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?

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Drug Discovery World

NOVEMBER 29, 2022

Using artificial intelligence, researchers at Chalmers University of Technology, Sweden have succeeded in designing synthetic DNA that controls the cells’ protein production. . The technology can contribute to the development and production of vaccines and drugs for severe diseases much faster and at lower cost. .

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Medical Xpress

NOVEMBER 22, 2022

A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates gene expression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.

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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.

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Drug Discovery World

MARCH 21, 2023

Diana Spencer explores the different ways the drug discovery sector is tackling colorectal cancer. So, what are the recent developments and how is the drug discovery sector tackling this common cancer? Improved diagnostics Diagnostics and screening are essential to the early treatment of colorectal cancer.

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XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Visium Spatial Gene Expression Solution. Feature barcoding using gel beads. Single Cell Data Analysis.

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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.

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Drug Discovery World

FEBRUARY 14, 2024

A class of molecules playing a crucial role in the regulation of gene expression and other cellular processes can restore normal skin structure rather than producing a scar, a University of Manchester-led study suggests. The post Small RNAs promote wound-healing without scarring appeared first on Drug Discovery World (DDW).

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pharmaphorum

JANUARY 13, 2022

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases. It’s not the big pharma’s first foray into the transcription factor area.

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Pharmaceutical Technology

AUGUST 11, 2022

Alnylam has reported that it aims to develop a therapy that targets the INHBE gene and reduces the overall risk of T2D and coronary heart disease for at-risk patients. Targeting gene expression that results in increased abdominal fat and leads to obesity is a key factor for the reduction of T2D and wider metabolic syndrome.

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Drug Discovery World

DECEMBER 12, 2022

DDW highlights the most interesting global news and comments within the drug discovery and development sector this month. . Everyone enjoys a good news story and the drug discovery and development sector is no exception. The post The Dose: DDW’s drug discovery highlights appeared first on Drug Discovery World (DDW).

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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.

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The Pharma Data

FEBRUARY 24, 2022

UNC researchers now show that these two factors can directly associate with one another, modulating cancer-cell-specific programs of gene expression. ” They found that EZH2 possesses two different binding patterns on chromatin in acute leukemia cells, eliciting two distinct gene-regulatory programs (Figure 1). .”

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Drug Discovery World

AUGUST 16, 2023

Triple negative cancers are not driven by any of the three molecules that can be blocked by targeted hormone receptor drugs – the HER2 protein and two hormone receptors for oestrogen and progesterone – and so current treatment options are limited.

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pharmaphorum

NOVEMBER 22, 2022

Cambridge, Massachusetts-based FogPharma has closed another impressive fundraising round, adding $178 million to the pot as it ushers its polypeptide-based drug candidates towards the clinic. Ut comes just as FogPharma is preparing to start clinical trials of alpha-helical polypeptide candidate FOG-001.

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The Pharma Data

MARCH 3, 2022

Use of the drug verapamil to treat Type 1 diabetes continues to show benefits lasting at least two years, researchers report in the journal Nature Communications. The suggestion that verapamil might serve as a potential Type 1 diabetes drug was the serendipitous discovery of study leader Anath Shalev, M.D.,

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Drug Discovery World

SEPTEMBER 27, 2023

Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism. Epigenetics: the software of gene regulation Gene activity is the driving force behind our biological processes, and disruptions in normal gene activity can trigger diseases.

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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.

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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.

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Drug Discovery World

AUGUST 3, 2022

Emerging as a new treatment option in rare and orphan disease areas, oligonucleotide therapeutics have matured into a drug class with a broad indication spectrum. Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake.

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pharmaphorum

JANUARY 29, 2021

Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. billion in funding.

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Pharmaceutical Technology

JUNE 13, 2023

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. GlobalData forecasts total sales from this drug class to exceed $1bn by 2029.

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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. Its job is to carry coding information that is essential to the translation and processing of functional proteins. mRNA is a molecule made up of a single strand of ribonucleic acid (RNA).

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Drug Discovery World

MARCH 26, 2024

UCDs are a group of rare, severe, inherited metabolic diseases impacting protein metabolism. Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 targets carbamoyl phosphate synthetase 1 (CPS1), a key enzyme that catalyses the first step of the urea cycle.

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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. .

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Roots Analysis

AUGUST 31, 2023

Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate.

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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.

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Pharmaceutical Technology

JANUARY 24, 2023

The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome. Developed with the University of Edinburgh, the EXACT self-contained technology enables MeCP2 protein therapeutic levels while avoiding overexpression-related toxicities.

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Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.

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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. To do this, Nautilus will useits protein analysis platform to provide TGen with data on the proteins involved in DPIG.

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Roots Analysis

OCTOBER 22, 2023

Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence gene expression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.

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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. GlaxoSmithKline, the U.S.

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Drug Discovery World

APRIL 21, 2023

Atrandi Biosciences’ novel technology platform enables researchers to gain insights into the biology of single cells, offering immediate applications in single-cell sequencing, drug and antibody discovery, functional metagenomics, microbial analysis, directed evolution, and synthetic biology. It will be led by Björn C.

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Drug Discovery World

FEBRUARY 6, 2023

A key breakthrough area where big data analytics has shown tremendous scope and application is in systems biology, which has bolstered holistic drug research and development, ultimately to provide patients with access to the right therapies. This could be expanded to assessing predictors at baseline.

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Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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The Pharma Data

OCTOBER 19, 2020

ANGLE’s proven patent protected platforms include a circulating tumor cell (CTC) harvesting technology and a downstream analysis system for cost effective, highly multiplexed analysis of nucleic acids and proteins. The system is epitope independent and can capture all types of CTCs as well as CTC clusters in a viable form (alive).

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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.

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