Drug Discovery World

OCTOBER 6, 2023

In celebration of the Nobel Prize for Medicine going to two of the early proponents of mRNA technology for creating therapeutics, Katalin Karikó and Drew Weissman, this week our round-up highlights the importance of genetics, genomics and gene editing in drug discovery.

Drugs 52

Drugs Gene Editing Genome Genomics 52

Drug Discovery World

OCTOBER 10, 2023

Over recent months, there have been a number of ground-breaking discoveries in drug discovery for mental health conditions. The article in the journal Psychopharmacology focuses on new data showing that ACD856, the lead clinical drug candidate in the NeuroRestore platform, exhibits antidepressant effects in various preclinical models.

Drugs 59

Drugs Gene Expression Gene Immune Response 59

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Drug Discovery World

SEPTEMBER 27, 2023

Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism. Unlike genetics, where DNA sequences remain constant, epigenetic modifications are cell type-specific and can change over time due to a balance of enzymatic processes.

DNA 115

DNA Genetics Gene Medicine 115

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?

Gene Expression 59

Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

AUGUST 1, 2023

Synthetic biology is increasingly important to drug discovery and development. Evonetix will use the funding to brings its semiconductor chips to commercial scale and extend gene assembly capabilities for its binary assembly technology to deliver accurate, gene-length DNA in a benchtop instrument.

Drugs 52

Drugs Gene Expression Engineer Gene 52

Drug Discovery World

OCTOBER 4, 2023

Scientists have discovered the link between gene expression, DNA methylation, and brain structural changes in depression, revealing new potential therapeutic targets. The post Researchers uncover molecular pathways underlying depression appeared first on Drug Discovery World (DDW).

Research 52

Research Gene DNA Gene Expression 52

Drug Discovery World

AUGUST 16, 2023

For example, women whose primary tumours have a higher presence of immune cell genes, and genes linked to their activity, are more likely to respond to docetaxel than carboplatin. Although immune features remained relatively stable, RNA-based features related to DNA damage repair pathways changed significantly.

Genetics 52

Genetics Gene Hormones RNA 52

pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets.

Genome 59

Genome Genomics Gene Gene Expression 59

XTalks

MAY 12, 2021

In this episode, Ayesha discusses a new tool that uses CRISPR to modulate gene expression without editing DNA sequences. Dubbed “CRISPRoff,” the technology targets the epigenome to silence genes involved in diseases, with applications in cancer, AML and other conditions with a heritable component.

Life Science 52

Life Science Gene Expression Gene DNA 52

XTalks

MARCH 13, 2024

Therapeutic Developments Related to Glaucoma The introduction of new classes of glaucoma medications, such as prostaglandin analogs, nitric oxide-donating drugs and Rho kinase inhibitors, has added to the pharmacological options available for managing IOP.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

pharmaphorum

MARCH 10, 2021

Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, the gene therapy has been given the all-clear by bluebird’s scientists after reviewing the case of AML that emerged in a trial of the drug in sickle cell disease. Shares in bluebird ticked up following the announcement.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Roots Analysis

JANUARY 14, 2024

With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally. Like all drugs, biologics are regulated by the FDA.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Roots Analysis

NOVEMBER 3, 2023

NAMPT inhibitors are a class of drugs that target the enzyme nicotinamide phosphoribosyltransferase (NAMPT). NAD + is involved in a wide range of cellular processes, including energy metabolism, DNA repair, and gene expression. Several trials focused on evaluation of such drugs were discontinued in the past.

Development 40

Development Drugs Trials FDA Approval 40

Drug Discovery World

APRIL 16, 2023

Additional analyses are ongoing to identify biomarkers potentially associated with better outcomes, including gene expression profiles and PD-L1 expression. The post Personalised mRNA cancer vaccine prolongs survival in melanoma appeared first on Drug Discovery World (DDW).

