pharmaphorum

JULY 5, 2022

Cell therapy specialist Mogrify has struck a deal with Japanese drugmaker Astellas to look at ways to deploy regenerative medicine to treat hearing loss caused by factors such as chronic exposure to loud noises. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide.

In-Vivo 92

In-Vivo Bioinformatics Genetics Licensing 92

Drug Discovery World

DECEMBER 12, 2023

AATD patients face a critical unmet need given the limited effectiveness of current standard-of-care options for either the liver or the lung manifestations of the disease,” said Dr Jeffrey Teckman, an expert in AATD from St Louis University School of Medicine and a long-time advisor to Korro.

RNA 52

RNA In-Vivo Genotype Genetics 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.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. Gene splicing using nucleases is used to design gene therapeutics for various genetic disorders.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

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 142

Gene Editing Gene Gene Therapy In-Vivo 142

The Pharma Data

JANUARY 10, 2021

11, 2021 (GLOBE NEWSWIRE) — Editas Medicine, Inc. EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. CAMBRIDGE, Mass.,

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Roots Analysis

JANUARY 14, 2024

In fact, biologics are anticipated to play a significant role in shaping the pharmaceutical industry and will eventually serve as an integral part of traditional medicines. Overview of Biologics Biologics refer to medicinal / therapeutic products that are either manufactured using living organisms or semi-synthesized from biological sources.

Contract Manufacturing 40

Contract Manufacturing Manufacturing Protein Bacteria 40

Drug Discovery World

MARCH 20, 2024

Traditional 2D cell cultures often fail to replicate the cellular complexity of human tissues and show deviations in the expression of human-specific antigens or key transporter proteins. Their adaptability for genetic modifications further positions them as an optimal tool for exploring the mechanisms of action of toxic compounds.

Research 59

Research Clinical Trials Clinical Trials In-Vivo 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. Croasdale-Wood will then participate in a keynote chat, interviewed by Miho.

Engineer 52

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. These efforts, along with many others, are opening doors for new possibilities in translational medicine.

DNA 98

DNA Genome Genomics RNA 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

Drug Discovery World

JANUARY 12, 2023

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. . Oksana Sirenko looks at the advantages and limitations of using organoids from human cell sources in drug development and drug screening. . What are organoids? .

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Gene Editing Gene Drugs In-Vivo 98

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

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. .

DNA 98

DNA Drugs Antibody Engineer 98

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. Cas-9 represents the Cas9 protein, an enzyme that cuts foreign DNA. “Then the RNA plus the protein [Cas9] will cut — like a pair of scissors — the DNA at that site, and ideally nowhere else.”.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

Drug Discovery World

JANUARY 23, 2023

Strand’s mRNA therapeutics enable precise control of the location, timing, intensity and duration of therapeutic protein expression for improved efficacy and lower toxicity. Our team has been focused on engineering mRNA so that it’s only used to express cargo protein in certain cells regardless of where the molecule ends up in the body.

RNA 98

RNA Genome Genomics Engineer 98

Drug Discovery World

NOVEMBER 8, 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.

Antibody 64

Antibody Engineer Protein Scientist 64

XTalks

AUGUST 17, 2020

protein-based biologics) and vaccine treatments. This typically involves taking T cells from the body, engineering chimeric receptor antigens (CAR) and inserting them into the cells, expanding the cells ex vivo and injecting them back into the body. protein-based biologics) : are those which bind to specific components (i.e.

Protein 98

Protein Engineer Immune Response In-Vivo 98

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

pharmaphorum

JANUARY 17, 2023

As it stands, mRNA vaccines and therapeutics can only provide benefits if they can reach the living cells and be processed into protein. 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.

Vaccination 82

Vaccination Vaccine In-Vivo In-Vitro 82

Drug Discovery World

JULY 22, 2022

In vivo models, particularly HIS models, allow preliminary investigation of cell-based therapeutic strategies—often in combination with other therapeutic modalities—to overcome the limitations and challenges of T cell-based immuno-oncology treatments and identify effective options for clinical investigation. Jespersen et al.

In-Vivo 52

In-Vivo Engineer In-Vitro Antibody 52

The Pharma Data

DECEMBER 9, 2020

Vigil’s pipeline strategy is to target these diseases by combining a high-level understanding of microglia physiology, disease genotyping, and patient phenotyping to identify specific genetic variations associated with microglial dysfunction. Li is a prominent neuroscientist and former professor at Johns Hopkins Medicine.

Marketing 52

Marketing In-Vivo Protein In-Vitro 52

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.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

Roots Analysis

SEPTEMBER 5, 2023

Moreover, traditional medicine was primarily derived from natural sources and the knowledge of such therapeutic remedies was passed down either through verbal communication or written records. It is also worth noting that the process of drug discovery is extremely demanding, both in terms of capital requirements and time.

Drugs 52

Drugs In-Vivo Development Research 52

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 million deaths worldwide” 1.

Drugs 59

Drugs Gene Clinical Trials Clinical Trials 59

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

Roots Analysis

OCTOBER 19, 2023

This discovery led by Alexander Fleming, started a new era in medicine, and eventually saved millions of lives. Bacteria plays a crucial role in maintaining the ecosystem balance. However, there are few species of bacteria that can cause several infectious diseases ( such as strep throat, salmonellosis, tuberculosis, whooping cough ).

Bacteria 40

Bacteria Production In-Vivo In-Vitro 40

Drug Discovery World

AUGUST 15, 2023

Although such electrophysiological systems may be more predictive of potential cardiac safety and efficacy than non-functional binding assays, many apparently attractive new drugs are still being discontinued later in development. The authors of this review introduced the concept of ‘Eroom’s Law’, which is Moore’s Law spelt backwards.

Drugs 52

Drugs In-Vivo Development Research 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. . billion, compared to $19.9 million price point per dose. Cancer is king .

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

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.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

XTalks

AUGUST 5, 2020

The detailed complexity of these processes can be captured by creating models that combine correlates of gene and protein expression, providing insight into the molecular composition of tissues. Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level.

Gene Expression 112

Gene Expression Development RNA Drugs 112