XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.

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Gene Therapy Gene Trials Clinical Trials 95

XTalks

JUNE 28, 2023

Gene therapies for Duchenne muscular dystrophy (DMD) have been an area of intense research and Sarepta’s Elevidys is now the first one to be approved by the US Food and Drug Administration (FDA). DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

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Gene Therapy Gene Protein FDA Approval 98

Pharmaceutical Technology

JUNE 14, 2023

However, more immeasurable characteristics such as personality, behaviour, and even intelligence are all influenced by genetics to varying degrees. metres of supercoiled DNA contained within its nucleus. All that DNA is organised into hereditary units called genes, with humans having about 25,000 genes collectively known as the genome.

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Genome Project Medicine Genome Genomics 130

Medical Xpress

MAY 9, 2023

Leucine-rich repeats containing 4 (LRRC4)—a gene abundantly found in the brain and located on human chromosome 7q31–32—plays a pivotal role in memory formation, autism, spinal cord injury, as well as in determining the malignant potential, development, and progression of glioblastoma (GB), an aggressive cancer involving the brain and/or spinal cord. (..)

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Gene Containment Development Genetics 81

Scienmag

APRIL 8, 2021

This full assembly may contain clues to ape and human divergence; certain immune, brain and heart disorders; and other biomedical questions Credit: Kendra Hoekzema The full assembly of human chromosome 8 is reported this week in Nature.

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Gene DNA Containment Genetics 90

Advarra

MARCH 31, 2023

The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.

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Engineer Genetics Clinical Research Research 52

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. A gene contains instructions within our DNA. Which means it’s not any show, it’s the show,” he said.

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Regulation Genetics Scientist RNA 52

XTalks

JANUARY 21, 2025

The liquid portion of the blood (serum or plasma) containing antibodies is separated and tested using the Liason Biotrin parvovirus B19 IgG plus test on a Liason XL analyzer. There, plasma is separated, and tumor DNA is identified by detecting genetic mutations, methylation patterns and fragmentation signals.

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FDA Approval Manufacturing Development RNA 105

Scienmag

MAY 7, 2021

The two meters of -stretched- DNA contained in human cells are continuously twisting and untwisting to give access to genetic information: when a gene is expressed to generate a protein, the two strands of DNA are separated to give access to all the machinery necessary for this expression, resulting in an excessive accumulation of coiling […]. (..)

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DNA Regulation Genome Genomics 62

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. Defining the Boom in Gene Therapy Research The gene therapy field is experiencing explosive growth in today’s competitive research environment.

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Gene Therapy Gene Research Genetics 52

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. It’s a great tool for controlling gene expression.”.

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DNA Gene Gene Silencing Genome 98

pharmaphorum

SEPTEMBER 14, 2020

Last week geneticist Dr Charles Steward shared with us his experiences of searching for a genetic cause for his children’s rare neurological diseases. While there may be just under 20,000 confirmed protein coding genes, it turns out that much of the genome outside of these genes is also important in regulating how the genome is controlled.

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Genome Genomics Medicine Gene 119

XTalks

NOVEMBER 10, 2020

Expeditious and accurate diagnoses are necessary for patients to access healthcare services and treatment options for rare genetic diseases. Increasing the efficiency of case analysis and interpretation is essential to providing timely care for patients with genetic diseases.

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Genome Genomics Medicine Genetic Disease 98

The Pharma Data

NOVEMBER 19, 2020

MD, FAAP, Cincinnati Children’s Hospital, who will discuss genetic forms of hearing loss in children. Dr. John Greinwald is a tenured professor of Otolaryngology and Pediatrics with over 20 years of experience with a focus on the genetic causes and treatment of deafness. To register for the call, please click here.

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Genetics Containment Genetic Disease Gene 52

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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Biobanking Gene Gene Silencing Drugs 52

Roots Analysis

AUGUST 31, 2023

Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. Gene switches are sites on genes where regulatory molecules can bind to trigger transcription process, leading to expression of a particular gene.

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XTalks

DECEMBER 1, 2021

The S gene that encodes the characteristic protein harbors a deletion mutation (69-70del), which was also identified in the Alpha variant. Qiagen said it will continue to monitor the potential impact of Omicron on its COVID-19 PCR tests by conducting bi-weekly surveillance of genetic variations.

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Gene Genome Genomics Genetics 105

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. Previous research found fingerprints of positive selection within a gene that encodes the spike proteins. Viruses are constantly mutating and evolving,” Berrio said.

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RNA Protein Genome Genomics 98

pharmaphorum

AUGUST 5, 2021

Whereas targets like receptors contain an obvious ligand-binding site for drug molecules to interact with, PPIs comprise a more complex structure. The interface is highly hydrophobic, hence any compounds containing water would be repelled. Current challenges and conventional approaches in targeting PPIs.

