Pharmaceutical Technology

MARCH 28, 2023

Vertex Pharmaceuticals Cell and Genetic Therapies chief and executive vice-president Bastiano Sanna said: “We have multiple programmes in our T1D portfolio including VX-880 and VX-264, which are in the clinic, as well as our hypoimmune programme, in preclinical development.

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XTalks

SEPTEMBER 15, 2020

A vaccine developed by Swedish company Diamyd Medical has demonstrated significant treatment efficacy in a predefined genetic subgroup of individuals with type 1 diabetes in a Phase IIb clinical trial. The trial showed a preservation of function of insulin-producing ?-cells cells at 15 months post-diagnosis. cells (see video above).

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Medical Xpress

JANUARY 17, 2025

Patients are being offered transplants of genetically modified insulin-producing cells and the initial results are promising. A completely new way of treating type 1 diabetes is being tested at Uppsala University Hospital. We put a few questions to Per-Ola Carlsson, Professor of Medical Cell Biology, who is leading the study.

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Pharmaceutical Technology

JUNE 29, 2022

In 2021, Novo Nordisk Pharmatech, a leading pharmaceutical-grade insulin and Quats product supplier announced plans to enter the enzyme market. Novozymes has a long legacy of enzyme discovery and the ability to genetically engineer these speciality enzymes to be superior. Developing best-in-class speciality enzymes.

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Delveinsight

AUGUST 18, 2021

In Type 2 diabetes, the body cells become resistant to insulin. Several genetic and lifestyle factors are observed to be the factors for the development of Type 2 diabetes. The pancreas produces insulin that helps glucose get into cells to be used for energy. Insulin pumps are an alternative to self-injection.

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XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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Pharmaceutical Technology

DECEMBER 27, 2022

Drug pricing was a hot-button topic, and William Newton found that while e-commerce chains could cut insulin prices , any major change won’t happen overnight. In a data-backed analysis, she further explored how the most number of designations are awarded to cancer drugs , and genetic disorders have the largest share of orphan therapies.

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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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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

SEPTEMBER 13, 2021

Philadelphia, September 13, 2021—Researchers at Children’s Hospital of Philadelphia (CHOP) have demonstrated that an experimental device can improve blood sugar control in patients who developed diabetes after their pancreas was removed to treat their hyperinsulinism, a genetic disease in which the pancreas produces too much insulin.

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The Pharma Data

DECEMBER 17, 2020

7, 2020 — After starting a drug that’s officially approved to treat a type of blood cancer, a young man with type 1 diabetes was able to stop using insulin. He’s been off insulin since August 2018 — more than two years. This patient had a genetic mutation that ruxolitinib is known to work on.

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XTalks

JULY 15, 2024

Congenital HI is a genetic disorder that causes excessive insulin production by the pancreas, which lowers plasma or blood sugar. Agonist engagement of the receptor is involved in insulin and glucose regulation, and hence has therapeutic implications for diseases like diabetes and obesity.

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XTalks

JULY 15, 2024

Congenital HI is a genetic disorder that causes excessive insulin production by the pancreas, which lowers plasma or blood sugar. Agonist engagement of the receptor is involved in insulin and glucose regulation, and hence has therapeutic implications for diseases like diabetes and obesity.

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The Pharma Data

JULY 28, 2021

1 Obesity and insulin resistance are key drivers in the development of type-2 diabetes and often lead to cardiorenal complications, as well as liver disease. Obesity and insulin resistance remain key drivers in the development of type-2 diabetes and areas of significant unmet medical need.”. Yancopoulos, M.D., Yancopoulos, M.D.,

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Scienmag

MAY 4, 2021

The researchers believe that Brd4 could be used as a target for obesity and insulin resistance. Credit: Lin-Feng Chen A new study, published in JCI Insight, looks at how Brd4, a regulator of the innate immune response, influences diet-induced obesity. Approximately one-third of the adults and one in five children in the U.S.

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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. The genetic bleeding disorder is caused by the absence of blood clotting factor IX. Users can also manually log details such as blood glucose readings, meals, exercise or insulin doses.

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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. This early-onset type of diabetes differs from type-1 diabetes in that it occurs within the first six months of life and […].

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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?

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Rethinking Clinical Trials

AUGUST 18, 2022

If we accept and acknowledge that race is a social construct, not a category that is tied to biological or genetic differences, then what does that mean when we see clear differences and disparities in health outcomes for these populations? It leads to physiological changes, it leads to insulin resistance, changes in your immunology.

