Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. When it gives the green light, H3K4me3 allows RNA polymerase II to move along DNA, transcribing it into RNA as it moves.

Gene Expression 52

Gene Expression Gene DNA RNA 52

XTalks

JUNE 20, 2022

Alnylam Pharmaceuticals, a leading RNA interference (RNAi) therapeutics biopharmaceutical company, announced it received approval from the US Food and Drug Administration (FDA) for its RNAi therapeutic Amvuttra (vutrisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (ATTR) amyloidosis in adults.

FDA Approval 98

FDA Approval Protein Genetics RNA 98

Roots Analysis

FEBRUARY 23, 2022

Further, NGS library preparation kits offer several benefits, such as low requirement of starting material, generation of longer RNA / DNA sequences and production of superior quality nucleotide libraries, thereby, catering to existing unmet needs of the research domain. Next Generation Sequencing (NGS) Library Preparation Kits.

Genetics 52

Genetics DNA RNA Reagent 52

pharmaphorum

JANUARY 26, 2023

In March, the collaborative T2T consortium published the first complete telomere-to-telomere sequence of the human genome, filling in the last 8% of the 3 billion base pairs that make up our DNA. Understanding individual drug reactions and metabolism Pharmacogenomics is emerging as an important area.

Genome 128

Genome Genomics Genome Project Genetics 128

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. What are the challenges of drug discovery in this area? How do they work?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. GBM is a WHO grade IV brain tumour with dismal prognosis. “Put

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

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.” .

Gene Editing 98

Gene Editing Gene Gene Therapy Genetics 98

The Pharma Data

MAY 9, 2022

No drugs are approved for these aggressive tumors, which occur in up to 20% of cases and can lead to patient disability or even death. The drug is a newer cancer treatment called abemaciclib. The paper was published today in Nature Genetics. ” Magill also is a member of the Robert H.

Drugs 52

Drugs Scientist DNA Genetics 52

Roots Analysis

AUGUST 15, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic predispositions, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

Marketing 40

Marketing Genetics Genome Genomics 40

Roots Analysis

FEBRUARY 21, 2022

Advancement in DNA sequencing technologies have resulted in noteworthy developments in various healthcare-related research fields, such as diagnostics and personalized medicine. As per our analysis, majority of the kits use purified RNA / DNA as the input sample. Diverse Landscape of NGS Library Preparation Kits. Web: [link].

Marketing 52

Marketing Gene Sequencing DNA Genomics 52

Roots Analysis

SEPTEMBER 9, 2024

It is worth highlighting that in 2023 alone, close to 20 protein-based drugs were approved for the treatment of myriad of disease indications including oncological and metabolic disorders. This process initially involves transcription of DNA to messenger RNA (mRNA). The mRNA is then translated into to form functional proteins.

Protein 40

Protein Gene Production DNA 40

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.

Genome 98

Genome Genomics Development Genetics 98

XTalks

NOVEMBER 30, 2023

In 2022, the US Food and Drug Administration (FDA) granted approval to the medication Sunlenca (lenacapavir) for adults living with multi-drug resistant HIV-1 infection. Developed by Gilead, Sunlenca is the first drug in a novel class known as capsid inhibitors to receive FDA approval for treating HIV-1.

FDA Approval 111

FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

Drug Discovery World

OCTOBER 16, 2023

The United Kingdom has established itself as a hub for drug discovery expertise worldwide. 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.

Drugs 75

Drugs Life Science Gene Therapy Genome 75

Delveinsight

FEBRUARY 25, 2021

Beam adds to drug delivery stable with USD 120 Million GuideTx buy. 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. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

XTalks

APRIL 6, 2022

Cancerous cells and tumors release small fragments of DNA into the bloodstream called circulating tumour DNA (ctDNA). The researchers at the Cancer Research UK Cambridge Institute published this data from the LUng cancer CIrculating tumour DNA (LUCID) study in the journal Annals of Oncology.

DNA 105

DNA In-Vitro Doctors Doctor 105

pharmaphorum

OCTOBER 12, 2022

Now, he leads Whitelab Genomics as its artificial intelligence (AI) platform powers the development of genomic therapies – an emerging field in which genetic sequences are injected into cells to target and repair damaged genes. The payload, Del Bourgo tell us, is the therapeutic DNA or RNA sequence that will cure or fix damaged cells.

Genome 104

Genome Genomics Medicine Development 104

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.

RNA 59

RNA Protein Gene Expression Vaccine 59

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). RNU4-2 is very active in the developing brain, and changes in this gene affect how the cell processes other RNA molecules, including those that go on to make proteins.