Vaccination 52

Vaccination Vaccine Immune Response Gene Expression 52

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

pharmaphorum

JANUARY 29, 2021

‘Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. billion in funding.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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? and Preparing for new-era drug modalities with technology. appeared first on Drug Discovery World (DDW). which covers three written for Volume 23 – Issue 4, Fall 2022 of DDW.

Gene Expression 52

Gene Expression DNA Genome Genomics 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? And Preparing for new-era drug modalities with technology. appeared first on Drug Discovery World (DDW). which covers three written for Volume 23 – Issue 4, Fall 2022 of DDW.

Gene Expression 52

Gene Expression DNA Genome Genomics 52

Drug Discovery World

AUGUST 1, 2022

This technology defines gene expression patterns from rare cells harbouring latent HIV by generating millions of single-cell reaction containers in the form of water-in-oil emulsions, in which messenger RNA capture and virus DNA detection can be performed sequentially while maintaining segregation among cells.

Gene Expression 52

Gene Expression Scientist Gene Allergies 52

Drug Discovery World

OCTOBER 17, 2023

DDW Editor Reece Armstrong speaks to Benedikt Nilges , Head of Technology and Data Analytics at OMAPiX about spatial biology’s use in drug discovery and bettering our understanding of disease. This limits its efficacy in later stages of drug development or in the discovery and characterisation of biomarkers.

Gene Expression 52

Gene Expression Drugs Protein Gene 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. As such, teams are now researching a variety of medical applications for mRNA-based drugs. Reece Armstrong explores the potential and future opportunities for mRNA-based therapies.

RNA 52

RNA Protein Vaccination Vaccine 52

Delveinsight

JULY 30, 2020

The drug, UCB0107, is designed to impede or decrease the buildup of tau proteins in the brain, which leads to nerve cell damage and death. Omega’s platform is designed to adjust gene expression to healthy levels rather than switching genes on and off. GSK culls a host of pipeline meds after failures, prioritization.

Genome 52

Genome Genomics Antibody Gene 52

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. parvovirus B19, Flaviviridae, Ebola, and Coronaviridae), DNA viruses (e.g., It also enables the downregulation of gene expression by directly knocking out the cytoplasmic mRNA transcripts.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

DECEMBER 15, 2022

Gene editing tools such as zinc finger nucleases, transcription activator-like effector nucleases (TALEN) and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases have been heralded for their enormous potential to treat diseases and genetic disorders. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. In the last few years, researchers have become interested in using in vitro transcribed (IVT) mRNA as a drug delivery agent.

RNA 40

RNA Protein Genetics Gene Expression 40

Drug Discovery World

JANUARY 23, 2023

Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2. Base editing uses a guide RNA in partnership with a nickase version of Cas9, mutated to cut only a single strand of DNA, along with a deaminase enzyme to enable alteration of a single nucleotide 23,24.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

APRIL 14, 2023

Whilst the cause of DIPG isn’t known, the disease is characterised by specific mutations in genes coding for histone proteins – a family of basic proteins which can associate with DNA due to their positive charges. The post Democratising proteomics for cancer and beyond appeared first on Drug Discovery World (DDW).

Protein 52

Protein Genome Genomics DNA 52

The Pharma Data

DECEMBER 27, 2020

The drug, Yeliva (opaganib) is a first-in-class, oral spingosine kinase-2 (SK2) selective inhibitor with triple-action effect on the pathophysiological processes linked to COVID-19. The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer.

Trials 52

Trials Antibody Gene Therapy Clinical Trials 52

The Pharma Data

OCTOBER 19, 2020

CTCs enable the complete picture of a cancer to be seen, as, being an intact cell, they allow DNA, RNA and protein analysis and thus provide comparable analysis to a tissue biopsy. In addition, the live CTCs harvested can be cultured, which offers the potential for testing response to drugs outside the patient.

Protein 40

Protein Gene Expression RNA DNA 40

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. Drugs, Testing and Vaccines. Drug Approvals.

Life Science 111

Life Science Vaccination Vaccine Gene 111