Drug Discovery World

OCTOBER 27, 2023

With cues from a diverse population of tissue-specific cell types, extracellular matrix proteins, and biomechanical forces, cells grown in organ-chips closely emulate their in vivo counterparts and replicate tissue-level functions. But can organ-chips entirely replace other models? Not yet, according to Ewart.

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The Pharma Data

DECEMBER 1, 2020

NASDAQ:NTLA), a leading genome editing company focused on developing curative therapeutics using CRISPR/Cas9 technology both in vivo and ex vivo , announced today the pricing of an underwritten public offering of 4,794,521 shares of its common stock at a public offering price of $36.50 CAMBRIDGE, Mass., Goldman Sachs & Co.

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Camargo

NOVEMBER 11, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Unpacking the (Black) Box: Antares Licenses Urology Product with Boxed Warning. of hyponatremia, or low blood sodium levels. In October, the U.S.

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Pharma in Brief

JUNE 14, 2022

OPSUMIT® is a tablet containing macitentan. The Court dismissed allegations that the asserted patent claims were invalid for obviousness, lack of utility, overbreadth, and insufficiency of disclosure. Background. Janssen Inc. Janssen ) markets OPSUMIT® in Canada for the treatment of pulmonary arterial hypertension ( PAH ). Sandoz Canada Inc.

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Drug Discovery World

AUGUST 14, 2023

For example, the FDA’s Center for Biologics Evaluation and Research (CBER) 3 , which regulates biological products for human use, differentiates between in vivo gene therapy (new genes are inserted into the body to target cells) and ex vivo gene therapy (cells are modified outside the body and then administered back into the patient).

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Camargo

APRIL 28, 2021

The typical pathway toward initiating pediatric clinical research begins by gathering data in preclinical in vivo studies and in adult healthy volunteers or patients. If the model does not contain covariates relevant for young patients, they are added to the model based on the known information.

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XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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The Pharma Data

APRIL 8, 2022

Every breath stretches the lungs’ tissues with each inhale and relaxes them with each exhale. The mere motions of breathing are known to influence vital functions of the lungs, including their development in babies, the production of air-exchange-enhancing fluid on their inner surfaces, and maintenance of healthy tissue structure. During his Ph.D.

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The Pharma Data

OCTOBER 29, 2020

The oral presentation entitled “ Agonist anti-ChemR23 mAb blunts tissue neutrophil accumulation and triggers chronic colitis inflammation resolution ” shows efficacy results for OSE-230 in chronic inflammatory preclinical models and ex vivo human models. 7 therapies.

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Drug Discovery World

OCTOBER 21, 2022

Not only is October Breast Cancer Awareness Month but October 21 is Wear it Pink 2022, Breast Cancer Now’s annual fundraising day. The last month has seen a number of leaps in our understanding of difficult to treat and treatment resistant breast cancers, so here are Drug Discovery World’s top 5 research breakthroughs.

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The Pharma Data

JANUARY 12, 2021

INCHEON, South Korea–( BUSINESS WIRE )– Celltrion Group today announced top-line results from its randomised, double-blind, and placebo controlled global Phase II/III clinical trial of CT-P59, an anti-COVID-19 monoclonal antibody treatment candidate. days quicker compared to placebo. Patients treated with CT-P59 (40mg/kg) recovered 3.4

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XTalks

AUGUST 17, 2020

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. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.

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The Pharma Data

JANUARY 12, 2021

Dr Graham is a medicines development expert and Infectious Diseases Epidemiologist with global Biotech and Pharma R&D experience in Phase I-IV therapeutics as well as in-vivo & in-vitro diagnostics, across many modalities. THIS ANNOUNCEMENT CONTAINS INSIDE INFORMATION FOR THE PURPOSES OF ARTICLE 7 OF REGULATION (EU) NO 596/2014.

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Life Science Antibody In-Vivo In-Vitro 52

The Pharma Data

APRIL 22, 2023

“To our knowledge, this study is the first to show in vivo [in a living organism] protection against severe COVID-19 by an AI-designed T-cell vaccine,” said Girish Kirimanjeswara, associate professor of veterinary and biomedical sciences, Penn State. The results were published on April 11 in Frontiers in Immunology.

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The Pharma Data

APRIL 22, 2023

These backpacks are loaded with anti-inflammatory molecules that direct the carrier cells’ differentiation into anti-inflammatory cells in vivo. The Atlas of MS reported in 2020 that someone is diagnosed with MS every five minutes around the world, adding to about 2.8 million individuals that currently have to live with the disease.

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The Pharma Data

JANUARY 11, 2021

Our antibody clones in their original IgG format have shown potent neutralization activity in in vitro assays and, in the case of our lead clone, in an in vivo animal model. Upon binding, the antibodies block the spike protein from interacting with ACE2 and thereby prevent virus-induced cell-killing, also known as cytopathic effect.

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Drug Discovery World

AUGUST 3, 2022

These peptides typically contain up to 30 amino acids and can be cationic, amphipathic or both. Currently, only a small number of CPP-based oligonucleotide delivery examples have been reported for in vivo application. Here, Bachem explores the science behind this approach. What are cell-penetrating peptides? Nanoparticles 6.

