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

BioTech 365

MARCH 10, 2021

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: A new map of protein binding locations in yeast advances understanding of gene regulation.A massive effort to map the precise binding locations of over 400 different kinds … Continue reading →

Protein 40

Protein Gene Regulation Research 40

BioSpace

SEPTEMBER 11, 2020

Can you imagine organizing more than 10 terabytes of data into a useful network of information, like creating Google maps from scratch? I certainly can’t, but Eclipse BioInnovations, a San Diego-based biotech start-up, is aiming to do just that for RNA therapeutics.

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RNA Regulation Protein Gene 106

Scienmag

SEPTEMBER 22, 2020

Brazilian researchers observed that in uninfected adipocytes, the hormone irisin altered the expression of genes that regulate ACE-2, which encodes a protein to which the virus binds in order to invade human cells.

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Hormones Gene Protein Regulation 97

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. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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Gene Therapy Gene Clinical Trials Clinical Trials 65

pharmaphorum

JANUARY 12, 2022

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file for regulator approval. point increase on the NSAA scale with the gene therapy, while the control group saw a 0.7-point

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Gene Therapy Gene Regulation Trials 106

XTalks

JULY 26, 2021

But several researchers and experts have brought to light some food safety concerns of plant-based meat, and why it must be treated differently than that of animal protein. Because of their near-neutral pH and high protein and moisture content, plant-based meats are susceptible to microbial growth. FDA Regulation.

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Protein Contamination Allergies Bacteria 98

The Pharma Data

JUNE 14, 2023

Scientists discover small RNA that regulates bacterial infection People with weakened immune systems are at constant risk of infection. Abell Chair in Molecular and Cellular Biology – and Pengbo Cao, a postdoctoral researcher in Whiteley’s lab, discovered a gene that drives the switch. and Nelson D.

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RNA Regulation Scientist Bacteria 40

Medical Xpress

MARCH 24, 2023

A new research paper, titled "VCP/p97, a pleiotropic protein regulator of the DNA damage response and proteostasis, is a potential therapeutic target in KRAS-mutant pancreatic cancer," was published in Genes & Cancer on March 10, 2023.

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DNA Protein Regulation Gene 92

pharmaphorum

SEPTEMBER 6, 2021

BioMarin Pharma has had another setback in its gene therapy development programme, announcing this morning that the FDA has placed a phase 1/2 trial of its candidate for phenylketonuria (PKU) on hold while it investigates a safety signal. “We are committed to understand and mitigate any risk of cancer causation.”

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Gene Therapy Gene Genetic Disease DNA 94

Scienmag

AUGUST 28, 2020

Credit: OIST A protein that’s common throughout the body plays a key role in regulating glucose levels, says new research conducted in the Cell Signal Unit at the Okinawa Institute of Science and Technology Graduate University (OIST) and Riken Center of Integrative Medical Sciences.

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Protein Regulation Research Gene 52

BioTech 365

OCTOBER 26, 2020

Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: A ‘genomic rosetta stone’ for discovering the rules of gene regulation.As

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Genome Genomics Gene Regulation 40

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. bluebird bio’s Philip Gregory.

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Gene Therapy Gene Trials Gene Expression 59

Scienmag

MARCH 17, 2022

Cells use RNA as a versatile tool to regulate the activity of their genes. Small snippets of RNA can fine-tune how much protein is produced from various genes; some small RNAs can shut genes off altogether.

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RNA Gene Protein Regulation 76

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

Pharmaceutical Technology

JUNE 9, 2023

RP-A601 is an adeno-associated virus-based gene therapy currently being evaluated as a one-time, potentially curative treatment to improve survival and quality of life for PKP2-ACM patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene FDA Approval Trials 147

Scienmag

JANUARY 11, 2022

Enhancers are regions of DNA that do not code for proteins, but control how genes are expressed. Super-enhancers are clusters of enhancers that together regulate genes with important roles in cell identity. Scientists at St. Their assessment showed the super-enhancer has four […].

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Development DNA Gene Protein 80

pharmaphorum

DECEMBER 22, 2021

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient. AAV vectors are well-suited to gene therapies for DMD because they are effective at targeting muscle tissue.

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Gene Therapy Gene Trials Clinical Trials 59

Pharmaceutical Technology

MAY 4, 2023

Originated by the company’s in-house discovery engine, ATX-01 is designed to target microRNA 23b (miR-23b), a pathogenic gene associated with regulating the muscleblind-like protein (MBNL) expression involved in DM1. It has a dual mechanism of action that targets toxic dystrophia myotonica protein kinase and MBNL protein.

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Clinical Trials Clinical Trials Trials Protein 130

Outsourcing Pharma

JUNE 7, 2023

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that primarily affects males, caused by a mutation in the gene that encodes for a protein called dystrophin. This protein is crucial for the normal functioning of muscles, and its absence leads to progressive muscle weakness and wasting.

