Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

Pharmaceutical Technology

MARCH 10, 2023

On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. In the past, researchers have often had to seek help from outside the UK, thus delaying clinical trials and patient access.

Gene Therapy 264

Gene Therapy Manufacturing Gene Clinical Trials 264

pharmaphorum

AUGUST 5, 2021

Protein-protein interactions (PPIs) are becoming increasingly relevant in the pathology of many diseases, including cancer. PPIs are an integral part of the physiology of living organisms, as complexes which control biological pathways mediated by proteins. These regions are critical for optimal interactions between proteins.

Protein 125

Protein Gene Bioassay DNA 125

Pharmaceutical Technology

JANUARY 25, 2023

A researchers’ team from the University of Texas at San Antonio’s (UTSA) Department of Chemistry and its partners are developing compounds to treat Glioblastoma multiforme (GBM) tumours. The National Cancer Institute (NCI) has recently awarded a $3m grant to the researchers’ team.

Development 246

Development Research Hormones Gene 246

Pharmaceutical Technology

MAY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Oncolytic viral proteins. Sillajen and Advaxis are the other key patent filers of oncolytic viral proteins.

Protein 130

Protein Gene Genetics Gene Therapy 130

Bio Pharma Dive

JUNE 23, 2022

After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however.

Gene Therapy 278

Gene Therapy Gene Protein Research 278

Pharmaceutical Technology

FEBRUARY 13, 2023

While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genome 264

Pharmaceutical Technology

JULY 7, 2022

For many decades, investigators have been working on innovative therapeutic modalities known as cell and gene therapies, which use modified versions of the body’s own cellular and genetic material to treat and potentially cure these diseases. A new frontier in cancer research.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

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. So yes, you’ve got protein, but it’s not going to transfect or infect.” That’s not the case in CGT.”

Gene Therapy 109

Gene Therapy Gene Genetics DNA 109

The Pharma Data

MARCH 7, 2022

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. Researchers from the University of Washington School of Medicine in Seattle describe this finding in the journal Cell Reports.

Protein 52

Protein Gene Genome Genomics 52

Medical Xpress

APRIL 6, 2023

Researchers have known for years that cancer begins with mutations in certain types of genes. One of these types of cancer genes are so-called "tumor suppressors." Understanding how cancer develops is critical for designing effective, personalized cancer therapies.

Protein 81

Protein Research Gene Development 81

XTalks

JANUARY 4, 2024

Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. Treatment involves replacing the missing FIX clotting protein, traditionally through regular infusions.

Gene Therapy 111

Gene Therapy Gene Gene Editing Sales 111

Scienmag

FEBRUARY 10, 2022

Also known as transcriptional activators for their ability to induce transcription of genes into RNA messages, these proteins are essential for the cells to function properly. Yet little is known about these proteins, and it wasn’t clear how many activators there might be in human cells – until now.

Gene 59

Gene Research RNA Protein 59

Medical Xpress

DECEMBER 8, 2022

Researchers at the Johns Hopkins Kimmel Cancer Center have revealed how the gene HOXA5 may work to suppress formation of breast cancers. A new study indicates that it binds to another protein in an inflammatory cell pathway, boosting that protein's cancer-suppressing properties.

Gene 92

Gene Protein Research 92

BioPharma Reporter

MAY 22, 2023

US biopharma Insmed has announced progress across its early-stage research programs at the company's investor and analyst event, The Future of Rare at Insmed: Functional Genes, AI-Enhanced Proteins, Glowing Algae, and More.

Research 96

Research Protein Gene Development 96

Pharmaceutical Technology

DECEMBER 5, 2022

Sanofi has signed an agreement with the Queensland Government in Australia to establish a $190m (A$280m) research facility in Brisbane. The hub will connect Queensland scientists with the teams at Sanofi’s mRNA Centre of Excellence in the US and France, putting them at the front of international vaccine development and biomedical research.

Research 246

Research Gene Therapy Vaccination Vaccine 246

XTalks

JULY 14, 2022

Every gene in a cell is expressed at exact levels due to complex gene regulatory networks. 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. Using CRISPR Technology to Disrupt Networks of Immune Genes.

Gene 98

Gene Regulation Protein Gene Editing 98

Scienmag

JULY 7, 2022

A collaboration including researchers from Kobe University, University of Tokyo and Tohoku University has successfully identified and disrupted genes in the yeast Pichia pastoris (1) in order to increase its secretory production of useful proteins (2).

Protein 88

Protein Production Gene Development 88

Pharmaceutical Technology

FEBRUARY 20, 2023

While there is no cure to this neurodegenerative condition, academics and companies are pushing through with research that could help patients and their families. Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. The trial features four cohorts.

Development 246

Development Gene Therapy Protein Gene 246

Scienmag

NOVEMBER 25, 2021

A team led by researchers at Baylor College of Medicine and the Czech Academy of Sciences has uncovered a new piece of the puzzle of how gene expression is orchestrated. Published in the journal Science, the findings reveal a novel mechanism that coordinates the assembly of components inside cells that control gene expression.

