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

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

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

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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Drug Discovery World

JANUARY 26, 2023

Scientists at The Institute of Cancer Research, London, combined biochemical profiling technologies with mathematical analyses to reveal how genetic changes lead to differences in the size of cancer cells – and how these changes could be exploited by new treatments. The major difference was cell size.

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DNA Protein Genetics Research 52

WCG Clinical

NOVEMBER 17, 2023

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52

Genome Genomics Gene Editing Genetic Engineering 52

Drug Discovery World

MAY 17, 2024

Cancer trials are notorious because they are dependent on the genetic profile of patients and hence highly-selective. The researchers delved into the genetic makeup of tumours, recognising that genetic mutations heavily influence how tumours respond to drugs. The next challenge is related to the enrollment rates.

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XTalks

JANUARY 3, 2024

Hereditary transthyretin amyloidosis (hATTR), formerly known as familial amyloid polyneuropathy, is a rare condition caused by mutations in the gene responsible for encoding transthyretin (TTR). This disorder is marked by the extensive deposition of amyloid in extracellular spaces, resulting in dysfunction across various organs and tissues.

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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

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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Drug Discovery World

MAY 29, 2024

Marc Hummersone, Senior Director of Research and Development (R&D) at Astrea Bioseparations, shares insight on the challenges and opportunities in cell and gene therapy (CGT) with DDW’s Megan Thomas. That takes a lot of effort, and some very clever people to get that amount of genetic information into a single plasmid DNA.

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XTalks

FEBRUARY 27, 2023

Alpha-mannosidosis is an extremely rare genetic metabolic disease affecting approximately one in 500,000 people. The lysosomal storage disorder is caused by mutations in the MAN2B1 gene, which codes for lysosomal alpha-mannosidase, an enzyme that degrades glycoproteins (proteins attached to sugar residues).

FDA Approval 52

FDA Approval Life Science Containment Genetics 52

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

FDA Approval 59

FDA Approval Gene Therapy Gene Clinical Trials 59

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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Gene Gene Therapy Gene Expression Regulation 40

The Pharma Data

NOVEMBER 22, 2020

They use mRNA that codes for the spike protein in the SARS-CoV-2 virus, which is embedded in a nanolipid particle—basically a minuscule fat molecule. When injected into the body, cells churn out the spike protein, which causes the body’s immune system to recognize the virus. It contains the genetic materials of the spike protein.

Vaccination 52

Vaccination Vaccine Protein Genetics 52

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

AUGUST 6, 2020

Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant. CBD is the most abundant non-psychoactive cannabinoid compound found in the cannabis plant, making up about 40 percent of the plant’s extract, which contains over 100 different cannabinoid compounds.

FDA Approval 64

FDA Approval Protein Containment Genetic Disease 64

Drug Discovery World

OCTOBER 27, 2023

Animal models, Ewart explains, are limited by their genetic and physiological differences from humans. With cues from a diverse population of tissue-specific cell types, extracellular matrix proteins, and biomechanical forces, cells grown in organ-chips closely emulate their in vivo counterparts and replicate tissue-level functions.

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

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.

Genome 80

Genome Genomics Genetics Genome Project 80

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. These clones can be manipulated and mutated in vitro to alter the expression and function of a protein.

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DNA Gene Protein In-Vitro 40

XTalks

MARCH 10, 2023

It is caused by a mutation in the FXN gene , which is responsible for the production of the protein frataxin. Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway.

FDA Approval 52

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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

JANUARY 10, 2021

FBX-101 is a first-in-human AAV gene therapy. Krabbe disease is a rare and fatal pediatric leukodystrophy caused by mutations in the galactosylceramidase (GALC) gene. INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1.

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Gene Therapy Trials Gene Clinical Trials 52

Drug Discovery World

MAY 11, 2023

Kerstin Pohl , Senior Global Marketing Manager, Gene Therapy & Nucleic Acid at SCIEX, looks at the application of liquid chromatography-mass spectrometry for analysing host cell proteins. Gene-based therapeutics and vaccines hold immense potential for more efficient and personalised treatment and prevention of complex diseases.

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Production Gene Protein Vaccination 52

pharmaphorum

JANUARY 3, 2023

As December 2022 closed out, Pfizer announced positive top-line results from its phase 3 BENEGENE-2 study evaluating fidanacogene elaparvovec (SPK-9001), its investigational gene therapy for treatment of adult males with moderately severe to severe haemophilia B.

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Gene Therapy Gene Medicine Trials 98

The Pharma Data

NOVEMBER 20, 2020

Progeria and Progeroid Laminopathies are separate and distinct ultra-rare, genetic, premature aging diseases that accelerate mortality in young patients. Progeria is caused by a point mutation in the LMNA gene, yielding the farnesylated aberrant protein, progerin. ABOUT ZOKINVY (LONAFARNIB). LIMITATIONS OF USE.

FDA Approval 52

FDA Approval Genetics Clinical Trials Clinical Trials 52

XTalks

FEBRUARY 11, 2021

This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics. Rosalind Franklin.

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Genetics DNA Genome Genomics 119

Roots Analysis

APRIL 15, 2024

Over the years, these large molecules have demonstrated promising therapeutic outcomes in the treatment of myriad of chronic and challenging disorders, including autoimmune disorders, infectious diseases, genetic disorders, and oncological disorders.

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Manufacturing Production Contract Manufacturing Contamination 40

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.

Biobanking 52

Biobanking Genetics Protein Medicine 52

The Pharma Data

MARCH 5, 2021

At the Kundl site, Novartis is a pioneer and has decades of experience in pharmaceutical production of proteins and in more recent years of nucleic acids. This media update contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Disclaimer. Source link:[link].

Manufacturing 52

Manufacturing Vaccination Vaccine Production 52

Drug Discovery World

DECEMBER 14, 2023

The Institute has particular strengths in understanding how cancer hijacks the cell’s energy factories and how it manipulates the body to provide the resources it needs to grow and spread, like protein scaffolding and blood vessels. Discoveries in these areas will identify new treatments which will become the cancer drugs of the future.”

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Drug Discovery World

JULY 29, 2022

Scientists modelling the cancer-suppressing p53 gene identify how the 20 different molecules unique to elephants get activated for increased sensitivity and response against carcinogenic conditions – with implications for cancer treatments in humans.

Gene 52

Gene DNA Protein Genetics 52

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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Gene Editing Gene In-Vivo Genome 52

Roots Analysis

MARCH 16, 2023

Messenger ribonucleic acid (mRNA) is a single-stranded molecule that is complementary to a gene’s DNA. It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein.

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Vaccination Vaccine DNA Protein 52

The Pharma Data

OCTOBER 18, 2020

Telomeres degrade and shorten with age and can become excessively damaged in certain genetic diseases, as well as from lifestyle factors such as smoking, poor diet, and chronic stress. Dyskeratosis congenita (DC) is a disease of pre-mature aging, which occurs due to a deficiency of telomere maintenance proteins.

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DNA Scientist Genetic Disease Research 52

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. The company achieved core revenues of $67 million, reflecting a remarkable 48 perecent year-over-year growth.

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Genetics Vaccination Vaccine DNA 111

The Pharma Data

NOVEMBER 4, 2020

Relying on independent research and development, the Company has formed gene recombination and fermentation engineering technology, and developed a number of core technologies, such as recombinant collagen, enzymatic and genetic engineering synthesis of bioactive peptides. For additional information, please visit [link].

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