Pharmaceutical Technology

FEBRUARY 14, 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. These systems include human and mouse cell lines, and even in vivo in live animals.

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In-Vivo DNA Antibody Gene 130

Drug Discovery World

APRIL 8, 2024

Aviv Regev, Head and Executive Vice President of Research and Early Development at Genentech, opened the session with a discussion of how advances in genomic sequencing are fostering the development of data-rich cell atlases and what that data could mean for new insights into cancer biology and novel therapeutics.

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In-Vivo In-Vitro Antibody Protein 52

Drug Discovery World

AUGUST 14, 2023

Over the past few decades, the incredible progress in personalised medicine has led to the development of life-saving cell and gene therapies (CGT). The agencies also have different requirements for the selection of healthy donors in the development of allogeneic cell therapies. billion in 2020 to $15.5

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Gene Therapy Marketing Gene DNA 52

pharmaphorum

FEBRUARY 19, 2021

Once someone is infected with HIV it stays with them for life thanks to the retrovirus’s ability to inject its DNA code into the host. The cash will also help develop two other preclinical drugs, EBT-104 for Herpes Simplex Virus and EBT-107 for hepatitis B. The post Could CRISPR cure HIV?

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Gene Editing In-Vivo DNA In-Vitro 105

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.

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Gene Editing Gene Gene Therapy In-Vivo 145

The Pharma Data

JUNE 7, 2023

a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics. 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.

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Gene Therapy Gene Gene Editing In-Vivo 40

Pharmaceutical Technology

MARCH 9, 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. However, not all innovations are equal and nor do they follow a constant upward trend.

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In-Vivo Antibody Genetics Gene 130

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. GuideTx will aid Beam in developing LNP-based medicines.

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DNA In-Vivo Genetics Medicine 52

pharmaphorum

NOVEMBER 24, 2021

Norwegian biotech Vaccibody has changed its name to Nykode Therapeutics, and celebrated its new identity with a sizeable deal with Regeneron to develop vaccines for cancer and infectious diseases. Nykode’s Vaccibody technology generates DNA plasmids directed against neoantigens, which are only found on tumours.

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Vaccination Vaccine In-Vivo Manufacturing 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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FDA Approval Drugs In-Vivo In-Vitro 52

Drug Discovery World

JULY 5, 2023

Global Cancer Segment Market Manager at Agilent Technologies, recently sat down with DDW Editor Reece Armstrong to tell us about the developments in the ‘golden age’ of cancer research. Mark Garner, PhD., RA: Please can you tell us a bit more about your role at Agilent? MG: I am responsible for Agilent’s cancer segment.

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Research Protein Scientist Immune Response 98

XTalks

DECEMBER 13, 2023

The landmark approvals were awarded to bluebird bio’s Lyfgenia (lovo-cel) and Vertex Pharmaceuticals and CRISPR Therapeutics’ jointly developed Casgevy (exa-cel). 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.

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Gene Therapy FDA Approval Gene In-Vivo 98

Drug Discovery World

APRIL 25, 2024

Reece Armstrong offers an overview of cell line development and outlines the benefits the technology is bringing to biopharmaceutical developers. Cell line development is an essential underpinning of the drug development process, enabling teams to test, optimise and manufacture therapeutics at a commercial scale.

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Development Protein In-Vivo Life Science 59

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. They are different from small molecules in terms of their size and complexity.

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Contract Manufacturing Manufacturing Protein Bacteria 40

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.

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Gene Splicing Gene Recombinant DNA Technology DNA 130

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. Regulatory bodies are also taking note of the applications of AI in drug development.

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Life Science Gene Editing Development Clinical Trials 119

Drug Discovery World

NOVEMBER 15, 2022

Known for its ability to offer precision and flexibility while expanding the experimental scale, synthetic biology tools can reduce development timelines and costs, and lend a competitive advantage over traditional methods. . Rapid and accurate DNA synthesis .

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DNA Drugs Antibody Engineer 98

XTalks

AUGUST 12, 2020

The identification of T cells that essentially ‘hide’ the virus has significant implications for developing therapeutic strategies to improve the treatment and long-term management of HIV. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

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RNA Protein DNA In-Vivo 52

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

Drug Discovery World

JANUARY 12, 2023

Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . The genome is the blueprint for life, consisting of genes made up of thousands of pairs of DNA bases. What are organoids? .

