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.

Gene Therapy 40

Gene Therapy Gene Gene Editing RNA 40

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.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

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.

Research 98

Research Protein Scientist Immune Response 98

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

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.

Gene Therapy 98

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.

Development 59

Development Protein In-Vivo Life Science 59

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.

RNA 52

RNA Protein DNA In-Vivo 52

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 .

DNA 98

DNA Drugs Antibody Engineer 98

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 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

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 .

Gene Therapy 52

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? .

Gene Editing 98

Gene Editing Gene Drugs In-Vivo 98

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. This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It

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

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

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

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

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.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

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.

Manufacturing 52

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

Gene Editing 52

Gene Editing Gene Engineer DNA 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

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

OCTOBER 14, 2020

Priothera will use the funds to progress the clinical development of mocravimod, a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogeneic hematopoietic stem cell transplantation (HSCT) for treating AML. All jobs have been maintained and the workforce has grown from about 220 to 350.

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing Trials 52