CRISPR Pioneer Goes Small to Transform Genetic Medicine with Epigenetic Engineering
BioSpace
JULY 11, 2022
Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.
Development Drugs Gene Expression Genetics 
Gene expression delivery tool ‘slides’ instructions into cells
Drug Discovery World
NOVEMBER 1, 2022
Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.
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This week in drug discovery (2-6 October)
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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Gene Therapy and Pharmacokinetics
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.
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Genetic biomarkers could personalise therapy for TNBC
Drug Discovery World
AUGUST 16, 2023
A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. The presence of these biomarkers could be used to accelerate the development of more personalised treatments for triple negative breast cancers.
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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders
Pharmaceutical Technology
JULY 29, 2022
In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.
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Combining the Powers of Single Cell Sequencing and AI in Understanding Disease Biology and Drug Development
XTalks
AUGUST 5, 2020
Single cell RNA sequencing technology has been extensively applied to understand heterogeneity among tumor cells, within the tumor microenvironment and during cell development. Visium Spatial Gene Expression Solution. On Visium Gene Expression slides, there are areas called capture areas, which have a lawn of barcode on them.
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New drug inhibits the growth of cancer cells
Scienmag
DECEMBER 22, 2020
Blocking gene expression in mitochondria in mice stops cancer cells from growing Credit: Hauke S. Hillen A newly developed compound starves cancer cells by attacking their “power plants” – the so-called mitochondria. The new compound prevents the genetic information within mitochondria from being read.
Epigenetics discovery could lead to new class of cancer drugs
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. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.
Turning science into business: Amplifying mRNA by targeting regRNAs
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?
Moving the needle of CAR-T beyond oncology
Drug Discovery World
FEBRUARY 27, 2024
DDW’s Megan Thomas speaks with Dr Blythe Sather, Vice President and Head of Research at Tune Therapeutics, about how genetic tuning will transform the reach for CAR-T therapies, the different diseases that can benefit, and the disruptive technology making this happen. MT: What is genetic tuning?
AI tool may help doctors select best drugs for COVID patients
pharmaphorum
NOVEMBER 12, 2021
Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. The post AI tool may help doctors select best drugs for COVID patients appeared first on.
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Colorectal Cancer Awareness Month: Drug discovery innovation
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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The epigenetic edge: Harnessing precision medicine’s potential
Drug Discovery World
SEPTEMBER 27, 2023
HKG Epitherapeutics is a biotechnology company developing novel tools for the early detection of cancer and promoting healthy aging. Embarking on a new era of medicine The advent of genomics has ushered in the era of personalised medicine, enabling us to analyse the genetic makeup of individuals with unprecedented accuracy.
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How transcriptomics is driving drug discovery
Drug Discovery World
FEBRUARY 13, 2023
Dr Stephen Barat, Senior Vice President and Therapeutics Division Head at HTG Molecular Diagnostics tells DDW how transcriptomics is driving drug discovery and precision medicines. RNA transcript expression levels can provide insight into underlying biology – eg. DDW: Could you explain the process of transcriptomics? SB: Absolutely.
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Webinar: How to achieve high-throughput single-cell sequencing
Drug Discovery World
APRIL 17, 2024
It will also feature an introduction by Melanie Masuda, Director of Market Development, ScaleBio. Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Register for free here.
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CRISPR screening: How to achieve 1 million cell data sets cost-effectively
Drug Discovery World
APRIL 11, 2024
It will also feature an introduction by Melanie Masuda, Director of Market Development, ScaleBio. Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Register for free here.
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CRISPR screening: Achieving high-throughput single-cell sequencing
Drug Discovery World
APRIL 3, 2024
It will also feature an introduction by Melanie Masuda, Director of Market Development, ScaleBio. Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Register for free here.
Genome 52
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CRISPR screening at scale: An instrument-free, cost-effective approach
Drug Discovery World
MARCH 27, 2024
It will also feature an introduction by Melanie Masuda, Director of Market Development, ScaleBio. Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel. Register for free here.
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Synthetic biology tools advancing and accelerating drug discovery efforts
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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Researchers uncover molecular pathways underlying depression
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. Parallel findings in humans underscored the involvement of interferon-related genes in major depressive disorder.
