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 141

XTalks

FEBRUARY 27, 2024

TIL vs CAR T-Cell Therapies While CAR T-cell therapies involve inserting a CAR engineered to target a specific cancer cell protein into a patient’s T cells, TIL-based T-cell therapies like Amtagvi involve separating TILs from a patient’s tumor (in autologous therapies) and expanding them ex vivo.

FDA Approval 52

FDA Approval Manufacturing In-Vivo Trials 52

Drug Discovery World

JANUARY 23, 2023

Researchers have discovered a clinical drug that sensitises glioblastoma (GBM) cells to radiotherapy and could replace the current standard of care. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1. 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

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.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

XTalks

APRIL 22, 2022

Flow cytometric receptor occupancy assays are being increasingly used in preclinical and clinical studies. Both the areas of drug development and clinical trials are increasingly using in vitro assays to help determine the efficacy of an investigational therapeutic. What is Flow Cytometry?

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Clinical Trials Clinical Trials Trials Antibody 98

Drug Discovery World

JULY 5, 2023

It’s a brilliantly exciting time where we’re beginning to harvest the fruits of decades’ worth of breakthrough research undertaken by the world’s brightest scientists, and we’re seeing those advances in basic science translated to the clinic, where they’re making a difference in patients’ lives. Mark Garner, PhD.,

Research 98

Research Protein Scientist Immune Response 98

XTalks

AUGUST 17, 2020

Cue Biopharma is a Cambridge, Massachusetts-based clinical-stage biotechnology company that is focused on developing injectable immune biologics using its proprietary Immuno-STAT (Selective Targeting and Alteration of T cells) platform. protein-based biologics) and vaccine treatments. Cell-Based Immunotherapy vs. Immune Biologics.

Protein 98

Protein Engineer Immune Response In-Vivo 98

XTalks

DECEMBER 13, 2023

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Both gene therapies are approved for individuals 12 years of age and older with sickle cell disease. How do Casgevy and Lyfgenia Work?

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Gene Therapy FDA Approval Gene In-Vivo 98

Drug Discovery World

FEBRUARY 22, 2024

With no in-house laboratory space, the company works closely with multiple international contract research organisations (CROs), including Axxam, which carry out all of the Libra R&D, from compound screening assays, to in vivo pharmacokinetic/pharmacodynamic and ADME work, as well as in vitro and in vivo pre-clinical safety assessments.

Clinical Trials 52

Clinical Trials Clinical Trials Trials In-Vivo 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. demonstrated the ability to model clinical responses to ACT in autologous HIS mice 4. Jespersen et al.

In-Vivo 52

In-Vivo Engineer In-Vitro Antibody 52

The Pharma Data

OCTOBER 29, 2020

30, 2020 (GLOBE NEWSWIRE) — OSE Immunotherapeutics (ISIN: FR0012127173; Mnemo: OSE) presented preclinical efficacy data for novel agonist monoclonal antibody therapy, OSE-230, at the 2020 Federation of Clinical Immunology Societies (FOCIS) Annual Meeting being held virtually on October 28-31, 2020. NANTES, France, Oct.

Antibody 52

Antibody In-Vivo Development Clinical Trials 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

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.

Vaccination 52

Vaccination Vaccine Immune Response Protein 52

Delveinsight

JANUARY 12, 2021

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. million after Birinapant successfully becomes a part of the Phase I trials. million after Birinapant successfully becomes a part of the Phase I trials.

Licensing 52

Licensing Licensing Antibody Gene Therapy 52

Drug Discovery World

OCTOBER 4, 2022

New York-based Oligomerix is an emerging clinical-stage biotechnology company focused on developing disease-modifying therapeutics for neurodegenerative diseases characterised by aberrant tau protein ranging from rare tauopathies such as progressive supranuclear palsy (PSP) and frontotemporal dementia (FTD) to Alzheimer’s disease (AD).

Development 52

Development In-Vivo Compound Screening Clinical Development 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. . But has its application been as revolutionary as industry bods predicted? . The simple answer is no. The unavailability of good data .

