Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Gene Therapy Gene Gene Editing Manufacturing 243

Scienmag

MARCH 4, 2022

By combining CRISPR technology with a protein designed with artificial intelligence, 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.

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Protein Gene Medicine Research 83

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.

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Gene Therapy Gene Gene Editing Genetics 238

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.

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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.

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Protein Gene Genetics Gene Therapy 130

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. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Manufacturing Gene Clinical Trials 264

Bio Pharma Dive

DECEMBER 12, 2022

New data at ASH show the therapy can maintain levels of a key blood-clotting protein for years. Roche’s subsidiary Spark has offered few updates on its hemophilia A treatment since being acquired in 2019.

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Gene Therapy Gene Protein 324

Bio Pharma Dive

FEBRUARY 16, 2023

The company’s platform technology is built on research from Zhang’s lab at the Broad Institute, which focuses on a proteins that could be used to package and deliver nucleic acids.

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Packaging Packaging Gene Protein 316

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. Cold chain logistics, meanwhile, have never been so important.

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Gene Therapy Gene Medicine Genetics 245

BioSpace

JANUARY 18, 2021

Sizable venture rounds from cardiac gene-editing company Verve Therapeutics and the retooled fusion protein maker Vera Therapeutics will push their lead therapies into and through the clinic over the next two years.

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Gene Editing Protein Gene 93

Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. An approval decision on the gene therapy, also known as EtranaDez, is expected by the end of this month. million price tag.

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Gene Therapy Gene FDA Approval Medicine 278

Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

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Gene Therapy Gene Clinical Trials Clinical Trials 130

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. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

Protein 52

Protein Gene Genome Genomics 52

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.

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Gene Therapy Gene Protein Research 286

Pharmaceutical Technology

MARCH 28, 2024

Discover how 4D Molecular Therapeutics Inc's patented variant AAV capsid proteins enhance muscle cell infectivity for gene therapy, revolutionizing treatment for genetic disorders.

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Pharmaceutical Technology

JUNE 8, 2023

GenScript ProBio has collaborated with the New York Blood Center Enterprises’ business unit, called Comprehensive Cell Solutions (CCS), to accelerate the development and manufacture of cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD.

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Gene Therapy FDA Approval Gene Protein 97

Bio Pharma Dive

DECEMBER 9, 2021

The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.

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Gene Therapy Gene Protein Medicine 332

Bio Pharma Dive

APRIL 27, 2023

Partner Regeneron said the data, which showed substantial reduction of a key protein, suggests hope for the “bold dream” of silencing genes in the brain.

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Protein Gene 257

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.

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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.

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Pharmaceutical Technology

APRIL 13, 2023

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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Gene Therapy Gene FDA Approval Licensing 147

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).

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Gene Editing Gene Genome Genomics 52

BioPharma Reporter

OCTOBER 17, 2023

SpliceBio, a genetic medicines company harnessing protein splicing to develop the next generation of gene therapies, has partnered with Spark Therapeutics to develop a gene therapy for an inherited retinal disease.

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Gene Gene Therapy Development Genetics 59

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.

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Protein Gene Research 73

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).

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Protein Production Gene Development 88

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.

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Gene Expression Gene Protein Medicine 66

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).

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Protein Medicine Scientist Research 52

Pharmaceutical Technology

AUGUST 11, 2022

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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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.

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Gene Therapy Gene Protein FDA Approval 98

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

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Gene Therapy Gene Trials Protein 105

Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.

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Gene Therapy Gene Trials Clinical Trials 98

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

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 […].

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Protein Genetics RNA Development 98

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. . Two methods currently used to deliver protein-making packages into cells, ‘mini promoters’ and serotype-mediated gene expression, vary widely.

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Gene Expression Gene Packaging Packaging 52

Scienmag

JUNE 16, 2022

Scientists are starting to understand the precise workings of a type of gene that, unlike other genes, does not code for proteins – the building blocks of life.

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Medical Xpress

MARCH 31, 2023

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

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Protein Production Gene Research 75