Scienmag

MARCH 17, 2021

An international team of scientists from the University of Turku, Finland and PennState University, USA have solved a long-standing mystery of how living organisms distinguish RNA and DNA building blocks during gene expression paving the way for the design of new antiviral drugs. All cellular […].

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Gene Expression RNA DNA Drugs 74

Drug Discovery World

JANUARY 17, 2023

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Limitations of AAV gene therapy . We’re hoping to use what we’ve learned so far about LNPs to develop an improved gene editor delivery system.” .

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Gene Editing Gene Gene Therapy Genetics 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. It’s a great tool for controlling gene expression.”.

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DNA Gene Gene Silencing Genome 98

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: You describe your therapeutics as RNA Amplifiers. How do they work?

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Gene Expression Gene Genetic Disease RNA 59

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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The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. This space will provide dedicated and configurable lab and office space, access to Lilly scientists, and opportunities for collaboration.

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Genetics Medicine RNA Scientist 52

Drug Discovery World

NOVEMBER 7, 2022

From a general healthcare perspective, access to this sort of data is invaluable, but this is particularly noteworthy in that DNA tests have historically been limited in these regions on account of cost and availability of practitioners, services and resources.

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Genome Genomics Development Genetics 98

The Pharma Data

MAY 9, 2022

But now Northwestern Medicine scientists, in an international collaboration with scientists at the University of California, San Francisco and the University of Hong Kong, have identified a drug that inhibits growth of the most aggressive meningiomas and how to most accurately identify which meningiomas will respond to the drug.

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XTalks

APRIL 6, 2022

Cancerous cells and tumors release small fragments of DNA into the bloodstream called circulating tumour DNA (ctDNA). Scientists at the University of Cambridge evaluated the use of a personalized blood test that detects ctDNA to help predict the relapse risk of lung cancer.

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DNA In-Vitro Doctors Doctor 105

Drug Discovery World

APRIL 11, 2023

The molecule, known as lissodendoric acid A, appears to counteract other molecules that can damage DNA, RNA and proteins and even destroy whole cells.

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Research Drugs Life Science RNA 52

Drug Discovery World

JULY 8, 2024

Beard Distinguished Professor of Experimental Surgery and the Founding Director of the Translational Research Institute at Duke University, explores the rise of RNA therapeutics. However, the RNA landscape has drastically changed in recent years and we now know that the molecule is more than just a transient, linear carrier of information.

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RNA Protein Gene Expression Vaccination 59

Delveinsight

FEBRUARY 25, 2021

The gene-editing technologies, which have reached the clinic, CRISPR, zinc finger nucleases, and TALENs, make their edits by nicking DNA at the target site. The GuideTx technology screens for lipid nanoparticles inside the body that can be used to deliver RNA to cells throughout the body.

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DNA In-Vivo Genetics Medicine 52

Drug Discovery World

JUNE 27, 2024

Scientists at the University of Sydney have developed a gene-editing tool with greater accuracy and flexibility than CRISPR. SeekRNA uses a programmable ribonucleic acid (RNA) strand that can directly identify sites for insertion in genetic sequences, simplifying the editing process and reducing errors.

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Gene Editing Gene DNA Genetic Engineering 59

XTalks

MARCH 8, 2022

From leading COVID-19 vaccine developments to being at the helm of the latest gene therapies and inventing ground-breaking DNA technologies like CRISPR, women have been at the forefront of some of the most leading-edge scientific discoveries in recent years, both as innovators and leaders.

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Life Science Gene Therapy DNA Vaccination 105

XTalks

DECEMBER 24, 2020

From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. CRISPR Gene Editing Inventors Win Nobel Prize.

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Drug Discovery World

OCTOBER 16, 2023

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding. It has a vibrant community of life-science companies, from global pharma to smaller start-ups.

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Drugs Life Science Gene Therapy Genome 75

Drug Discovery World

JULY 17, 2024

A global collaboration led by scientists at the University of Oxford has discovered that genetic variants in a specific gene cause a rare neurodevelopmental disorder (NDD). The specific gene is called RNU4-2. The study estimates that these specific changes in the RNU4-2 gene can explain 0.4%

Genetics 52

Genetics Genome Project Genome Genomics 52

Drug Discovery World

AUGUST 1, 2023

1 August was first chosen as World RNA Day in 2018 as a play on AUG (adenine, uracil and guanine), a triple sequence of RNA (called a codon) that initiates protein synthesis by the cell. Since then, it has been observed to publicise the importance of this molecule in the generation of proteins in the body.

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RNA Drugs Gene Silencing Vaccination 52

Drug Discovery World

MAY 2, 2023

Steve Wowk , VP, Business Unit and General Management of Integrated DNA Technologies, shared with DDW the value of CRISPR-Cas9 in drug discovery. The CRISPR-Cas9 system, awarded the Nobel Prize in Chemistry in 2020 for its applications for gene editing, was initially encountered in the bacterial immune system.

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Genome Genomics Gene DNA 52

The Pharma Data

NOVEMBER 17, 2021

Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma.

