pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. billion in funding.

Gene Editing 140

Gene Editing Gene Gene Therapy In-Vivo 140

The Pharma Data

JUNE 7, 2023

Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

Gene Therapy 40

Gene Therapy Gene Gene Editing In-Vivo 40

Roots Analysis

JANUARY 14, 2024

Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. They are different from small molecules in terms of their size and complexity.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

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.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

Drug Discovery World

JULY 5, 2023

For example, in CAR-T-cell therapy, scientists can take T-cells from a patient and ex vivo (outside the body) genetically engineer them to effectively target and kill cancerous cells, then infuse them back in to the patient. Mark Garner, PhD., RA: Please can you tell us a bit more about your role at Agilent?

Research 98

Research Protein Scientist Immune Response 98

XTalks

DECEMBER 13, 2023

The CRISPR system is dubbed to be molecular “scissors” that can cut out parts of genes that are abnormal, deactivating or replacing them with new strands of normal DNA. The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel).

Gene Therapy 98

Gene Therapy FDA Approval Gene In-Vivo 98

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. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

RNA 52

RNA Protein DNA In-Vivo 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

Drug Discovery World

NOVEMBER 15, 2022

Rapid and accurate DNA synthesis . Conventional DNA synthesis, typically used for making sequences such as primers, employs phosphoramidite chemistry, a method that was developed in the 1980s and is still considered the gold standard for DNA synthesis. .

DNA 98

DNA Drugs Antibody Engineer 98

Drug Discovery World

APRIL 25, 2024

Grand View Research states that the market for cell lines is currently bolstered by the fact that the majority of biopharma companies use cell lines to synthesise recombinant monoclonal antibodies and proteins. It is a crucial for the development of biopharmaceuticals since they use a living host to produce the therapeutic in question.

Development 59

Development Protein In-Vivo Life Science 59

Drug Discovery World

NOVEMBER 9, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’.

Engineer 52

Engineer Antibody Protein In-Vivo 52

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. These genetic switches assist transcription factors in binding to the promoter region.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. 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

DECEMBER 15, 2022

These platforms all induce double strand cuts in the chromosomal DNA that can be sealed by the cell, leading to a specific gene disruption, or resulting in a new target site for inserting DNA segments. Reduce/eliminate chromosomal disruptions caused by double-strand DNA breaks. In one example, Choi et al.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Drug Discovery World

NOVEMBER 30, 2023

This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It The next presentation attended in this track was the keynote presentation, which was given by Kate Smith, PhD, Head of UK Protein & Cell Sciences at GSK. GSK has developed high-throughput mammalian and E.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Drug Discovery World

JULY 18, 2023

Rolf Turk , Senior Manager, Genomics Medicine at Integrated DNA Technologies, examines how CRISPR is being used to enhance cancer therapies. Years of development and fast-paced research have continued to unlock its potential, expanding how CRISPR can be used to treat, detect, and prevent common diseases such as cancer and Covid-19.

DNA 98

DNA Genome Genomics RNA 98

Drug Discovery World

JANUARY 12, 2023

The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. Altering one base pair can drastically change how the gene’s protein product functions, while mutations can lead to the development of a range of genetic disorders. . What are organoids? .

Gene Editing 98

Gene Editing Gene Drugs In-Vivo 98

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

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

JANUARY 17, 2024

Brett Swansiger, Chief Commercial Officer at ANGLE discusses the importance of liquid biopsies in precision medicine trials for cancer. As such there is a need to develop fast and accurate methods to assess drug safety and efficacy, facilitating early success or failure.

Medicine 59

Medicine DNA Genome Genomics 59

Roots Analysis

AUGUST 8, 2023

Furthermore, a wide variety of drugs and macromolecules, including DNA , proteins, and imaging agents, can be encapsulated in liposomal vesicles due to their unique ability to entrap both lipophilic and hydrophilic substances. Bangham, in 1961 at the Babraham Institute, University of Cambridge.

Drug Delivery Systems 52

Drug Delivery Systems Drug Delivery Drugs In-Vivo 52

The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

Drug Discovery World

FEBRUARY 2, 2023

RRx-001 directly and irreversibly binds to a specific amino acid cysteine residue on a protein complex called the NLRP3 inflammasome, which serves as the “on switch” for inflammation. A late-stage clinical trial in head and neck cancer for the treatment of severe oral blistering or oral mucositis caused by radiation therapy is planned.

Manufacturing 52

Manufacturing Clinical Trials Clinical Trials Drugs 52

The Pharma Data

AUGUST 3, 2021

Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

MARCH 20, 2023

Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post-dosing, children who were treated after presenting symptoms of SMA maintained all previously achieved motor milestones.

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

Drug Discovery World

JANUARY 23, 2023

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

Gene Editing 52

Gene Editing Gene Engineer DNA 52

The Pharma Data

OCTOBER 14, 2020

VBL has recently demonstrated ex-vivo activity of anti-MOSPD2 antibodies in patients with relapsing-remitting and progressive multiple sclerosis (MS), as well as in animal models of rheumatoid arthritis (RA), nonalcoholic steatohepatitis (NASH) and inflammatory bowel disease (IBD). 1. -- -->. -- [if lte IE 8]--> .

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing Trials 52