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.

Immune Response 41

Immune Response Reagent Research Protein 41

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).

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 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.

RNA 98

RNA Protein Genome Genomics 98

Drug Discovery World

NOVEMBER 29, 2022

Using artificial intelligence, researchers at Chalmers University of Technology, Sweden have succeeded in designing synthetic DNA that controls the cells’ protein production. . Now we have succeeded in designing our own DNA that contains the exact instructions to control the quantity of a specific protein,” said Aleksej Zelezniak. .

DNA 52

DNA Development Research Drugs 52

Pharmaceutical Technology

FEBRUARY 15, 2023

Cas, also called CRISPR associated proteins, are a family of nucleases such as Cas9, Cas12a, Cas12b, and CasX that process these sequences. The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells.

In-Vivo 162

In-Vivo Genome Genomics Gene Editing 162

Drug Discovery World

MARCH 26, 2024

For example, progress made in developing gene mutant-specific inhibitors, antibody-drug conjugates (ADC), or cellular therapies. In addition to STC-15 and our METTL1, our pipeline contains a number of preclinical programs that are advancing rapidly for the treatment of viruses, neurodegeneration, cancer, and immuno-inflammatory conditions.

RNA 64

RNA In-Vivo In-Vitro DNA 64

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.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

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.

Gene 105

Gene Genome Genomics Genetics 105

Pharmaceutical Technology

JULY 29, 2022

And we are very prepared and have data that will answer those questions, what would be the advantage of shifting to an Omicron-containing vaccine, whether it's monovalent or bivalent.” Although the UK has fewer facilities, around 70% of those offer dose manufacturing with containment.

Manufacturing 130

Manufacturing Vaccination Vaccine Packaging 130

Drug Discovery World

JANUARY 26, 2023

“We also hope our discovery will lead to new treatment strategies – for example creating drugs to target the proteins that regulate cell size.” Genetic mutations in melanoma Melanoma is driven by two different genetic mutations – 60% of cases are caused by a BRAF gene mutation, while 20-30% are caused by an NRAS mutation.

DNA 52

DNA Protein Genetics Research 52

XTalks

APRIL 13, 2021

synuclein, a protein involved in the formation of amyloid deposits in the brain characteristic of Parkinson’s disease and other neurodegenerative disorders, is also implicated in melanoma. The scientists found that the amyloid-forming protein is expressed at higher levels in melanoma cells than in normal healthy skin. In addition to ?-synuclein,

Research 98

Research Protein In-Vitro Gene Therapy 98

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

Clinical Trials 75

Clinical Trials Clinical Trials Vaccination Vaccine 75

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

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.

FDA Approval 89

FDA Approval Gene Genetics Protein 89

Drug Discovery World

NOVEMBER 14, 2022

AAV vectors for diabetes gene therapy. A collaboration between PerkinElmer’s SIRION Biotech business and the Centre for Genomic Regulation (CRG) agreed to jointly develop new generation adeno-associated virus (AAV) vectors for type 1 and type 2 diabetes gene therapy in the pancreas. . A step closer to preventing type 1 diabetes.

Development 52

Development Gene Therapy Drugs Gene 52

The Pharma Data

FEBRUARY 12, 2022

They took a close look at the role of a protein called MFSD1 – the mammalian relative of a protein they had previously identified as affecting cell migration in fruit flies. Therefore, first author Marko Roblek from the Siekhaus group created mouse cancer cells lacking the protein. Protein prevents detachment.

Protein 40

Protein Doctors Doctor Gene 40

Pharmaceutical Technology

DECEMBER 9, 2022

The move is particularly timely given the significant growth expected in global sales of cell and gene therapies. Beyond our expertise in small molecules, we have built a solid reputation with biopharmaceutical companies for the characterisation and analysis of monoclonal antibodies, therapeutic proteins and biosimilars. Switzerland.

Development 130

Development Manufacturing Production Vaccination 130

Pfizer

AUGUST 3, 2022

PF-07265803 (formerly ARRY-371797) is an investigational compound being studied for the treatment of symptomatic DCM due to an LMNA gene mutation. The information contained in this release is as of August 3, 2022. Based on these results, the Phase 3 trial and further development of PF-07265803 will be discontinued. Disclosure Notice.

Development 101

Development Vaccination Vaccine Production 101

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

Pharmaceutical Technology

JUNE 21, 2023

CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.

Gene Therapy 246

Gene Therapy Gene Protein Sales 246

XTalks

AUGUST 26, 2020

Researchers studied the proteins that act as chemical signals and conducted experiments to see how those signals were affecting the macrophages. They found a protein called Rspondin3, which is released at high levels during inflammatory injury. This protein plays a crucial role in macrophage programming.

Bacteria 105

Bacteria Immune Response Protein Medicine 105

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.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

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

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. How do organ-chips support gene therapy delivery? What are the limitations of the technology?

Research 72

Research Gene Therapy In-Vivo In-Vitro 72

The Pharma Data

DECEMBER 10, 2020

Through its Genome5000 program, Lexicon scientists studied the role and function of nearly 5,000 genes and identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Safe Harbor Statement.

Containment 52

Containment Clinical Development Development Protein 52

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.

DNA 40

DNA Gene Protein In-Vitro 40

XTalks

FEBRUARY 27, 2023

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). In doing so, the enzyme can degrade the mannose-containing oligosaccharides and prevent their accumulation throughout the body.

FDA Approval 52

FDA Approval Life Science Containment Genetics 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.

Production 52

Production Gene Protein Vaccination 52

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

Drug Discovery World

JANUARY 9, 2024

However, government initiatives to contain healthcare costs, the sustained high cost of capital and global geopolitical disputes are negatively impacting investor appetites for the sector. For patients reluctant to receive novel therapies, such as mAbs or gene therapy, efanesoctocog alfa will likely be an appealing option.

Drugs 69

Drugs Antibody Gene Editing Medicine 69

Drug Discovery World

DECEMBER 29, 2023

Dr Sofia Ferreira, Director of Applications at Refeyn, discusses how various technologies can address the main challenges in membrane protein characterization, describing how novel technologies, such as mass photometry, can streamline and support analytical processes. Scientists use a multitude of membrane mimetics to overcome this issue.

Protein 52

Protein In-Vitro Research Drugs 52

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

FDA Approval Engineer Clinical Trials Clinical Trials 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.

Gene Therapy 98

Gene Therapy Gene Medicine Trials 98

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.

Gene Therapy 52

Gene Therapy Trials Gene Clinical Trials 52

The Pharma Data

JANUARY 3, 2021

INZ-701 is a soluble, recombinant protein containing the extracellular domain of native human ENPP1 fused to the Fc domain of the immunoglobulin IgG1 that is designed to correct a defect in the mineralization pathway caused by ENPP1 and ABCC6 deficiencies. Additional details can be found: [link]. About INZ-701.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Containment 52

Drug Discovery World

JULY 26, 2022

RA: Could this type of treatment be used alongside other oncology therapies such a cell & gene therapies? BM: Oncolytic viruses can be used in combination with other cancer treatments, including cell & gene therapies. The virus is allowed to replicate within the cells, which are then frozen.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

Roots Analysis

APRIL 15, 2024

These complex entities, such as antibodies, antibody drug conjugates, cell therapies, gene therapies, and therapeutic proteins, are highly specific molecules that are designed to precisely target biomarkers associated with a particular disease pathway.

Manufacturing 40

Manufacturing Production Contract Manufacturing Contamination 40