Scienmag

OCTOBER 23, 2020

Oncotarget recently published a novel format for recombinant antibody-interleukin-2 fusion proteins exhibits superior tumor-targeting properties in vivo which reported that here, the authors describe four novel formats for the L19-IL2 fusion Credit: Correspondence to – Dario Neri – dario.neri@pharma.ethz.ch

Recombinant Antibody 53

Recombinant Antibody Protein Antibody In-Vivo 53

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

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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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 […]. (..)

In-Vivo 52

In-Vivo Protein Research Development 52

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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In-Vivo Gene Editing Gene Therapy Gene 52

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 →

DNA 40

DNA In-Vivo Protein Gene 40

Pharmaceutical Technology

FEBRUARY 23, 2023

Biopharmaceutics is a scientific discipline that examines the interrelationship of the physicochemical properties of the drug, the dosage form in which the drug is given, and the route of administration on the rate and extent of systemic drug absorption ( Applied Biopharmaceutics and Pharmacokinetics, Shargel, Wu-Pong and Yu, 5th Edition ).

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Development Drugs Bioavailability In-Vivo 246

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

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.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

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. The cells are prepared outside of the body ( ex vivo ) and then infused into the patient.

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Gene Therapy FDA Approval Gene Clinical Trials 95

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.

In-Vivo 52

In-Vivo Production Protein Drugs 52

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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Clinical Trials Clinical Trials Trials Antibody 98

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.

Gene Editing 52

Gene Editing DNA Genome Genomics 52

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

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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Development Antibody Protein Drug Delivery Systems 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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Vaccination Vaccine 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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In-Vivo Packaging Packaging Biobanking 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 113

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

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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Clinical Trials Clinical Trials Trials In-Vivo 40

Roots Analysis

SEPTEMBER 5, 2023

Further, owing to lack of understanding of how drug molecules were likely to behave in vivo , many lead molecules that progressed into the clinical stages of development had to be discontinued when they failed to demonstrate adequate therapeutic benefit.

Drugs 52

Drugs In-Vivo Development Research 52

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

Pharmaceutical Technology

FEBRUARY 15, 2023

Cas, also called CRISPR associated proteins, are a family of nucleases such as Cas9, Cas12a, Cas12b, and CasX that process these sequences. 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: CRISPR nucleases.

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In-Vivo Genome Genomics Gene Editing 162

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

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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In-Vivo DNA Antibody Gene 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

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

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.

RNA 52

RNA Protein DNA In-Vivo 52

The Pharma Data

OCTOBER 14, 2020

VBL has recently demonstrated ex-vivo activity of anti-MOSPD2 antibodies in patients with relapsing-remitting and progressive multiple sclerosis (MS), as well as in animal models of rheumatoid arthritis (RA), nonalcoholic steatohepatitis (NASH) and inflammatory bowel disease (IBD). 1. -- -->. -- [if lte IE 8]--> .

Clinical Trials 52

Clinical Trials Clinical Trials Manufacturing In-Vivo 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.

Drug Delivery Systems 52

Drug Delivery Systems Drug Delivery Drugs In-Vivo 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.

Contract Manufacturing 40

Contract Manufacturing Protein Manufacturing Production 40

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

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.

Marketing 52

Marketing In-Vivo Protein In-Vitro 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. 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 Gene 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.

FDA Approval 52

FDA Approval In-Vivo Clinical Trials Clinical Trials 52