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Roots Analysis

SEPTEMBER 20, 2023

Cell therapies are based on the premise that the patient’s own cells ( autologous ), or those from a healthy donor ( allogeneic ), can be genetically re-programmed to combat various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.

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The Pharma Data

DECEMBER 27, 2020

Changes in heparan sulfate (HS) concentration in cerebrospinal fluid (CSF) are being monitored in patients treated with LYS-SAF302 to provide evidence of in vivo biological activity of the drug and demonstrate proof of concept. Average reductions were highly statistically significant. About Lysogene. Sarepta Therapeutics, Inc.

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The Pharma Data

DECEMBER 13, 2020

The company believes the clinical comparability requirement will be met if the time to neutrophil engraftment in patients from the company’s ongoing expanded access program (EAP) using omidubicel produced at Gamida Cell’s planned commercial manufacturing sites is consistent with the results achieved in the Phase 3 clinical trial. About Omidubicel.

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The Pharma Data

OCTOBER 21, 2020

An in vivo efficacy study examined the oral administration of KB-0742 in mice that had been engrafted with the human CRPC cell line and compared tumor growth to standard docetaxel chemotherapy (intraperitoneally at 15 mg/kg, once weekly), or escalating doses of KB-0742 (orally at 3, 10, and 30 mg/kg, once daily) over 21 days.

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The Pharma Data

DECEMBER 10, 2020

.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.

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The Pharma Data

JANUARY 4, 2021

Kiromic chPD1 has shown in preclinical data to show a cytotoxic response in 9 different in vivo models with 100% long-term PFS with the induction of host memory responses. chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T). About Kiromic.

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Pharmaceutical Technology

MAY 24, 2023

Autologous therapies are characterized as a circular supply chain; the patient’s own sample starts the supply chain, and it is then subjected to ex-vivo modifications with the resulting therapy administered back to that same patient. What are the general differences in the supply chain of CGT vs. traditional clinical trials?

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Drug Discovery World

NOVEMBER 7, 2023

A single whole slide image contains millions, if not billions, of pixels that can be analysed. DDW’s Megan Thomas looks at a day in the life of Richard Bouffard, Histology Director at Virscio. MT: Where do you work, and can you tell us what you’re working on at the moment? MT: Where do you think the biggest opportunities are in this field?

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Research In-Vivo Gene Therapy Development 59

Drug Discovery World

DECEMBER 15, 2022

Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo. In vivo approaches involve delivering the gene editing components directly into the patient. In one example, Choi et al.

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Gene Editing Gene In-Vivo Genome 52

Roots Analysis

SEPTEMBER 18, 2023

Due to the infrastructure available for processing, freezing and storing of these container systems, blood bags are the preferred container for cell therapy biopreservation. While some biobanks ( especially non-clinical ones ) use mechanical freezers for storage, liquid nitrogen is one of the most reliable cryostorage mediums.

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Roots Analysis

AUGUST 8, 2023

The hydrophobic portion contains two fatty acid chains with 10-24 carbon atoms, while the hydrophilic part primarily consists of phosphoric acid linked to a water-soluble molecule. Introduction to Liposome Liposome was initially identified and described as swollen phospholipid systems, called Banghasomes, by British hematologist Alec D.

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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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Roots Analysis

AUGUST 31, 2023

Further, the transcription factors contain two molecular domains, the DNA binding domain and the activation domain. Moreover, some transcription factors contain additional domains, such as ligand binding domain in order to interact with chemical signals. Figure below presents an overview of the mechanism of action of gene switch.

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The Pharma Data

JANUARY 19, 2021

a privately-held neuron regeneration therapeutics company, today announced the publication of results of their novel pharmacotherapy: NNI-362 to allosterically stimulate neural regeneration in human cultures and in vivo models of aging and disease. CLARKSVILLE, Md., 20, 2021 (GLOBE NEWSWIRE) — Neuronascent Inc. , About NNI-362.

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Roots Analysis

MARCH 1, 2023

coli , Salmonella typhi ), as well as its ability to synthesize an essential amino acid required for growth Mutation Assay: Transgenic rodent somatic and germ cell gene mutation assay specifies an in vivo assay that perceives gene mutation causing agents.

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The Pharma Data

JANUARY 10, 2021

EDIT-301 is an experimental, ex vivo gene editing cell medicine in development for the treatment of sickle cell disease. The Company is required to develop and submit to the FDA an improved potency assay prior to enrolling the efficacy phase of the RUBY trial. Enrolled patients will receive a single administration of EDIT-301.

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Drug Discovery World

FEBRUARY 1, 2024

From this enriched selection, promising candidates are identified only after each antibody is characterised both in vitro and in vivo. Time can mean more than money when you’re studying a deadly pathogen. When all is said and done, this process may take months or years to complete.

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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.

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Drug Discovery World

NOVEMBER 30, 2023

DDW’s Megan Thomas attended PEGS Europe 2023 in Lisbon, Portugal, from Tuesday 14 November to Thursday 16 November 2023. The company’s goal is to accelerate functional characterisation and shortenoptimisation time in antibody discovery and t-cell workflows. GSK has developed high-throughput mammalian and E.

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Pharma Marketing Network

JANUARY 20, 2021

Assuming these findings are replicated in clinical trials, this vaccine, with its more-appealing route of administration and convenient (refrigerator) storage requirements could make an important contribution to containing the disease.

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