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

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. . We used these peptides to act as zip codes to deliver nanoparticles carrying genetic materials to the intended address within the eye.” .

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Gene Editing Gene Gene Therapy Genetics 98

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

Scienmag

NOVEMBER 16, 2020

Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., doi:10.1186/s13073-020-00788-5… SINGAPORE, 3 Nov 2020 – Long RNA molecules carrying DNA codes that […]. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020). Genome Med 12, 89 (2020).

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In-Vivo RNA DNA Genome 53

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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Drugs Scientist DNA Genetics 52

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

JANUARY 23, 2023

Scientists from Korea and the US have unveiled the mechanism of radioresistance in GBM cells, identifying therapeutic targets to overcome radioresistance. Armed with this understanding, the researchers further demonstrated that the genetic inhibition of DGAT1 suppresses radioresistance.

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FDA Approval Drugs In-Vivo In-Vitro 52

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. 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.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

DECEMBER 14, 2022

Developed by MIT researchers Jonathan Gootenberg and Omar Abudayyeh, PASTE (Programmable Addition via Site-specific Targeting Elements) gene editing technology can insert genes as long as 36,000 DNA base pairs to liver cells in mice as well as several types of human cells.

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Gene Editing DNA Gene Gene Sequencing 98

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

Pharma Mirror

APRIL 21, 2024

This represents an increasingly promising field of precision medicine, holding significant prospects for preventing and treating numerous challenging or genetic diseases. Scientists have been striving to optimize mRNA stability, immunogenicity, translation efficiency, and delivery systems to achieve efficient and safe mRNA delivery.

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pharmaphorum

DECEMBER 21, 2020

Their technology is based on synthetic messenger RNA – short for ribose nucleic acid – which is a short transcript of a longer DNA code. As it’s just a messenger molecule, it does not affect the body’s own genetic code when it is injected as a vaccine – but what it does do is instruct cells to code for copies of a certain protein.

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Vaccination Vaccine Doctors Doctor 93

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. Though some genetic mutations are harmful, others can be protective.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

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). Reece Armstrong explores the potential and future opportunities for mRNA-based therapies.

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RNA Protein Vaccination Vaccine 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. doi: 10.1126/sciadv.abg9551. Source link: [link].

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Scientist RNA DNA Genetics 52

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. Experts speak DDW asked a range of drug discovery experts where they thought the drug discovery expertise happening in the UK.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

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 97

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

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

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. pyogenes dCas9.

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DNA Gene Gene Silencing Genome 98

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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Genetics Genetic Analysis Vaccination Vaccine 52

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. Viral vectors have traditionally been used in gene editing where the shells of viruses carry the template DNA into cells, but this method has its disadvantages.

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Gene Editing DNA Gene Genome 98

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. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g.,

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DNA Genome Genomics RNA 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 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

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

In cell therapy, regenerative and adoptive immunotherapies continue to bridge the gap between genetic engineering strategies and their clinical application. This circumvents several technical challenges inherent to the genetic manipulation of differentiated cells in ex vivo culture or intact tissues.

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Gene Editing Gene Engineer DNA 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

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Protein Genome Genomics DNA 52

The Pharma Data

JANUARY 13, 2021

.” The Johnson & Johnson vaccine also would be hardier than either of the already approved vaccines because it relies on DNA technology rather than messenger RNA to prompt an immune system response. The new vaccine remains stable for a time in the refrigerator.

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Vaccination Vaccine Trials Immune Response 40

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. The female scientist duo became the first women to be jointly awarded a Nobel Prize, making it a winning moment for women in science.

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Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

FEBRUARY 20, 2023

DDL was founded in 1994, also originally started by a group of scientists operating in a university, also specialising in infectious diseases, but with more of a focus on HPV and Hepatitis. Viroclinics Biosciences – what it was called before the DDL purchase – was also well known for its specialist logistics infrastructure.

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Research Clinical Trials Clinical Trials Trials 130

XTalks

SEPTEMBER 14, 2020

The company’s candidate vaccine, mRNA-1273, is a synthetic messenger RNA that encodes the stabilized SARS-CoV-2 spike protein. CanSino Biologics is developing a vaccine that involves snipping a bit of the virus’ genetic code off and coupling it with a harmless virus to provoke a SARS-CoV-2 immune response.

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Vaccination Vaccine Antibody Life Science 92