Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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Research Gene Gene Therapy Genome 264

Pharmaceutical Technology

JUNE 4, 2023

There are two main genes in the AAV genome, rep and cap, which encode nine different proteins. Transcription of the rep gene is initiated from the p5 or p19 promoters to, respectively, produce the large (Rep78 and Rep68) and small (Rep52 and Rep40) nonstructural Rep proteins.

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Gene Gene Therapy Genetics DNA 262

XTalks

JUNE 23, 2022

Ayesha also discussed the FDA approval of Alnylam’s RNAi therapeutic Amvuttra for the treatment of polyneuropathy associated with a rare protein disorder called hereditary transthyretin-mediated (ATTR) amyloidosis. Amvuttra RNAi Therapeutic Wins FDA Approval for Rare Genetic Protein Disorder.

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Life Science FDA Approval Protein Drugs 52

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. According to the FDA, life expectancy for those with DMD has increased over the years, with some patients surviving beyond 30 years.

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FDA Approval Genetics Gene Therapy Gene 105

XTalks

MAY 7, 2021

As Novavax awaits emergency use authorization (EUA) from the US Food and Drug Administration (FDA) for its protein-based COVID-19 vaccine NVX-CoV2373, the company shared new data this week on the vaccine’s efficacy against the South African B.1.351 1.351 variant.

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Protein Vaccine Vaccination Trials 97

XTalks

JULY 19, 2022

The two-dose protein-based, adjuvanted vaccine has been authorized for use in adults 18 years of age and older for the prevention of COVID-19 caused by SARS-CoV-2. Nevertheless, 77 percent of those polled said that even if a protein-based COVID shot were authorized in the US, they still would not get it.

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Vaccine Vaccination Protein Clinical Trials 94

Medical Xpress

DECEMBER 13, 2024

Researchers at Nano Life Science Institute (WPI-NanoLSI), Kanazawa University, have discovered how a protein called lamin A helps repair the protective barrier around a cell's DNA.

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DNA Genetics Life Science Protein 52

pharmaphorum

OCTOBER 4, 2022

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a deal which is expected to result in a combined sum of $215 million in cash and investments – projected to be enough for funding into 2025. This is due to close concurrently with the merger.

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Gene Therapy Gene Life Science Genetics 115

XTalks

FEBRUARY 7, 2025

Utilizing its proprietary Compass platform, Maze analyzes genetic data to create targeted therapies that address the root causes of disease. With its lead programs aimed at APOL1-mediated kidney disease, the company plans to improve patient outcomes through genetically-informed treatments.

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Marketing Genetics Vaccination Vaccine 66

XTalks

APRIL 1, 2025

Hemophilia A and B are rare genetic bleeding disorders caused by a deficiency or dysfunction of clotting factors VIII (FVIII) and IX (FIX), respectively. Through RNA silencing, it targets the expression of antithrombin, a protein that inhibits blood clotting. The frequent intravenous infusions can be challenging for patients.

FDA Approval 52

FDA Approval RNA Clinical Trials Clinical Trials 52

XTalks

OCTOBER 21, 2020

The answer may lie in the genetic code of the virus, which scientists at Duke University have found contains several silent mutations that affect protein folding. The researchers focused on the spike proteins that protrude from the surface of the coronavirus, which are responsible for viral attachment and entry into host cells.

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RNA Protein Genome Genomics 98

XTalks

JANUARY 14, 2021

Hahn, MD said, “The FDA will continue to monitor SARS-CoV-2 genetic viral variants to ensure authorized tests continue to provide accurate results for patients.”. The FDA says that while they may be impacted by new genetic variants of SARS-CoV-2, “the impact does not appear to be significant.”. As scientists learn more about the new B.1.1.7

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Genetics Genome Genomics Protein 98

XTalks

OCTOBER 8, 2024

To activate these instructions, cells create a copy called messenger RNA (mRNA), which exits the nucleus and directs the cell’s machinery to produce a specific protein. The duo’s research explained how cells with identical genetic material can develop into specialized types, such as muscle and nerve cells.

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Regulation Genetics Scientist RNA 52

XTalks

JULY 14, 2022

For example, when T cells (a type of white blood cells that fight off infections and cancer) are activated in our immune systems, thousands of proteins in these cells change. The proteins are interconnected and changes in one protein level can impact that of another.

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Gene Regulation Protein Gene Editing 98

XTalks

MARCH 7, 2024

Breast Cancer Multiomics: Unified Insights in Tumor Heterogeneity This on-demand webinar introduces a cutting-edge single-cell multiomics method designed for garnering detailed genetic insights into breast cancer. The test accurately identified a range of genetic abnormalities, including trisomies and microdeletions.

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Genetics Hormones Genome Genomics 115

XTalks

JANUARY 26, 2023

Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations. As with many cancers, CLL and SLL are more difficult to treat in the relapse/refractory setting.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

JANUARY 6, 2021

One nanobody in particular, called NIH-CoVnb-112, was shown to be able to prevent COVID-19 infection from the novel coronavirus by targeting both the spike protein on SARS-CoV-2, as well as the angiotensin converting enzyme 2 (ACE2) receptor that it binds to on human cells. The study was led by neuroscientists Thomas J. “TJ”

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Antibody Protein Bacteria In-Vitro 105

XTalks

FEBRUARY 19, 2025

It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. In 2024, Deciphera was acquired by ONO Pharmaceutical for $2.4 TGCT is a rare, non-cancerous tumor that develops inside or near joints.

