Scienmag

NOVEMBER 16, 2020

Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).

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In-Vivo RNA DNA Genome 53

WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

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In-Vivo Clinical Trials Clinical Trials FDA Approval 52

Drug Discovery World

FEBRUARY 15, 2024

The etherna paradigm for cLNP design is ushering in the rapid generation of novel families of delivery systems for not only vaccination but also for in vivo tolerisation for a host of autoimmune diseases.” The post LNP formulations produce strong immune responses, data shows appeared first on Drug Discovery World (DDW).

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Immune Response In-Vivo Vaccination Vaccine 52

Drug Discovery World

JANUARY 23, 2023

This action sensitised GBM cells to radiotherapy both in vitro and in vivo (in mouse models). “Our research has revealed cladribine as a radiosensitiser for GBM treatment by drug repurposing, which can offer multiple advantages,” says Prof Youn. “As GBM is a WHO grade IV brain tumour with dismal prognosis.

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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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RNA In-Vivo Gene Therapy Genetic Disease 52

Delveinsight

FEBRUARY 25, 2021

Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.

DNA 52

DNA In-Vivo Genetics Medicine 52

Drug Discovery World

JANUARY 23, 2023

Following the release of Clarivate’s report ‘RNA Technology Companies to Watch’, DDW’s Diana Spencer speaks to Strand Therapeutics’ Jacob Becraft and HAYA Therapeutics’ Samir Ounzain to find out how this technology could revolutionise disease treatment.

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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

The Pharma Data

DECEMBER 13, 2020

About EdiGene, Inc EdiGene is a biotechnology company focused on leveraging the cutting-edge genome editing technologies to accelerate drug discovery and develop novel therapeutics for a broad range of genetic diseases and cancer. She started her career as a General Surgeon at Zhongshan Hospital of Chongqing. Source link.

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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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pharmaphorum

JUNE 28, 2021

So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.

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pharmaphorum

JANUARY 17, 2023

The intracellular barriers include endosomal escape, RNA sensors, and endonucleases. Ben Hargreaves speaks to Liberate Bio, a company that is hoping to address the delivery issues that could prevent mRNA, as well as siRNA and ASO therapeutics, from reaching their full potential.

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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

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

SEPTEMBER 6, 2022

This area of medicine, which turns our own bodies into agents of combat to fight disease, has quickly become one of the most promising fields in treating deadly diseases such as cancer, central nervous system disorders and even musculoskeletal conditions. . Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7

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Gene Therapy Gene Manufacturing Medicine 52

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.

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Gene Editing Gene In-Vivo Genome 52

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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Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

JULY 18, 2023

Incorporating in vivo pooled CRISPR screens has allowed for the interrogation of thousands of genetic perturbations in cells, leading to the determination of the involvement of molecules (PTPN2, ADAR1, or DHX37) in immune checkpoint inhibitor (ICI) responsiveness and T cell activation.

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Drug Discovery World

JANUARY 23, 2023

In cell therapy, regenerative and adoptive immunotherapies continue to bridge the gap between genetic engineering strategies and their clinical application. This circumvents several technical challenges inherent to the genetic manipulation of differentiated cells in ex vivo culture or intact tissues.

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Gene Editing Gene Engineer DNA 52

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation. GeneTx and Ultragenyx are co-developing the therapy.

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Marketing Gene Drugs Protein 52

The Pharma Data

NOVEMBER 22, 2020

Peer went onto say that he believes that, in the near future, there will be many personalized treatments based on genetic messengers. “Operationally, you design a stretch of 20 [nucleotide] base pairs that match a gene that you want to edit,” George Church, a professor of genetics at Harvard Medical School, told the news source.

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Gene Editing DNA Genome Genomics 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. .

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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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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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