Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. We were excited to unveil data showing that two distinct chemical series exhibit METTL1 inhibition in vitro at low nanomolar concentrations with minimal interference with other RNA and protein methyltransferases.

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RNA In-Vivo In-Vitro DNA 64

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. billion, compared to $19.9 Cancer is king .

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Gene Therapy Gene Manufacturing Medicine 52

Pharmaceutical Technology

FEBRUARY 14, 2023

Innovation S-curve for the pharmaceutical industry Mammalian expression vectors is a key innovation area in pharmaceutical Mammalian expression vectors are used to introduce a specific fragment of DNA into mammalian systems for RNA or protein expression. These systems include human and mouse cell lines, and even in vivo in live animals.

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XTalks

AUGUST 5, 2020

In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.

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Gene Expression Development RNA Drugs 112

Pharma Marketing Network

DECEMBER 21, 2020

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e.,

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

The Pharma Data

DECEMBER 13, 2020

“Our company and R&D portfolio are entering into an exciting phase, as evidenced by the recent close of Series B financing and submission of the first gene editing product IND in China,” said Dong Wei, Ph.D.?CEO Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.

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Business Development Development Gene Editing In-Vivo 40

Delveinsight

FEBRUARY 25, 2021

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. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. Beam employs three approaches to deliver its genetic medicines to cells.

DNA 52

DNA In-Vivo Genetics Medicine 52

pharmaphorum

JUNE 28, 2021

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

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In-Vivo Gene Editing Trials Drugs 105

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

AUGUST 3, 2022

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. This method is generally used to deliver small interfering RNA (siRNA), but it can also be used for larger oligonucleotides too. Oligonucleotide. Anti-miRNA.

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Bioavailability In-Vivo RNA In-Vitro 52

Drug Discovery World

SEPTEMBER 14, 2023

Moreover, in July 2023, AstraZeneca and Vaxess Technologies commenced a collaboration for the evaluation of a novel RNA-based pandemic influenza prototype vaccine in patch format. Business updates Pfizer reported second quarter 2023 revenues totalling $12.7 billion, a decrease of $15.0 Vaccines were up 9.1%

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Drugs Vaccination Vaccine Medicine 59

Drug Discovery World

DECEMBER 12, 2023

KRRO-110 is designed to co-opt an endogenous enzyme Adenosine Deaminase Acting on RNA (ADAR) to repair a pathogenic single nucleotide variant (SNV) on RNA and restore production of normal A1AT.

RNA 52

RNA In-Vivo Genotype Genetics 52

XTalks

AUGUST 12, 2020

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).

RNA 52

RNA Protein DNA In-Vivo 52

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

SEPTEMBER 25, 2023

RNA therapeutics company SiSaf has published positive safety and efficacy data for its siRNA therapy for Autosomal Dominant Osteopetrosis 2 (ADO2). The data demonstrate that siRNA complexed with SiSaf’s silicon stabilised hybrid lipid nanoparticles (sshLNP) was able to significantly downregulate expression of an ADO2-specific mutant gene.

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RNA In-Vivo Clinical Trials Clinical Trials 59

Drug Discovery World

SEPTEMBER 28, 2022

Artificial intelligence (AI) is notorious for its industry hype, touted to revolutionise the way multiple sectors operate over the next five to 10 years. Its application in pharma, most commonly in drug discovery, has been steadily growing with global partnerships between tech vendors and pharma companies becoming more commonplace.

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Pharma Companies Drugs In-Vivo Genetics 52

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.

DNA 98

DNA Genome Genomics RNA 98

pharmaphorum

JULY 13, 2022

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.

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Gene Editing Trials Drugs Gene 98

Drug Discovery World

APRIL 12, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

Drug Discovery World

JANUARY 20, 2023

Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates. Existing in vivo and in vitro models aren’t up to the challenge Preclinical tools exist for the evaluation and screening of potential drug candidates.

Development 52

Development In-Vivo Drugs In-Vitro 52

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.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Delveinsight

JANUARY 18, 2021

Angelman syndrome is a result of the missing or dysfunctional gene known as UBE3A. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system.

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Marketing Gene Drugs Protein 52

Drug Discovery World

OCTOBER 13, 2022

Prior to the pandemic, messenger RNA (mRNA) drug development was niche and predominantly pointed at different disease targets, including infectious diseases and cancers. Why mRNA has potential . One aspect that makes mRNA technology popular with drug developers is that its proteins can be easily edited. The rise of global demand .

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Drugs Manufacturing Vaccination Vaccine 52

Drug Discovery World

JANUARY 17, 2024

CTCs reflect high levels of tumour heterogeneity, represent clonal evolution, and are suitable for treatment selection, real-time longitudinal disease monitoring and in vitro/in vivo culture. Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer.

Medicine 59

Medicine DNA Genome Genomics 59

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. The product is the most advanced gene-editing approach in development for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease. . Photo courtesy of Science Advances.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Drug Discovery World

NOVEMBER 15, 2022

If you want to order gene-length sequences, rather than short oligos for PCR, the waiting times are around two or three weeks at least. Anita Ramanathan outlines four synthetic biology approaches that are improving and accelerating drug discovery. . Improving the quality of the DNA is as important as improving the speed of synthesis.” .

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DNA Drugs Antibody Engineer 98

Drug Discovery World

NOVEMBER 13, 2023

Noel Pauli, PhD, Group Leader, Antibody Engineering, Adimab, on: ‘Harnessing in vivo diversities with a yeast-based platform for the discovery of antibodies against multi-pass membrane proteins’. Ali Madani, PhD, Founder and CEO, Profluent Bio, on: ‘Protein engineering with large language models’.

Protein 59

Protein Antibody Engineer Scientist 59

Drug Discovery World

FEBRUARY 22, 2024

According to Medicine Discovery Catapult, these include: “Navigating the complexity of the CNS; understanding the disease pathology, and accessing predictable in vitro and in vivo cell models 2 ”. Dementia is the only major cause of death without a treatment to prevent, slow or stop disease progression. “In In 2019, dementia accounted for 1.8

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

Drug Discovery World

MAY 10, 2023

Until recently, drug carcinogenicity assessment required two different in vivo studies, one of which was typically a two-year lifetime study in rats. Dr Megan MacBride and Dr Caroline Horizny Mitchell , Taconic, examine recent changes to animal testing in drug design. billion and take over 10 years.

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Bioassay Genotoxicity Drugs In-Vivo 52