Genetics 52

Genetics Genome Project Genomics Genome 52

Drug Discovery World

JULY 11, 2023

Drug discovery and development timelines can span 10-15 years or more from initial discovery to market approval and typically require the analysis of massive amounts of data. How AI and ML are influencing drug development Biopharma has experienced significant growth in using these technologies for the past 10 years.

Big Data 40

Big Data Development Life Science Drugs 40

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. Messenger RNA (mRNA) is a single-stranded ribonucleic acid transcribed from the DNA chain, carrying the encoding information for protein synthesis.

In-Vitro 130

In-Vitro Genetic Disease Drugs RNA 130

Drug Discovery World

MAY 15, 2024

DNA vectors compared to standard linear mRNA-based expression. “We Following these results, we can rapidly advance to design and test circVec in several AAV and DNA-based vectors. AATD is a challenging genetic disease to treat. In recent experiments, Circio has observed up to four months circVec durability in vivo.

RNA 52

RNA In-Vivo Gene Therapy Genetic Disease 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. Though some genetic mutations are harmful, others can be protective.

RNA 52

RNA Drugs Gene Silencing Vaccine 52

The Pharma Data

DECEMBER 5, 2020

5, 2020 — A pair of new gene therapies promise a potentially lasting cure for sickle cell disease by subtly altering the genetic information in patients’ bone marrow cells, researchers report. David Williams, uses a virus to introduce RNA into the extracted bone marrow that turns off BCL11A. Food and Drug Administration.

Gene Therapy 52

Gene Therapy Gene Gene Editing Genetics 52

The Pharma Data

OCTOBER 19, 2020

“Molecular profiling is a powerful tool that Winship’s physicians can utilize to optimize cancer treatments by accessing relevant genetic information that can have a meaningful impact on patient care at a personalized level.” Brille , Vice Chairman of Caris Life Sciences.

Life Science 52

Life Science Clinical Trials Clinical Trials RNA 52

Drug Discovery World

JUNE 27, 2024

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. Dr Ataide added: “SeekRNA can precisely cleave the target site and insert the new DNA sequence without the use of any other proteins.

Gene Editing 59

Gene Editing Gene DNA Genetic Engineering 59

Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA 40

RNA Protein Genetics Gene Expression 40

Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

Genetics 40

Genetics Gene Expression RNA DNA 40

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.

Vaccine 96

Vaccine Vaccination Doctors Doctor 96

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials FDA Approval 52

XTalks

DECEMBER 13, 2023

Related: Top 30 Pharma Companies in 2023: Statistics and Trends 1) Moderna Compound annual growth rate: 415 percent Moderna, headquartered in Massachusetts, is a prominent biotechnology company specializing in RNA therapeutics, particularly mRNA vaccines. This substantial investment is accelerating the growth of their new drug pipeline.

Genetics 111

Genetics Vaccine Vaccination DNA 111

Drug Discovery World

NOVEMBER 15, 2022

Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. . It has made its way into almost every aspect of drug discovery, whether it’s exploring basic biology, modelling diseases, identifying and validating targets, or performing drug screens. .

DNA 98

DNA Drugs Antibody Engineer 98

XTalks

NOVEMBER 7, 2022

Liquid biopsy tests in oncology involve isolating entities such as circulating tumor cells (CTC), circulating tumor DNA (ctDNA) and tumor-derived exosomes. The cfDNA that originates from tumors is called circulating tumor DNA (ctDNA). Also, exosomes are mostly being used for the detection of circulating RNA and microRNA. “An

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Roots Analysis

MAY 21, 2024

Over the last decade, one of the major challenges faced by pharmaceutical companies across the globe is low drug solubility. In fact, it has been observed that around 40% of the pharmaceutical drugs approved by regulatory organizations exhibit poor bioavailability / solubility.

Drug Delivery 40

Drug Delivery Drugs Gene Therapy Bioavailability 40

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). As such, teams are now researching a variety of medical applications for mRNA-based drugs.

RNA 52

RNA Protein Vaccine Vaccination 52

Worldwide Clinical Trials

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195

Genetics Protein Gene Genotype 195

Roots Analysis

JANUARY 28, 2024

Pharmaceutical Research: Big data analytics can be applied in various areas of pharmaceutical research, including drug discovery and development, precision medicine , clinical trial optimization, pharmacovigilance and drug safety, supply chain optimization, and real-time monitoring and surveillance.

Big Data 40

Big Data Medicine Marketing Clinical Trials 40