Gene Expression 66

Gene Expression Gene Protein Medicine 66

Scienmag

MAY 14, 2021

Approach opens a new avenue to study the biology of acute leukemia and for the development of new drugs Credit: Grembecka / Cierpicki Labs The protein made by the ASH1L gene plays a key role in the development of acute leukemia, along with other diseases.

Protein 70

Protein Development Research Gene 70

Drug Discovery World

FEBRUARY 13, 2024

It goes on to show that NX-2127, a potent targeted protein degrader with differentiated activity against BTK and IKZF1/3, can overcome this resistance across a broad range of acquired mutations. The post Protein degrader overcomes resistance in B-cell malignancies appeared first on Drug Discovery World (DDW).

Protein 52

Protein Medicine Scientist Research 52

Pharmaceutical Technology

FEBRUARY 21, 2023

The adeno-associated virus five (AAV5)-based gene therapy, Hemgenix, is intended to treat adult patients with moderately severe and severe haemophilia B without a Factor IX inhibitors history. Hemgenix is claimed to be the first gene therapy to receive approval for treating haemophilia B in the EEA and the EU.

Gene Therapy 130

Gene Therapy Gene Medicine Production 130

Medical Xpress

MARCH 31, 2023

A team of medical researchers affiliated with a host of institutions in the U.S. has used base editing to restore the natural production of the SMN protein in mice, effectively curing spinal muscular atrophy (SMA) in the rodents.

Protein 75

Protein Production Gene Research 75

Drug Discovery World

FEBRUARY 22, 2024

Following the first regulatory approval of a CRISPR-based drug in late 2023, over ten years since the CRISPR-Cas9 system was elucidated, there is considerable optimism about the future potential for gene editing technologies. appeared first on Drug Discovery World (DDW).

Gene Editing 52

Gene Editing Gene Genome Genomics 52

Scienmag

NOVEMBER 11, 2020

Credit: Masato Kanemaki Researchers can now more accurately and precisely target specific proteins in yeast, mammalian cells and mice to study how knocking down specific protein traits can influence physical manifestation in a cell or organism. “Conditional gene knockout and small interfering RNA […].

Protein 98

Protein Genetics RNA Development 98

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Gene splicing using nucleases. They are engineered to cut specific genomic targets in order to modify the expression of single genes and proteins.

Gene Splicing 130

Gene Splicing Gene Recombinant DNA Technology DNA 130

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 Containment 52

Drug Discovery World

NOVEMBER 1, 2022

Researchers say they have successfully used a cell’s natural process for making proteins to ‘slide’ genetic instructions into a cell and produce critical proteins missing from those cells. . We wanted to develop a gene expression delivery tool that’s broadly useful in both preclinical and clinical models,” said Blackshaw. .

Gene Expression 52

Gene Expression Gene Packaging Packaging 52

Medical Xpress

MARCH 29, 2023

Proteomics is the study and analysis of proteins, including their structures, functions, interactions, and modifications within a biological system. Humans have approximately 20,000 genes that produce over a million different protein variants, including an estimated 100,000 protein isoforms.

Protein 73

Protein Gene Research 73

Drug Discovery World

JULY 24, 2023

The Defense Advanced Research Projects Agency (DARPA) has announced that Ginkgo Bioworks, which is building a platform for cell programming and biosecurity, has been awarded a four-year contract worth up to $18 million to reimagine how to manufacture complex therapeutic proteins.

Protein 52

Protein Manufacturing Gene Sequencing Production 52

Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

Gene Therapy 64

Gene Therapy Gene Clinical Trials Clinical Trials 64

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 caused by the absence of dystrophin, a protein that helps maintain the integrity of muscle cells.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

Medical Xpress

DECEMBER 7, 2022

New research published in the Annals of Clinical and Translational Neurology has identified three genes and their expressed proteins that may be involved in the pathogenesis of multiple sclerosis.

Gene 72

Gene Scientist Protein Research 72

Medical Xpress

FEBRUARY 22, 2023

Mount Sinai researchers have published a study in Alzheimer's & Dementia: The Journal of the Alzheimer's Association that sheds new light on the role of DNA methylation in Alzheimer's disease (AD).

DNA 98

DNA Research Protein Gene 98

Drug Discovery World

FEBRUARY 23, 2024

Data on VY-TAU01, Voyager’s lead anti-tau antibody candidate, and on Voyager’s tau silencing gene therapy programme will be presented at the upcoming 2024 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD 2024), taking place March 5-9, 2024, in Lisbon, Portugal.

Gene Therapy 52

Gene Therapy Gene Antibody Protein 52

BioPharma Reporter

OCTOBER 22, 2020

Scientists have used gene therapy and a newly developed light-sensing protein to restore sight in mice: with the technology to start clinical trials in the US later this year.

Gene Therapy 98

Gene Therapy Gene Clinical Trials Clinical Trials 98