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Protein Genetics Gene Research 97

Drug Discovery World

MARCH 2, 2023

A new paper has solved the 20-year mystery of how epigenetic modifications act as traffic lights to control gene expression and could ultimately speed up the development of a new class of epigenetic cancer drugs. Epigenetics is still largely unexplored and referred to as the ‘dark matter’ of the genome.

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Gene Expression Gene DNA RNA 52

The Pharma Data

DECEMBER 3, 2020

New Tau Protein is Abnormal in Very Early Alzheimer’s Disease. Investigators with the University of Gothenburg identified new forms of tau protein that become abnormal in the very early stages of Alzheimer’s disease, even before the development of cognitive problems. “The Every week there are numerous scientific studies published.

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Protein Research Regulation Gene 52

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. What are the advantages compared to other gene modulating approaches?

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Gene Expression Gene Genetic Disease RNA 59

Drug Discovery World

NOVEMBER 27, 2023

The European Commission has extended use of the therapy to include children with CF aged two through five years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “As

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Protein Gene Medicine Gene Therapy 52

Pharmaceutical Technology

DECEMBER 8, 2022

Regulators in Japan have granted approval for the manufacturing plant of Kite in El Segundo, California, US, to produce Yescarta for the Japanese market. Directed against a cell membrane protein called CD19, Yescarta uses the immune system of the patient to destroy cancer. By Cytiva Thematic.

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Licensing Licensing Gene Therapy Gene 305

Scienmag

AUGUST 4, 2020

Led by Boyce Thompson Institute’s Andrew Nelson, four partners will identify RNA modifications and develop resources that may lead to hardier crops Credit: Photo credit: Anna Nelson Dittrich ITHACA, NY, August 4, 2020 — RNA perform a variety of functions in cells, helping with everything from regulating genes to building proteins.

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RNA Bioinformatics Protein Regulation 74

BioPharma Reporter

MARCH 22, 2022

Fujifilm Irvine Scientific, Inc, has announced the acquisition of Shenandoah Biotechnology, a company supplying recombinant proteins to the drug discovery, life science research, and cell and gene therapy markets.

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Gene Therapy Life Science Protein Gene 98

Drug Discovery World

SEPTEMBER 27, 2023

While genetics has made significant strides in healthcare, the field of epigenetics unveils a new dimension in precision medicine, offering insights into the dynamic interplay between genes and experiences that shape our health and disease.

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DNA Genetics Gene Medicine 116

pharmaphorum

JANUARY 6, 2021

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The post Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit appeared first on.

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Gene Silencing Gene RNA Contract Manufacturing 66

Drug Discovery World

AUGUST 2, 2023

New findings suggest PARP inhibitor drugs, used to treat cancer patients who have an altered BRCA1 or BRCA2 gene, may also be effective for patients which have changes in the SF3B1 gene. The researchers discovered that PARP inhibitor drugs reduced the ability of cancer cells with an altered SF3B1 gene to survive.

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Gene DNA Clinical Trials Clinical Trials 52

Drug Discovery World

JANUARY 11, 2024

Argininosuccinic aciduria is an inherited metabolic disorder that affects how the body breaks down protein – potentially leading to high levels of ammonia in the blood. Patients affected by the disease are found to also experience an imbalance of glutathione regulation, which is important for liver detoxification.

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Research Gene Therapy RNA Gene 59

Drug Discovery World

FEBRUARY 14, 2024

A class of molecules playing a crucial role in the regulation of gene expression and other cellular processes can restore normal skin structure rather than producing a scar, a University of Manchester-led study suggests. We expected the removal of microRNA-29 would help outer layers of the skin to grow faster.

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Gene Expression Regulation Protein Gene 52

Drug Discovery World

MARCH 22, 2023

The findings showed that targeting two regulators that control gene functions related to inflammation led to at least 10 times greater T cell expansion in models, resulting in increased antitumour immune activity and durability.

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Gene Editing Gene Engineer Regulation 52

Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. The mRNA constructs used in COVID-19 vaccines, for example, direct cells to produce a version of the “spike” protein studding the surface of SARS-CoV-2.

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Vaccination Vaccine Protein Antibody 52

pharmaphorum

JANUARY 13, 2022

AstraZeneca has expanded its efforts to develop new therapies targeting transcription factors – proteins that regulate processes in the cell that have become a hot topic for drug discovery in cancer and other diseases.

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Protein Gene Expression Regulation Gene 59

XTalks

DECEMBER 12, 2023

Less than a week after the approval, Novartis shared results from its APPEAR-C3G Phase III study of patients with C3 glomerulopathy (C3G) that showed iptacopan led to clinically meaningful reduction in proteinuria (protein in the urine) compared to placebo after six months of use.

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FDA Approval Bacteria Trials Protein 95

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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