130

XTalks

JULY 19, 2021

Phosphorus, a leading preventative genomics company, has developed the first comprehensive preventative genetic test for consumers. The test is called GeneCompass and features medical-grade technology to provide a holistic assessment of genetic health and wellness. The Phosphorus GeneCompass test has a list price of $249.

Genetics 98

Genetics Genome Genomics Gene 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.

Genomics 98

Genomics Genome Gene Editing Genetics 98

STAT News

MARCH 1, 2023

Genome editing summits are generally friendly, nerdy affairs, but for a moment at a Lisbon hotel last June, the conversation at the FASEB genome engineering conference grew tense. ” It was a rare moment: Tessera had raised around $600 million and passed a $1 billion valuation, but published little in academic medical journals.

Gene Editing 119

Gene Editing Genomics Genome Genetic Disease 119

Scienmag

SEPTEMBER 29, 2021

Describing a previously unknown genetic condition that affects children, researchers at University of California San Diego School of Medicine and Rady Children’s Institute for Genomic Medicine say they also found a potential method to prevent the gene mutation by administering a drug during pregnancy.

Genetics 84

Genetics Research Genomics Genome 84

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. “It would have saved me and the NHS a lot of money. .” Why do we always have to follow? Why not lead?”

Genomics 104

Genomics Genome Genetics Medicine 104

STAT News

APRIL 17, 2023

Genetic engineering has the potential to transform how we raise animals for meat and other products, making food safer, improving animal health and welfare, and shrinking animal agriculture’s environmental footprint. Pigs that are less likely to induce allergic reactions in humans. Read the rest…

Genomics 98

Genomics Genome Genetic Engineering Production 98

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

Genomics 98

Genomics Genome Medicine Genetic Disease 98

Pharmaceutical Technology

JUNE 14, 2023

However, more immeasurable characteristics such as personality, behaviour, and even intelligence are all influenced by genetics to varying degrees. All that DNA is organised into hereditary units called genes, with humans having about 25,000 genes collectively known as the genome. Each human cell has 1.8

Genome Project 130

Genome Project Medicine Genome Genomics 130

Medical Xpress

APRIL 21, 2023

Despite the increasing use of genomic sequencing in clinical practice, interpreting rare genetic mutations, even among well-studied disease genes, remains difficult.

Genetics 80

Genetics Genomics Genome Gene 80

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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Gene Editing DNA Gene Gene Therapy 235

pharmaphorum

JANUARY 24, 2023

Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates. The logical extension to this kind of approach is treating individual patients, with their individual genetic makeup.

Genetics 98

Genetics Drugs Genome Project Biobanking 98

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)

Gene 88

Gene Genomics Genome Genetics 88

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.

Gene Editing 98

Gene Editing DNA Gene Gene Sequencing 98

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.

Gene 80

Gene Gene Expression Genomics Genome 80

BioSpace

JULY 14, 2021

The next COVID pandemic could be prevented by using a gene drive to preemptively edit the genome of bats to prevent them from becoming hosts for coronaviruses, according to a proposal by scientists from Israel’s Interdisciplinary Center Herzelia and the National Institutes of Health.

Scientist 98

Scientist Genetics Genomics Genome 98

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).

Gene 98

Gene Research Genomics Genome 98

Scienmag

NOVEMBER 25, 2020

Credit: Photo: IPK Leibniz Institute/ Andreas Bähring In order to record all genetic information of an individual, its genome must be completely decoded. But to understand the genetic information of the entire barley species, much more […].

Genomics 88

Genomics Genome Scientist Genetics 88