pharmaphorum

JANUARY 29, 2021

California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).

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Gene Editing Gene Gene Therapy In-Vivo 144

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

AUGUST 19, 2022

Along with beti-cel, there is one being developed for sickle cell (approved in Europe as Skysona or eli-cel) and another being jointly developed with Bristol Myers Squibb for melanoma. But bluebird bio has said such setbacks are expected for a company at the frontier of developing gene therapies for rare diseases.

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Gene Therapy FDA Approval Gene Clinical Trials 95

Drug Discovery World

NOVEMBER 28, 2023

Mission Therapeutics develops therapeutics that promote mitophagy, which enables the removal of dysfunctional mitochondria, thereby improving cell health and function. Importance of healthy mitochondrial function Dysfunctional mitochondria are usually tagged for removal via mitophagy with a protein ‘flag’ called ubiquitin.

In-Vivo 52

In-Vivo Protein Drugs Research 52

The Pharma Data

JANUARY 11, 2021

a San Diego-based biotechnology company with an array of technology platforms for antibody discovery and optimization, and novel NK and T cell engager generation, today announced licensing of a panel of its anti-SARS-CoV-2 antibody clones to IGM Biosciences for COVID-19 therapy development.

Antibody 52

Antibody Licensing Licensing Development 52

Drug Discovery World

FEBRUARY 22, 2024

in 2023, fuelled by the FDA approval of its CRISPR-based ex vivo cell therapy Casgevy. Regeneron’s market capitalisation growth was attributed to the FDA approval of its high-dose Eylea – a VEGF fusion protein – for eye diseases, which may likely offset standard Eylea’s expected sales decline following looming biosimilar competition.

Marketing 52

Marketing FDA Approval In-Vivo Sales 52

Drug Discovery World

OCTOBER 27, 2023

Organ-chips combine cell culture with microfluidics to emulate the biological forces of different organ tissues and/or disease states, allowing pharmaceutical researchers to determine a drug candidates’ efficacy and toxicity ahead of clinical trials. Could organ-chips replace other research methods? Not yet, according to Ewart.

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Research Gene Therapy In-Vivo In-Vitro 72

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 mutation causes red blood cells to develop a crescent or “sickle” shape. The mutation causes red blood cells to develop a crescent or “sickle” shape.

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Gene Therapy FDA Approval Gene In-Vivo 98

Camargo

APRIL 28, 2021

The first tasks in striking this balance are dose selection and dosing interval determination, and this blog post offers an overview on how PK modeling and simulation can be applied to optimize the safety and efficacy of a product for pediatric populations in clinical trials. First Steps Toward Initiating Pediatric Clinical Studies.

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Clinical Trials Clinical Trials In-Vivo Drugs 130

XTalks

AUGUST 17, 2020

Immunotherapy continues to be a burgeoning field, with immune-based therapies being among the leading treatments being developed in oncology and autoimmunity. protein-based biologics) and vaccine treatments. protein-based biologics) : are those which bind to specific components (i.e. Immune system modulators (i.e.

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Protein Engineer Immune Response In-Vivo 98

pharmaphorum

APRIL 23, 2021

The alliance is based on Obsidian’s cytoDRIVE platform, which uses already approved small-molecule drugs alongside gene-editing medicines to control the expression of protein from a gene sequence. For example, adding a small-molecule might stabilise the medicine, allowing levels of the target protein to increase.

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Gene Editing Genetics Drugs Gene 52

XTalks

FEBRUARY 27, 2024

XTALKS WEBINAR: Development of CAR-T Cells for T-ALL Targeting CCR9 Live and On-Demand: Wednesday, March 6, 2024, at 11am EST (4pm GMT/UK) Register for this free webinar to explore the developments in cancer immunotherapy targeting CC chemokine receptor 9 (CCR9), a potential breakthrough for T-cell acute lymphoblastic leukemia (T-ALL).

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Delveinsight

JANUARY 18, 2021

The first sign of a child suffering from Angelman syndrome is a delay in development, such as the inability to sit without support or making incoherent babbling sounds. The gene is responsible for the normal and healthy development of an individual physically as well intellectually. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 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.

FDA Approval 52

FDA Approval Drugs In-Vivo In-Vitro 52

The Pharma Data

MARCH 25, 2022

2-4 Evusheld was generally well-tolerated in the trial.(2-4). 2-4 Evusheld was generally well-tolerated in the trial.(2-4). There is a growing body of evidence from multiple independent in vitro and in vivo (animal model) studies supporting the potential of Evusheld to protect against the BA.1,

Antibody 52

Antibody In-Vivo Medicine Vaccination 52

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. days; 95% C.I, days compared to placebo [5.7

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Clinical Trials Clinical Trials Trials In-Vivo 40

Drug Discovery World

OCTOBER 4, 2022

William Erhardt , President, Head of Development & Operations of biotech Oligomerix, tells Lu Rahman about the company’s differentiated approach to developing disease-modifying therapeutics for neurodegenerative diseases. . With its recent award of $3.35 In the US alone, AD prevalence is expected to increase from 6.5

Development 52

Development In-Vivo Compound Screening Clinical Development 52

The Pharma Data

OCTOBER 29, 2020

OSE-230 is an agonist antibody against ChemR23, also known as chemerin chemokine-like receptor 1 (CMKLR1), a G-protein coupled receptor (GPCR) expressed on myeloid immune cells known to modulate inflammation. NANTES, France, Oct. Most anti-inflammatory agents act using a mechanism that blocks pro-inflammation pathways.

