pharmaphorum

MARCH 11, 2021

Vertex Pharma has made its name with treatment for cystic fibrosis, but has big plans in cell and genetic therapies – and has just chalked up fast-track status from the FDA for a cell-based therapy for diabetes. . If VX-880 works, it could remove or reduce the reliance of patients with the disease on insulin injections.

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pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

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XTalks

JANUARY 21, 2025

There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals. Companion diagnostic for Pfizers hemophilia B gene therapy Beqvez (idanacogene elaparvovec-dzkt). The genetic bleeding disorder is caused by the absence of blood clotting factor IX.

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Pharmaceutical Technology

DECEMBER 22, 2022

The company is a clinical-stage biopharmaceutical startup that uses its artificial intelligence (AI)-enabled platform to measure brain biomarkers, including electroencephalogram (EEG) activity and behavioural patterns, wearable data, genetics and other factors, to drive targeted drug development in mental health. Civica JAAQ Sanofi.

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Scienmag

AUGUST 5, 2020

But when it does, it’s mostly due to a mutation in a single gene such as the KCNJ11 or insulin (INS). Credit: Tokyo Tech Diabetes seldom occurs in newborns–a condition known as neonatal diabetes.

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Delveinsight

FEBRUARY 10, 2021

Hutchinson Gilford Progeria Syndrome (HGPS) also refers to as progeria , is an extremely rare and fatal genetic disorder that results in premature aging and death. Progeria or HGPS is a rare genetic condition that causes children to age rapidly, starting in their first two years of life. What is progeria? What is the cause of progeria?

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Gene Genetics Insulin Production 97

Delveinsight

DECEMBER 10, 2020

The body’s immune system falsely attacks insulin-making beta cells in the pancreas in Type 1 diabetes. OCA-B, or B cell-specific Oct1/2 coactivator, binds to and controls about 150 genes involved in a process through which T cells are reactivated upon reencountering antigens they have earlier identified and memorised.

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The Pharma Data

JULY 28, 2021

Individuals with at least one inactive copy of the GPR75 gene had lower body mass index (BMI) and, on average, tended to weigh about 12 pounds less and faced a 54% lower risk of obesity than those without the mutation. 1 Strong associations were also seen with improvements in diabetes parameters, including glucose lowering. Yancopoulos, M.D.,

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Medicine Development Research Insulin 52

Pharma Packaging Solutions

JUNE 30, 2023

Hormones (such as insulin, glucagon, and growth hormone, to name a few) are produced by recombinant DNA, as are blood factors, thrombolytic agents, interferons, monoclonal antibodies, therapeutic enzymes, and more.

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Recombinant DNA Technology Packaging Packaging Gene Therapy 52

BioSpace

NOVEMBER 17, 2022

Editas Medicine is pausing its ocular gene therapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.

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XTalks

SEPTEMBER 17, 2020

Acromegaly Surrogate Endpoint: Serum growth hormone and serum insulin-like growth factor 1 (IGF-1) are acceptable surrogate endpoints for acromegaly clinical trials involving somatostatin analogs such as octreotide, lanreotide and pasireotide. Mutations in the gene encoding ?-Gal Prevalence: ABCG5 gene mutation: 1 in 2.6

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Roots Analysis

SEPTEMBER 9, 2024

The main components of protein expression are a vector (plasmid) containing the gene of interest, and a host cell, for instance mammalian or prokaryotic cell. Protein expression process can be divided into following steps: Transcription: The DNA sequence of a gene is transcribed into mRNA.

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Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. The team successfully protected HILOs from the immune system without genetic manipulation. The removal of an AdComm was seen as a positive.

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Gene Therapy Research Gene Antibody 52

Roots Analysis

JANUARY 14, 2024

Usually, the desired gene, such as human insulin gene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.

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The Pharma Data

MAY 13, 2021

The REAL problem lies in the fact that most of the foods we eat today have been Genetically Modified. food is “A food in which the gene from one species has been forced into the DNA of another species and the process itself creates all sorts of unpredicted side effects.”. A better definition of a G.M.O. When lab rats were given G.M.O.

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The Pharma Data

MAY 15, 2021

Now imagine the entire length of the field as the 300,000 years of human genetic evolution. So you can see that your genes haven’t had time to adapt to the embarrassing over-abundance of food that you have today. However your genetic code IS hardwired to go without food for short periods between times of plenty.

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pharmaphorum

SEPTEMBER 1, 2020

The first was insulin – Frederick Banting and colleagues managed to isolate insulin that could treat diabetes, up until that point a fatal condition. This knowledge of genetics and the underlying cause of many diseases, including cancer, has resulted in powerful new drugs.

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The Pharma Data

MAY 13, 2021

Insulin Resistance and Cancer Risk: An Overview of the Pathogenetic Mechanisms. Ectopic lipid storage and insulin resistance: a harmful relationship. Relationship between disease severity, hyperinsulinemia, and impaired insulin clearance in patients with nonalcoholic steatohepatitis. Reprod Toxicol. 2014 Nov;49:196-201.

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