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Nucleome raises £37.5m to shine light on ‘dark genome’
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. Dr Danuta Jeziorska.
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Artificial intelligence could be new blueprint for precision drug discovery
The Pharma Data
JULY 12, 2021
online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. the success rates in drug discovery?are PreCSN), the computational arm of iNetMed.
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The Dose: DDW’s drug discovery highlights
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. And finally… . References .
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The future outlook for mRNA therapies
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 exists in every cell within the human body and this, along with its role in the development of proteins, are the reasons why mRNA therapies have such potential in treating many diseases.
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World Glaucoma Week 2024: New Advances in Glaucoma Care
XTalks
MARCH 13, 2024
Biomarker testing is being pursued to identify biological signs of the disease and provide an approach to develop personalized treatment plans for each patient. As this technology continues to evolve, it is expected to help identify glaucoma in its nascent stages of development.
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What to expect from SLAS Europe 2023
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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Biologics – The Next Step in Revolutionary Medication
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.
Delivering on the promise of gene editing
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. Likewise, Lessard et al.
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bluebird seeks gene therapy trial restart after cancer scare
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. bluebird bio’s Philip Gregory.
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2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the Life Sciences
XTalks
DECEMBER 24, 2020
Focusing on biologics, vaccine development and policy platforms in anticipation of an eventual outbreak is why we’re where we are today in terms of the positive side [of the pandemic].”. Drugs, Testing and Vaccines. Amidst the vaccine rush, there has also been a drive for finding old and new drugs to treat COVID-19.
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Angelman Syndrome Market: Insights Into The Recent Late-Stage Drug Failures And Novel Approaches To Treating This Rare Neurogenetic Disorder
Delveinsight
JANUARY 18, 2021
Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. GeneTx and Ultragenyx are co-developing the therapy.
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Vtama (tapinarof) Cream Gains FDA Approval for the Treatment of Plaque Psoriasis in Adults
XTalks
MAY 31, 2022
The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. The disease usually develops around 15 to 25 years of age and can recur at any point of time in the affected individual’s life. Clinical Trials of Vtama.
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Decibel Therapeutics Announces $82M Series D Financing to Advance Gene Therapies to Restore Hearing and Balance
The Pharma Data
NOVEMBER 8, 2020
BOSTON–( BUSINESS WIRE )– Decibel Therapeutics , a clinical-stage biotechnology company developing novel restorative gene therapeutics to treat hearing loss and balance disorders, today announced the closing of an oversubscribed Series D financing, which raised $82.2M. 9, 2020 11:00 UTC. partner at OrbiMed.
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How big data analytics can make personalised care a reality
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. Figure 2: Advanced data visualisation examples for transcriptomics data.
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CRISPR breakthroughs: New solutions for common diseases
Drug Discovery World
JULY 18, 2023
Since its discovery in 2012, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) technology has revolutionised the biomedicine and cell and gene therapy fields, providing a versatile tool for precise and efficient genome editing. CRISPR diagnostics have successfully detected various pathogens, including RNA viruses (eg.
DNA 98
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Gene editing: beyond the hype
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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The use of base editing in stem-cell based therapies
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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Democratising proteomics for cancer and beyond
Drug Discovery World
APRIL 14, 2023
The hope is that a better understanding of the entire set of proteins (proteome) behind DIPG could help researchers develop better therapies and diagnostics for the disease. These mutated proteins then reprogramme the epigenome, from which the cancer develops.
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Fulcrum Therapeutics Announces Pricing of Public Offering of Common Stock – Jan 20, 2021
The Pharma Data
JANUARY 19, 2021
Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 CAMBRIDGE, Mass., Forward-Looking Statements.
Clinical Catch-Up: December 21-25 | BioSpace
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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EHA 2023: Kura Oncology’s menin inhibitor ziftomenib shows impressive Phase I results
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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Gene Switch: A Novel Platform for Switching Genes On and Off
Roots Analysis
AUGUST 31, 2023
It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.
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Tezepelumab granted Priority Review by U.S. FDA
The Pharma Data
JULY 8, 2021
Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. Tezepelumab is being developed by Amgen in collaboration with AstraZeneca. executive vice president of Research and Development at Amgen. Reese, M.D., About Tezepelumab.
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