Pharmaceutical Technology

MAY 19, 2023

MT-302 is a TROP2-targeting in vivo chimeric antigen receptor (CAR) that has been designed to express in the myeloid compartment. The financing will help to fast-track the development of other in vivo programming candidates into clinical trials. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

MARCH 7, 2024

Ginkgo Bioworks has acquired Patch Biosciences, with the intention to strengthen its gene therapy services, cell therapy services, and RNA therapeutics services. The collaboration achieved its goals of enhancing the AAV production titres of Biogen’s gene therapy manufacturing processes.

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The Pharma Data

JUNE 7, 2023

Bayer strengthens gene therapy portfolio with lipid nanoparticle technology from Acuitas Therapeutics Bayer AG is joining forces with Acuitas Therapeutics, Inc., This delivery technology protects the messenger RNA (mRNA) payload after administration allowing it to be safely and effectively delivered into cells.

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Drug Discovery World

MAY 17, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields.

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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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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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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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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.,

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Genetics Medicine Gene Therapy Marketing 52

WCG Clinical

MAY 28, 2024

The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.

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Drug Discovery World

MAY 15, 2024

In vivo proof-of-concept for Circio Holding’s differentiated circVec platform approach to gene therapy has been demonstrated in two posters at the American Society of Gene & Cell Therapy (ASGCT) 2024 annual meeting. The in vivo data showed statistically significant higher expression level and durability for circVec 2.1

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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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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

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.

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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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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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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.

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Gene Gene Therapy Gene Expression Regulation 40

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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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.

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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).

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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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Roots Analysis

AUGUST 23, 2024

This adaptability was clear in coronavirus vaccine development using mRNA technology; after researchers had sequenced the virus’s RNA during January 2020, scientists were able create viable vaccine candidates within just months. This modern marvel wasn’t just used against COVID-19 but can be tailored now to fight any number of diseases.

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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

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

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.

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DNA Genome Genomics RNA 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.

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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.

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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.

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Gene Editing Gene Engineer DNA 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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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.

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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.

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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.

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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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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’.

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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

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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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