Antibody 52

Antibody In-Vivo Development Clinical Trials 52

Delveinsight

JANUARY 12, 2021

Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. Birinapant is a clinical-stage SMAC mimetic that degrades Inhibitors of Apoptosis Proteins (IAPs) by binding to them, ultimately leading to cell death in tumor cells.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

MARCH 9, 2023

This includes the measurement of plasma kallikrein protein levels and clinical activity as determined by HAE attack rate measures. The post FDA clears in vivo gene editing therapy for trials in US appeared first on Drug Discovery World (DDW).

In-Vivo 52

In-Vivo Gene Editing Gene Trials 52

Drug Discovery World

JULY 22, 2022

For example, in randomised clinical trials of patients with non-small cell lung cancer (NSCLC), pembrolizumab has been shown to be effective in slowing down disease progression and improving survival rates 1. However, adverse effects such as cytokine release syndrome (CRS) and neurotoxicity still occur. Jespersen et al.

In-Vivo 52

In-Vivo Engineer In-Vitro Antibody 52

The Pharma Data

DECEMBER 10, 2020

The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response. The vaccine targeted both the inner nucleocapsid (N) and the outer spike (S) proteins of the virus to maximize the immune response.

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Vaccination Vaccine Immune Response Protein 52

Roots Analysis

SEPTEMBER 5, 2023

Moreover, there is always a high risk of failure associated with research and development (R&D) programs and, considering the increasing regulatory stringency, the approval of new drugs has become significantly more difficult. In recent years, quantum computing technology potential has evolved to revolutionize various industries.

Drugs 52

Drugs In-Vivo Development Research 52

Delveinsight

DECEMBER 21, 2020

These develop from star-shaped glial cells (astrocytes and oligodendrocytes) responsible for providing nourishment to the nerve cells within the brain. Research has shown that most of the cannabinoids bind to G-protein coupled cannabinoid receptors, CB1 and CB2, and act as agonists or inverse agonists. What causes cancer to relapse?

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials In-Vitro 52

Drug Discovery World

SEPTEMBER 28, 2022

AI-based diagnostic software and clinical trials technology proved to be two of the most attractive health investments, alongside mental health and wellness and medical imaging companies. . Artificial intelligence (AI) is notorious for its industry hype, touted to revolutionise the way multiple sectors operate over the next five to 10 years.

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

Drug Discovery World

FEBRUARY 22, 2024

Libra Therapeutics is a San Diego-based biotech focused on developing novel disease-modifying therapeutics, which restore the cellular balance that is disrupted in neurodegenerative diseases. Libra’s pipeline targets mechanisms that can mitigate protein pathology associated with CNS degenerative disorders.

Clinical Trials 52

Clinical Trials Clinical Trials Trials 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. . And whilst clinical trials activity decreased by around 15% compared to 2020, last year still saw a number of regenerative medicines reach the bedside.

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Drug Discovery World

AUGUST 4, 2022

Developability in the biopharmaceutical industry relates to the difficulty of making early candidate molecules with attractive biological activities into commercialised and marketable therapies. The ‘developability gap’ is the degree of uncertainty when the information needed to make such assessments is lacking at earlier stages of a process.

Development 52

Development Protein Manufacturing Drugs 52

Drug Discovery World

MARCH 20, 2024

Around 30% of drugs show adverse reactions during human clinical trials despite promising pre-clinical studies. One of the major causes of the high attrition rate is the poor predictive value of current preclinical models used in drug development. billion for bringing a new drug to market, a process extending over a decade.

Research 59

Research Clinical Trials Clinical Trials In-Vivo 59

Pharmaceutical Technology

FEBRUARY 15, 2023

They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins. According to GlobalData, there are 190+ companies, spanning technology vendors, established pharmaceutical companies, and up-and-coming start-ups engaged in the development and application of gene-splicing using nucleases.

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Gene Splicing Gene Recombinant DNA Technology DNA 130

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

JANUARY 23, 2023

Cargene Biopharma A company developing highly specific, stable, potent, siRNA-based oligonucleotide therapeutics via the company’s OSCAR algorithm and SMoP pharmacophore design platform.

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RNA Genome Genomics Engineer 98

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

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

JANUARY 10, 2021

Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. 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.

Medicine 52

Medicine Development Clinical Trials Clinical Trials 52

Drug Discovery World

DECEMBER 15, 2022

Now, after nearly 35 years of research and more than a decade of preclinical progress, several different gene editing modalities are being tested in early phase clinical trials. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 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. Furthermore, oncology clinical trials are frequently challenged by low enrolment rates, failure to achieve primary endpoints, study design complexity and limited funding 5.

Medicine 59

Medicine DNA Genome Genomics 59