Pharmaceutical Technology

APRIL 25, 2023

sCFH is an engineered and improved version of complement factor H (CFH) that can fit into adeno-associated virus (AAV) vectors with solid expression and applications confirmed in cultured human cells in vitro, in addition to various preclinical animal models in vivo.

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In-Vivo In-Vitro Sales Engineer 130

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 145

pharmaphorum

JULY 5, 2022

The two partners will take an in vivo approach to the problem of so-called sensorineural hearing loss (SNHL) looking at ways to replace sound-detecting hair cells in the inner ear (cochlea) that become damaged in this type of deafness. An estimated 1.57 Astellas is already working on other approaches to treating hearing loss.

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In-Vivo Bioinformatics Genetics Licensing 110

Pharmaceutical Technology

AUGUST 17, 2022

oRNA molecules have been demonstrated to possess increased stability in vivo compared to linear mRNA and can potentially create more quantities of therapeutic proteins within the body. . These programmes will include therapies and vaccines in infectious disease and oncology areas.

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RNA Vaccine Vaccination In-Vivo 147

Pharmaceutical Technology

FEBRUARY 23, 2023

Innovation S-curve for artificial Intelligence in the pharmaceutical industry In-silico drug discovery is a key innovation area in artificial Intelligence Drug research starts with the discovery stage that involves in vivo and in vitro models to shortlist lead drug compounds.

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Drugs DNA In-Vivo In-Vitro 189

Scienmag

JANUARY 25, 2021

These results will help researchers to identify at an early stage the people most at risk of developing the disease, well before the first cognitive symptoms appear Credit: ©ULiège/CRC In Vivo Imaging Study conducted by researchers from the GIGA CRC In vivo Imaging laboratory at ULiège demonstrates, for the first time in humans, how the […]. (..)

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In-Vivo Protein Research Development 52

Pharmaceutical Technology

FEBRUARY 14, 2023

Innovation S-curve for the pharmaceutical industry Mammalian expression vectors is a key innovation area in pharmaceutical Mammalian expression vectors are used to introduce a specific fragment of DNA into mammalian systems for RNA or protein expression. These systems include human and mouse cell lines, and even in vivo in live animals.

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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.

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Protein Engineer Immune Response In-Vivo 98

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 DNA Recombinant DNA Technology 130

pharmaphorum

JANUARY 10, 2022

The German group is paying $40 million upfront to kick off the alliance, which is focusing on the development of up to five CRISPR drugs that can be delivered in vivo for liver-targeted diseases. – are around one-third the size of the more commonly used Cas9.

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BioTech 365

SEPTEMBER 2, 2020

(Nasdaq: SRNE, “Sorrento”) and SmartPharm Therapeutics, Inc. (“SmartPharm”) announced today that Sorrento has completed the acquisition of SmartPharm, a gene-encoded protein therapeutics company developing non-viral DNA and … Continue reading →

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DNA In-Vivo Protein Gene 40

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. Conducting a clinical study in a pediatric patient population requires careful planning, as providing a benefit to a child without causing harm is a weighty responsibility.

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Clinical Trials Clinical Trials In-Vivo Drugs 130

The Pharma Data

JULY 2, 2023

Ferroptosis is characterized by the iron-dependent oxidative destruction of cellular membranes, which is counteracted by ferroptosis suppressor protein-1 (FSP1), one of the guardians of ferroptosis. Although FSP1 has been considered as an attractive drug target for cancer therapy, in vivo efficacious FSP1 inhibitors have been lacking.

In-Vivo 40

In-Vivo Protein Drugs Research 40

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

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.

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Vaccine Vaccination In-Vivo In-Vitro 97

FDA Law Blog

JUNE 25, 2024

Based on experience to date, along with currently available analytical technologies that can structurally characterize highly purified therapeutic proteins and model in vivo functional effects, FDA has decided to revise the Interchangeability Guidance such that switching studies are no longer required.

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Roots Analysis

AUGUST 23, 2024

Unveiling The Potential of mRNA Technology mRNA is a single-stranded molecule that relays the genetic instructions needed to make proteins from DNA in the cell nucleus to ribosomes. Ribosomes are cellular machines that read mRNA sequences and produce proteins. There are several advantages to approaching vaccination this way.

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Protein Vaccine Vaccination Immune Response 40

XTalks

APRIL 22, 2022

Flow cytometry is an analytical technique that is used to detect the presence of cell surface markers as well as intracellular proteins using antibodies in individual cells suspended in a buffer solution, which pass through a laser beam one at a time ( Figure 1 ). What is Flow Cytometry?

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The Pharma Data

NOVEMBER 22, 2020

Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.

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Gene Editing DNA Genomics Genome 52

The Pharma Data

JUNE 7, 2023

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. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.

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Gene Therapy Gene Gene Editing In-Vivo 40

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.

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Marketing Gene Drugs Protein 52

Roots Analysis

JANUARY 14, 2024

Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. They are different from small molecules in terms of their size and complexity.

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Contract Manufacturing Protein Manufacturing Production 40

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 Gene FDA Approval In-Vivo 98

The Pharma Data

DECEMBER 9, 2020

The company plans to do this by developing drugs that eliminate protein aggregation by either reducing the production of toxic proteins or removing them after the fact through autophagy. Atlas cofounded, seeded and incubated Vigil, with pre-clinical stage assets in-licensed from Amgen Inc., which will remain a key shareholder.

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Marketing In-Vivo Protein In-Vitro 52

Roots Analysis

SEPTEMBER 13, 2023

Conjugation is the process of formation of a single, stable hybrid, wherein one of the entities is a molecule, such as protein, antibody, peptide and small molecule. Bioconjugation is a subset of conjugation where one of the entity is a biomolecule, such as protein or an antibody.

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XTalks

AUGUST 12, 2020

Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. This may help explain why HIV is adequately controlled, but not eradicated, with current treatments. The research study was published in Cell Reports.

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RNA Protein DNA In-Vivo 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.

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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.

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Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

APRIL 22, 2023

The current COVID-19 vaccines are designed to trigger an antibody response to the SARS-CoV-2 spike protein, which is vulnerable to mutations that could make the vaccine less effective over time. In their study, the researchers challenged mice with a lethal dose of SARS-CoV-2 and found that 87.5%

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Vaccine Vaccination Protein Antibody 40

Roots Analysis

DECEMBER 8, 2021

Biopreservation is the exclusive biological approach designated for the preservation of cells, tissues and organs ex-vivo for the purpose of storage, shipment and transportation. Biopreservation media is one of the most crucial entity in conferring effectiveness. F) to 8°C (46.4°F).

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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] ).

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Antibody Licensing Licensing Development 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?

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In-Vivo Clinical Trials Clinical Trials In-Vitro 52

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.

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Drug Delivery Systems Drug Delivery Drugs In-Vivo 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.

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Gene Expression Development RNA Drugs 112

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.

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FDA Approval Manufacturing In-Vivo Trials 52

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. About Sickle Cell Disease Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS).

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Medicine Development Clinical Trials Clinical Trials 52

WCG Clinical

AUGUST 30, 2024

Data from the Alzheimer’s Association estimates that approximately 5.4 million Americans are living with Alzheimer’s disease today at an estimated annual cost of $183 billion dollars. Of Americans aged 65 and older, 1 in 8 has Alzheimer’s disease and nearly half of people aged 85 and older have the disease.

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