STAT News

DECEMBER 12, 2022

LONDON — England is launching a pilot program sequencing the genomes of up to 100,000 newborns to see if such a strategy can speed up the detection of genetic illnesses. The Newborn Genomes Programme will scan DNA for mutations that can cause some 200 conditions.

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Genomics Genome Genetic Disease 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.

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Gene Editing Genomics Genome Genetic Disease 119

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.

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Genomics Genome Medicine Genetic Disease 98

BioPharma Reporter

JUNE 19, 2023

Abu Dhabi’s Department of Health has collaborated with Mass General Brigham's International Center for Genetic Disease (iCGD).

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Genetic Disease Genomics Genome Genetics 94

STAT News

FEBRUARY 22, 2023

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. Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines.

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Gene Editing Gene Genetic Disease Research 126

BioTech 365

FEBRUARY 18, 2021

Illumina and the University Hospital of Tübingen Evaluate Potential of Whole Genome Sequencing to Improve Diagnosis of Full Range of Genetic Diseases Illumina and the University Hospital of Tübingen Evaluate Potential of Whole Genome Sequencing to Improve Diagnosis of Full … Continue reading →

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Genetic Disease Genomics Genome Genetics 40

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 University of California Los Angeles are currently investigating the use of genetically altered hepatocyte-like cells generated from induced pluripotent stem cells.

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RNA Gene Expression Gene Gene Therapy 245

BioTech 365

OCTOBER 21, 2020

Fabric GEM’s accuracy validated across five leading genomics centers of excellence in the U.S. –(BUSINESS WIRE)–Fabric Genomics, a global leader in clinical interpretation of genomic data, announced today the launch of Fabric GEM, a novel algorithm that … Continue reading →

Genomics 40

Genomics Genome Genetic Disease Genetics 40

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

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Genomics Genome Genetics Genome Project 102

Pharmaceutical Technology

FEBRUARY 23, 2023

The collaboration will combine the mRNA platform of Moderna with the gene editing technologies suite, including the base editing capabilities of Life Edit for the development of curative therapies to treat challenging genetic diseases. Life Edit will get an upfront payment and is eligible for potential milestone payments.

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Gene Editing Gene In-Vivo Gene Therapy 147

Scienmag

MAY 26, 2022

— CRISPR has ushered in the era of genomic medicine. A line of powerful tools has been developed from the popular CRISPR-Cas9 to cure genetic diseases. ITHACA, N.Y.

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Gene Editing Genetic Disease Gene Genomics 63

pharmaphorum

SEPTEMBER 9, 2020

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genomics 80

Genomics Genome Genetics Genome Project 80

pharmaphorum

OCTOBER 4, 2022

AstraZeneca’s rare disease firm Alexion is set to expand its genomic medicine portfolio with the acquisition of gene editing specialist LogicBio Therapeutics, in a deal worth approximately $68 million. per share, a rare 660% premium on LogicBio’s share price. .

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Gene Editing Gene Genomics Genome 64

XTalks

AUGUST 29, 2022

CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. This technology has powerful implications for therapeutic uses, such as replacing mutated or disease-causing genes or increasing the activity of cancer-fighting cells.

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Gene Editing DNA Gene Genomics 98

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. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genomics 98

Pharmaceutical Technology

MAY 8, 2023

Innovation S-curve for the pharmaceutical industry Zinc-finger nucleases is a key innovation area in pharmaceutical Zinc finger nucleases (ZFNs) are tools used in genetic engineering to cleave a chosen genomic sequence. ZFNs are also used to create a new generation of genetic disease models called isogenic human disease models.

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Genetics DNA Genetic Engineering Antibody 130

XTalks

APRIL 15, 2021

New advances in heart failure genomics are helping to address this challenge. Experts from Servier and Genuity Science recently spoke on a webinar about using genomics data to drive drug development in heart failure and identify new targets for novel therapeutics. Watch this on-demand webinar to hear from these experts.

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Development Drugs Genomics Genome 98

pharmaphorum

JULY 20, 2022

The Cambridge, Massachusetts biotech has discovered that areas of the non-coding parts of the human genome – referred to by its chief executive Josh Mandel-Brehm (pictured above) as the “dark side” of the genome – actually produce regRNAs that control the expression of the 2% that codes for proteins.

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RNA Genetic Disease Drugs Protein 52

pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

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RNA Genetics Dermatology Antibody 105

The Pharma Data

OCTOBER 27, 2020

.” The single-reaction panel targets five regions of the viral genome and provides excellent accuracy and sensitivity. “Most real time qPCR-based assays target only two or three targets on the viral genome.” “Most real time qPCR-based assays target only two or three targets on the viral genome.”

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Genetic Analysis Genomics Genome Reagent 52

Roots Analysis

FEBRUARY 21, 2022

Since the introduction of a gene sequencing method by Frederick Sanger in 1977, the field of genomic data collection and analysis has evolved significantly. Moreover, the manual protocols require extensive manipulation, costly reagents and long duration of skilled genomic library production. Advantages of Next Generation Sequencing.

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Marketing Gene Sequencing DNA Genomics 52

Roots Analysis

AUGUST 12, 2024

Molecular diagnostic tests are advanced techniques and tools used to analyze biological markers in the genome and proteome. These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans.

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Genetics Gene Expression RNA DNA 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.

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DNA Gene Gene Silencing Genomics 52

The Pharma Data

DECEMBER 13, 2020

which develops genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of diseases, today announced the appointment of Bo Zhang, Ph.D., 14, 2020 10:00 UTC. BEIJING & CAMBRIDGE, Mass.–( –( BUSINESS WIRE )– EdiGene, Inc. as Head of Business Development.

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Business Development Development Gene Editing In-Vivo 40

pharmaphorum

APRIL 20, 2022

Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Cancer is a genetic disease, caused by certain changes in the way that genes control cell function, such as how they grow and divide. Why cancer?

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Gene Editing DNA Gene Genetics 52

The Pharma Data

JANUARY 17, 2021

n-Lorem selects patients based on their genetic mutation and the organ affected rather than their specific diseases. “We It’s the cause of the mutation, not the name of the disease, that matters.”. They’re basically the same,” but each one uses a particular nucleotide to bind to specific genetic sequence.

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Genetics Clinical Trials Clinical Trials Drugs 52

Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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Genetics Trials Clinical Trials Clinical Trials 189

XTalks

DECEMBER 13, 2023

3) Fulgent Genetics Compound annual growth rate: 260 percent Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. The company achieved core revenues of $67 million, reflecting a remarkable 48 perecent year-over-year growth.

Genetics 111

Genetics Vaccine Vaccination DNA 111

The Pharma Data

OCTOBER 28, 2020

The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. About Ultragenyx.

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Protein Production Development Drugs 40

XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). In the UK, Libmeldy will be administered by a specialist service through the Centre for Genomic Medicine at Saint Mary’s hospital in Manchester.

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Gene Therapy Gene Drugs Gene Silencing 98

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

JANUARY 6, 2025

Additionally, CRISPR genome-wide screening holds great potential for identifying key disease-associated genes and uncovering novel therapeutic targets. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future. The global market for cell and gene therapies , valued at $5.9

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