Pharma Companies 52

Pharma Companies Drugs In-Vivo Genetics 52

The Pharma Data

JANUARY 11, 2021

Using its novel yeast display system and large diverse human antibody libraries, AvantGen has identified a panel of high affinity human monoclonal antibody clones that bind to two distinct epitopes on the receptor binding domain of the SARS-CoV-2 spike protein. These findings have been published in Frontiers in Immunology ( [link] ).

Antibody 52

Antibody Licensing Licensing Development 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. SVP, Chief Medical Officer, Novartis Gene Therapies. “We

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

Drug Discovery World

MARCH 20, 2024

Around 30% of drugs show adverse reactions during human clinical trials despite promising pre-clinical studies. 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.

Research 59

Research Clinical Trials Clinical Trials In-Vivo 59

Drug Discovery World

SEPTEMBER 6, 2022

And whilst clinical trials activity decreased by around 15% compared to 2020, last year still saw a number of regenerative medicines reach the bedside. Last year saw the sector surge, with levels of investment hitting an all-time high at $22.7 billion, compared to $19.9 billion, compared to $19.9 million price point per dose.

Gene Therapy 52

Gene Therapy Gene Manufacturing Medicine 52

Pharmaceutical Technology

FEBRUARY 15, 2023

They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins. In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases.

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Gene Splicing Gene Recombinant DNA Technology DNA 130

The Pharma Data

JANUARY 27, 2021

a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. The first US subjects in the PROOF-HD trial were enrolled in October 2020.

Clinical Trials 40

Clinical Trials Clinical Trials Trials In-Vivo 40

Drug Discovery World

SEPTEMBER 21, 2023

Kiave Yune Ho Wang Yin, PhD, Chief Business Officer at Transure Bioservices explores how humanized mouse models can be used to develop more relevant preclinical studies for IBD treatments and ultimately increase the likelihood of a treatment’s success in clinical trials.

Clinical Trials 59

Clinical Trials Clinical Trials In-Vivo Trials 59

XTalks

AUGUST 19, 2022

Beta thalassemia is a rare inherited blood disorder caused by mutations in the beta-globin gene leading to a reduced amount, or complete absence, of the beta chains of the oxygen-carrying hemoglobin protein in red blood cells. XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research.

Gene Therapy 95

Gene Therapy FDA Approval Gene Clinical Trials 95

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

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

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 30, 2023

This presentation covered three primary drivers for a protein expression model: speed, cost and sustainability. “It The next presentation attended in this track was the keynote presentation, which was given by Kate Smith, PhD, Head of UK Protein & Cell Sciences at GSK. GSK has developed high-throughput mammalian and E.

Antibody 59

Antibody Protein Engineer In-Vivo 59

Delveinsight

JANUARY 18, 2021

However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome. GeneTx and Ultragenyx are co-developing the therapy.

Marketing 52

Marketing Gene Drugs Protein 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

FEBRUARY 2, 2023

RRx-001 directly and irreversibly binds to a specific amino acid cysteine residue on a protein complex called the NLRP3 inflammasome, which serves as the “on switch” for inflammation. 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

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

Delveinsight

DECEMBER 21, 2020

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. The expressions of these receptors were detected in GBM cell lines – in ex-vivo primary cancer cells extracted and in-situ GBM tissue biopsies. What causes cancer to relapse?

In-Vivo 52

In-Vivo Clinical Trials Clinical Trials In-Vitro 52

The Pharma Data

MARCH 20, 2023

2,3 These data are among a Zolgensma data set being presented during the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, which also include, in part, real-world evidence data from the RESTORE registry. Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5

Gene Therapy 40

Gene Therapy Gene In-Vivo Licensing 40

Drug Discovery World

JULY 18, 2023

Current research is focused on designing CRISPR-enhanced CAR T cells that are resistant to immunosuppressive cytokines such as transforming growth factor-β (TGF-β) 2 , programmed cell death protein (PD-1) 3 , or other negative T cell regulators 4 (CTLA-4, LAG-3, and TIM-3), thus improving anti-tumour functions.

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DNA Genome Genomics RNA 98

Drug Discovery World

DECEMBER 13, 2023

Custom antibodies can be developed for pharmacokinetic assays and biodistribution assays, supporting therapeutic development by giving researchers data about the behaviour of their novel therapeutic in vivo. This article is sponsored by Fortis Life Sciences.

Life Science 59

Life Science Antibody Manufacturing In-Vivo 59

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