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Scientist RNA DNA Genetics 52

pharmaphorum

FEBRUARY 3, 2021

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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Gene Editing Gene DNA Engineer 98

Drug Discovery World

APRIL 30, 2024

Messenger RNA (mRNA) has come into focus within the drug discovery and development as an exciting tool to deliver genetic information. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA). Rare diseases are largely treated using gene therapies which employ modified viruses to bring the therapy to the disease cells.

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Drug Discovery World

NOVEMBER 15, 2022

Rapid and accurate DNA synthesis . Conventional DNA synthesis, typically used for making sequences such as primers, employs phosphoramidite chemistry, a method that was developed in the 1980s and is still considered the gold standard for DNA synthesis. .

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DNA Drugs Antibody Engineer 98

Roots Analysis

MAY 21, 2024

Advantages of Lipid Nanoparticle LNPs are gaining significant attention of formulation scientists in drug delivery due to the various advantages offered by them. Gene Therapies: LNPs have the ability to effectively deliver nucleic acids, including small interfering RNA (siRNA), messenger RNA (mRNA), and plasmid DNA to the target cells.

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Drug Delivery Drugs Gene Therapy Bioavailability 40

Drug Discovery World

JULY 11, 2023

Now, with the growth of publicly available genomics, transcriptomics, and proteomics databases, the ability to quickly carry out large-scale DNA, RNA, and protein screenings, and the availability of massive sets of de-identified patient data, the amount of high-value, analysable data has reached enormous proportions.

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Big Data Development Life Science Drugs 40

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. This can be achieved by the simultaneous knockout of endogenous TCR genes and the insertion of neoantigen-specific TCRs allowing cells to recognise specific mutations.

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DNA Genome Genomics RNA 98

Roots Analysis

AUGUST 23, 2024

Unveiling The Potential of mRNA Technology mRNA is a single-stranded molecule that relays the genetic instructions needed to make proteins from DNA in the cell nucleus to ribosomes. mRNA therapeutics can deliver healthy copies of the gene, enabling cells to produce the correct protein and potentially reverse the disease process.

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Protein Vaccination Vaccine Immune Response 40

Drug Discovery World

AUGUST 1, 2022

As a result, scientists have been unable to determine whether the cells possess distinctive attributes that HIV-cure-directed therapies may exploit. Using data produced by FIND-Seq, the scientists compared the gene expression patterns of HIV-infected memory CD4+ T cells to those of HIV-uninfected memory CD4+ T cells in the same individuals.

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Gene Expression Scientist Gene Allergies 52

Drug Discovery World

AUGUST 10, 2022

Later, scientists took advantage of antibodies’ natural tendency to seek out specific antigens and created antibody therapies that guide T cells towards the cells they are supposed to destroy. To overcome the challenge of identifying the ideal target, scientists are using several creative methods.

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Manufacturing Gene Genome Genomics 98

Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.

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Gene Editing Gene Engineer DNA 52

Drug Discovery World

FEBRUARY 29, 2024

They are called: Sitting down with… PharmEnable , Is single-cell gene expression the next trend in next generation sequencing? In the second article, we hear from Dr Dina Finan, PhD, Product Manager at 10x Genomics and Dr Nick Downey, PhD, NGS Collaborations Lead at Integrated DNA Technologies.

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Gene Expression DNA Genome Genomics 52

Drug Discovery World

FEBRUARY 28, 2023

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

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The Pharma Data

DECEMBER 3, 2020

Significant differences among COVID-19 diagnostic tests are resulting in inaccurate results that sometimes put populations at risk or quarantine people needlessly, contributing to skepticism about pronouncements from scientists and public health authorities. Other techniques are primed to replace it.

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RNA Protein DNA Antibody 52

The Pharma Data

NOVEMBER 22, 2020

Scientists in Israel have used the CRISPR Cas-9 gene editing system to destroy cancerous cells in mice without damaging other cells. To conduct the research, the scientists used hundreds of mice with two of the most aggressive forms of cancer: glioblastoma and metastatic ovarian cancer. Photo courtesy of Science Advances.

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Gene Editing DNA Genome Genomics 52

Drug Discovery World

APRIL 14, 2023

DDW Editor Reece Armstrong speaks to Nautilus Co-Founder and Chief Scientist Parag Mallick , about the company’s recent collaboration which aims to uncover the proteins that could be behind a rare and fatal childhood cancer. And whilst proteomics isn’t a new field of study, it has largely been left in the hands of proteomics scientists. “If

Protein 52

Protein Genome Genomics DNA 52

Drug Discovery World

NOVEMBER 30, 2023

It was by Nicholas Field, Principal Scientist, Purification Development, Lonza, titled: ‘Tailored CMC solutions to overcome the challenges in bispecific, Fab and Fc-fusion protein development programs’. To demonstrate the utility of the company’s SYNTAX system in gene assembly, the 1.7

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Drug Discovery World

OCTOBER 17, 2023

RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited? The first step to reach this goal will be to generate protein and RNA data from the same sample. Let’s take CAR T cells as an example.

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