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Gene Editing Gene Drugs In-Vivo 98

The Pharma Data

NOVEMBER 29, 2020

NASDAQ: HOOK, ‘HOOKIPA’), a company developing a new class of immunotherapeutics based on its proprietary arenavirus platform, today announced positive interim efficacy results, as well as additional safety and immunogenicity data, for its prophylactic Cytomegalovirus (CMV) vaccine candidate HB-101. NEW YORK and VIENNA, Austria, Nov.

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Vaccination Vaccine Clinical Trials Clinical Trials 52

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

Roots Analysis

AUGUST 31, 2023

Gene switches can be regulatory proteins or specific DNA sequences that act to either switch on or off the expression of a gene. Basic Components of Gene Switch Gene switches are composed of noncoding DNA sequences and transcription factors. These genetic switches assist transcription factors in binding to the promoter region.

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Gene Gene Therapy Gene Expression Regulation 40

Drug Discovery World

NOVEMBER 9, 2023

The first three include: Sungjin Park, PhD, CEO, Onegene Biotechnology, on: ‘UniStac: Enzyme-mediated conjugation technology for accelerated development of tetraspecific NASH drug’. Klaus Wagenbauer, PhD, Founder & CEO, Plectonic, on: ‘Programmable DNA-origami-based T cell engagers: PTE’.

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Engineer Antibody Protein In-Vivo 52

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

Roots Analysis

MARCH 1, 2023

Disruption of the integrity and function of DNA at the gene or chromosomal level can lead to heritable mutations, resulting in genetic disorders, birth abnormalities, or cancer. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

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Genotoxicity DNA Genetics Bacteria 52

Drug Discovery World

JANUARY 17, 2024

With oncology accounting for a third of pharma’s product pipeline, significant challenges include fierce competition, high research and development costs over, 10-15 years for clinical development, and pressures to rapidly meet the demand for new treatments 2-4.

Medicine 59

Medicine DNA Genome Genomics 59

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. Through base editing, drugs replace single nucleotide in the DNA strand with another, without making double-strand breaks (DSBs) in the gene.

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Gene Editing Trials Drugs Gene 98

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.

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Drug Delivery Systems Drug Delivery Drugs In-Vivo 52

Drug Discovery World

NOVEMBER 30, 2023

Lightcast is developing a novel, programmable microfluidic platform that allows precise and highly flexible control of individual microdroplets using software-generated light patterns. GSK has developed high-throughput mammalian and E.

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Antibody Protein Engineer In-Vivo 59

Drug Discovery World

NOVEMBER 13, 2023

On Day 3, Thursday 16 November 2023, the tracks include: the second part of machine learning for protein engineering, antibodies against membrane protein targets, engineering bispecific antibodies, next-generation immunotherapies, protein stability and formulation, and protein process development.

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Protein Antibody Engineer Scientist 59

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. Pioneering work in the mid-2000s, led by the Yamanaka group at Kyoto University, further advanced the field of PSCs.

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Gene Editing Gene Engineer DNA 52

The Pharma Data

NOVEMBER 22, 2020

Peer stated that his team plans to develop a treatment for all cancers and that their technique could be ready for humans within the next two years. CTX001 is being developed under a co-development and co-commercial agreement between the two organizations. Cas-9 represents the Cas9 protein, an enzyme that cuts foreign DNA.

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Gene Editing DNA Genome Genomics 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.

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Gene Therapy Gene In-Vivo Licensing 40

Drug Discovery World

FEBRUARY 2, 2023

Bryan Oronsky , Chief Development Officer at EpicentRx, discucsses how one drug sourced from the defense and aerospace industry, could have many potential disese targets. 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.

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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

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Clinical Trials Clinical Trials Gene Therapy Trials 52

XTalks

OCTOBER 1, 2020

And although academia and industry have often been pitted against each other in the realm of scientific discovery and development, there may be more similarities and synergies between the two than we may think. WeiQi Lin, MD, PhD, executive vice president of research & development and principal scientist, DURECT Corporation.

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Scientist In-Vitro Research Life Science 52