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FDA Approval Drugs Trials Clinical Trials 59

XTalks

DECEMBER 1, 2021

While researchers are still working out the epidemiological, clinical and molecular details of Omicron, so far, it is known that the variant is laden with up to 50 mutations, 30 or so of which are just in its outer spike (S) protein. The company said as a result, Omicron’s spike mutations do not impact the accuracy of its tests.

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Gene Genome Genomics Genetics 105

XTalks

JUNE 8, 2021

SMA is a rare genetic condition that leads to a loss of motor neurons that results in progressive muscle weakness and wasting, paralysis and, when left untreated in its most severe form, breathing difficulties leading to permanent ventilation or death for most patients by the age of two. With a price tag of over $2.5

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Gene Therapy Gene Protein Trials 110

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. Tailored oncology. About the author.

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Genome Genomics Clinical Trials Clinical Trials 105

XTalks

DECEMBER 20, 2021

Covovax is a recombinant protein-based vaccine and can be regarded as a more “traditional” vaccine as it isn’t genetic-based like mRNA or viral vector vaccines. Covovax is a protein subunit vaccine developed by Novavax and the Coalition for Epidemic Preparedness Innovations (CEPI).

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Vaccine Vaccination Protein Manufacturing 96

XTalks

JANUARY 4, 2024

Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

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Gene Therapy Gene Gene Editing Sales 111

XTalks

JANUARY 3, 2024

In individuals affected by ATTR, which includes both hereditary and wild-type (non-hereditary) variants, the TTR protein forms fibrils that accumulate in various tissues. These tissues comprise peripheral nerves, the heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow.

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FDA Approval Protein RNA Clinical Trials 105

XTalks

DECEMBER 13, 2023

3) Fulgent Genetics Compound annual growth rate: 260 percent Fulgent Genetics, headquartered in California, is a technology-driven company with a strong presence in clinical diagnostics and therapeutic development. Alpine Immune Sciences has recently announced an augmentation in its R&D investment. million and $38.8

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Genetics Vaccination Vaccine DNA 111

XTalks

MAY 26, 2023

DEB is a genetic disorder characterized by very fragile skin that rips and blisters easily even from minor friction (like rubbing or scratching) or injury, resulting in open wounds that are prone to skin infections and fibrosis. Vyjuvek is also the first drug approved to treat the disease and is Krystal’s first approved product.

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

JUNE 16, 2021

The company’s candidate recombinant nanoparticle protein-based COVID-19 vaccine, NVX-CoV2373, is being evaluated in the PREVENT-19 (the PRE-fusion protein subunit Vaccine Efficacy Novavax Trial COVID-19) Phase III trial, which has close to 30,000 participants across 119 states in the US and Mexico.

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Vaccine Vaccination Protein Trials 98

XTalks

JUNE 28, 2023

DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness. DMD is caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells. It primarily affects boys, affecting approximately one in 3,300 to 3,600 male births worldwide.

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Gene Therapy Gene Protein FDA Approval 98

XTalks

JUNE 5, 2023

Abrysvo is an unadjuvanted vaccine and is composed of two preF proteins selected to optimize protection against RSV A and B strains. “A The genetic material of RSV (respiratory syncytial virus) encodes 11 proteins. The F-protein facilitates the fusion of the virus with the membrane of the host cell. How Does Abrysvo Work?

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Vaccine Vaccination FDA Approval Protein 98

XTalks

OCTOBER 16, 2024

Hympavzi reduces the activity of TFPI, a naturally occurring anticoagulation protein. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year. This action increases the amount of thrombin, a critical enzyme in blood clotting, helping to prevent bleeding episodes.

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FDA Approval Trials Clinical Trials Clinical Trials 105

XTalks

AUGUST 17, 2020

protein-based biologics) and vaccine treatments. protein-based biologics) : are those which bind to specific components (i.e. A growing area in the immunotherapy space is the development of biological modulators, which are genetically engineered proteins that target specific components of the immune system.

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Protein Engineer Immune Response In-Vivo 98

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. Epigenetic Editing with CRISPR.

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DNA Gene Gene Silencing Genome 98

XTalks

MAY 1, 2023

This makes Qalsody the first approved treatment to target a genetic cause of ALS. Since SOD1 mutations were first identified as a cause of ALS 30 years ago, the familial ALS community has been searching for genetically targeted treatments. To date, over 200 mutations in the SOD1 gene have been associated with the development of ALS.

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FDA Approval Gene Genetics Protein 89

XTalks

JUNE 18, 2024

This innovative technology, capable of carrying large protein payloads, has demonstrated potential in restoring muscular function in a mouse model of Duchenne muscular dystrophy (DMD) through the delivery of a full-length dystrophin protein.

Protein 52

Protein RNA Genetics Gene Expression 52

XTalks

JANUARY 11, 2023

likely emerged through the genetic recombination of the Omicron BA.2.10.1 subvariants, where the two infected the same individual and swapped genetic material to create a new hybrid subvariant. This is concerning, as high-income countries are offering multiple boosters and throwing away unused vaccines.

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Scientist Vaccine Vaccination Genetics 98

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. Specifically, the vaccine contains human recombinant glutamate decarboxylase 65 (GAD65) protein conjugated to aluminum hydroxide (GAD-alum).

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Vaccine Vaccination Insulin Trials 103

XTalks

MARCH 13, 2024

Related: Transforming Eye Care: Innovating New Glaucoma and Dry Eye Disease Treatments — Featuring Pierre Simon, VP Marketing, Sight Sciences – Xtalks Life Science Podcast Ep. There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease.

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Gene Therapy Gene Life Science DNA 111