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Protein Gene Bioassay DNA 126

Roots Analysis

OCTOBER 16, 2023

DNA and Gene Cloning involves the isolation of a DNA sequence of any species ( often a gene ) and its insertion into a vector to enable growth without any alteration in the original DNA sequence. There are some motifs, such as hairpin loops which hinder the usability of a gene.

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DNA Gene Protein In-Vitro 40

XTalks

APRIL 15, 2021

A 2015 study published in Nature Genetics found that the availability of human genetic data made investigational drugs twice as likely to pass pivotal trials and eventually be approved. Figure 1: The use of Mendelian randomization to validate genetic drug targets.

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Development Drugs Genome Genomics 98

The Pharma Data

OCTOBER 26, 2020

The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris).

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Gene Therapy Gene Manufacturing Production 52

XTalks

MAY 15, 2023

Fabry disease, also known as Anderson-Fabry disease, is a rare genetic disorder that falls under the category of lysosomal storage diseases. Since the mutations are located on the X chromosome, males with a single altered copy of the GLA gene in each cell are affected by the condition.

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FDA Approval Gene Genetics Clinical Trials 98

The Pharma Data

JULY 8, 2021

Today, access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). Biogen Safe Harbor.

Genetics 52

Genetics Biobanking Protein Containment 52

The Pharma Data

JULY 15, 2021

The access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). About the UK Biobank.

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Biobanking Genetics Protein Medicine 52

Camargo

DECEMBER 10, 2021

In October 2017, the European Commission (EC) and the European Medicines Agency (EMA) collaborated with the Member States’ authorities to launch a plan to reduce discrepancies across the EU in applying certain legislation to advanced therapy medicinal products (ATMPs) containing or consisting of genetically modified organisms (GMOs).

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Development Genetics Production Clinical Trials 176

pharmaphorum

SEPTEMBER 9, 2020

Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate. He is currently the patient advocacy and engagement lead at Congenica, a digital health company that is investigating the genetic basis of rare diseases. For some people, a job is more than just a job.

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Genome Genomics Genetics Genome Project 102

XTalks

NOVEMBER 6, 2020

The Burnaby, BC-based company developed the oral DNA-based vaccine using its proprietary bacTRL Gene Therapy Platform, which uses genetically modified bifidobacteria as carriers of genetic vaccine elements on a DNA plasmid. BacTRL Gene Therapy Platform. Related: Red Meat Allergy Test Gets FDA Clearance. “We

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Clinical Trials Clinical Trials Vaccine Vaccination 75

Scienmag

JULY 14, 2021

Researchers have found a way to enhance radiation therapy using novel iodine nanoparticles Credit: Mindy Takamiya/Kyoto University iCeMS Cancer cell death is triggered within three days when X-rays are shone onto tumor tissue containing iodine-carrying nanoparticles. The findings, by scientists at Kyoto […].

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DNA Containment Scientist Research 97

The Pharma Data

DECEMBER 28, 2020

About Fulgent Genetics. Fulgent Genetics’ proprietary technology platform has created a broad, flexible test menu and the ability to continually expand and improve its proprietary genetic reference library while maintaining accessible pricing, high accuracy and competitive turnaround times. TEMPLE CITY, Calif.,

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Genetics Sales Gene Sequencing Marketing 52

Scienmag

SEPTEMBER 14, 2021

The term ‘DNA’ immediately calls to mind the double-stranded helix that contains all our genetic information. But the individual units of its two strands are pairs of molecules bonded with each other in a selective, complementary fashion.

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DNA Gene Containment Genetics 40

XTalks

JANUARY 2, 2024

Filsuvez topical gel is a sterile botanical drug product designed for topical use, containing birch triterpenes within an oil base. For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. Vyjuvek is a gene therapy that employs a herpes simplex virus type 1 (HSV-1) vector.

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FDA Approval Gene Therapy Gene Trials 111

The Pharma Data

JANUARY 27, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare, monogenic central nervous system (CNS) disorders, today announced that the U.S. The gene therapy utilizes an AAV1 viral vector to deliver a modified DNA encoding the GRN gene to a patient’s cells. PHILADELPHIA, Jan.

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Gene Therapy Gene Clinical Trials Clinical Trials 52

Scienmag

MAY 18, 2021

(Boston)–Researchers from Boston University School of Medicine (BUSM) report the formation of human cells containing a green fluorescent protein or GFP (one of the most important proteins in biology and fluorescence imaging) genetically fused with two interferon stimulated genes (ISGs), namely Viperin and ISG15.

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XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene. This makes Qalsody the first approved treatment to target a genetic cause of ALS.

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FDA Approval Gene Genetics Protein 89

pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets. Dr Danuta Jeziorska.

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Genome Genomics Gene Gene Expression 59

The Pharma Data

JANUARY 3, 2021

Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that U.S. GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta -galactosidase (?

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