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pharmaphorum

AUGUST 24, 2021

It seems likely that the type 1 diabetes programme will be focused on developing cells that can secrete insulin in response to blood glucose levels – acting as a replacement for pancreatic beta cells that are destroyed in the autoimmune disease – which is already a focus of Vertex’ research.

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The Pharma Data

MAY 22, 2023

Improving access to Lilly insulin in the U.S. including capping out-of-pocket costs at $35 or less per month (effective March 2023) and reducing the list price of Lilly’s most prescribed insulins by 70% (effective Q4 2023). . – and women serving in nearly half (49%) of all management positions globally.

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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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pharmaphorum

JULY 28, 2022

Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity. “We are currently testing this hypothesis, with the goal of pursuing a development candidate targeting INHBE in the near future,” said Paul Nioi, head of Alnylam’s human genetics group.

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The Pharma Data

DECEMBER 31, 2020

Rhesus Macaque Genome Reference Includes 85 Million Genetic Variants. Researchers at Baylor College of Medicine , the University of Missouri and the University of Washington created a new reference genome assembly, identifying more than 85 million genetic variants in the rhesus macaque. Source link.

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pharmaphorum

AUGUST 11, 2020

Some of it is lifestyle, some of it is emotional, some of it is genetic – which is why last January, the Royal College of Physicians recognised obesity as a disease. “We Novo Nordisk is itself investing in such technology, and is aiming to introduce more connected devices, such as digital insulin pens, in the future.

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The Pharma Data

DECEMBER 15, 2020

The Phase III trial evaluated the use of the diabetes vaccine Diamyd ® , an antigen-specific immunotherapy based on the auto-antigen GAD (glutamic acid decarboxylase), to induce immunological tolerance and stop the autoimmune destruction of insulin producing cells. People with type 1 diabetes cannot produce insulin.

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The Pharma Data

APRIL 24, 2023

Amphastar expects to provide dedicated commercial investment for BAQSIMI with the goal of enabling more people on insulin to be prepared with a glucagon rescue treatment for severe hypoglycemia. BAQSIMI is the first and only nasally administered glucagon for the treatment of severe hypoglycemia in people with diabetes. million in 2022.

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The Pharma Data

DECEMBER 30, 2020

The makeup of this biome is largely genetically determined; however, it is heavily influenced by several factors such as whether we are born naturally (vaginally) or by cesarean section, if we were breastfed, our use of antibiotics , and our exposure to chemicals, pesticides, and other toxins.

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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. Duchenne Muscular Dystrophy. Prevalence: 1 in 3,500.

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Delveinsight

DECEMBER 29, 2020

X-linked myotubular myopathy (XLMTM) is a rare genetic neuromuscular disorder and leads to muscle weakness, respiratory failure, and early death. It is a complication associated with diabetic patients who take insulin and certain anti-diabetic tablets. Approximately 1 in 40,000 to 50,000 newborn males are diagnosed with the disease.

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FDA Law Blog

JANUARY 5, 2023

One example is the Warning Letter FDA sent to Medtronic in late 2021 concerning a vulnerability in insulin infusion pumps. Digital providers and manufacturers not only have the concerns of the FDA to consider, but those of the FTC as well.

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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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Delveinsight

AUGUST 20, 2020

Generating insulin-producing pancreatic beta cells from stem cells to substitute those damaged by the immune system is a promising approach for diabetes treatment. The team successfully protected HILOs from the immune system without genetic manipulation. Immune-evading cells with PD-L1 control diabetes in mice.

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Delveinsight

DECEMBER 10, 2020

The body’s immune system falsely attacks insulin-making beta cells in the pancreas in Type 1 diabetes. Scientists at the University of Utah School of Medicine have found a way to control that autoimmune response by aiming a protein that is vital for T-cell activation. The protein is known as OCA-B.

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Trialfacts

NOVEMBER 26, 2023

relating to cancer, tuberculosis, human immunodeficiency virus infection and acquired immune deficiency syndrome, or any genetic muscle diseases or disorders) Must not have hypersensitivity or known allergy to any medications Must be able to attend study visits at the research site over approximately 14 weeks (without interference of vacations etc.)

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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. foods for long periods of time, some of the DANGEROUS and HARMFUL side effects included infertility, immune problems, accelerated aging, faulty insulin regulation, and changes in major organs and the gastrointestinal system.

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The Pharma Data

APRIL 12, 2023

Novo Nordisk has developed expertise to differentiate stem cells into a wide array of cells that may be used to replace damaged and lost cells which could lead to a specific disease, such as insulin-producing beta cells in type 1 diabetes, as well as manufacturing capabilities to produce the cells at scale.

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