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

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 64

Drug Discovery World

SEPTEMBER 27, 2023

Embarking on a new era of medicine The advent of genomics has ushered in the era of personalised medicine, enabling us to analyse the genetic makeup of individuals with unprecedented accuracy. Genetic variations inherited from our parents shape our response to drugs, disease susceptibility, physiology and metabolism.

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DNA Genetics Gene Medicine 115

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

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

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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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 74

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.

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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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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

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

XTalks

SEPTEMBER 30, 2020

The research shows that the activity of the immune messenger type 1 interferon (IFN) protein is diminished, either by genetic mutations or an autoimmune attack by neutralizing antibodies against it, in a subset of COVID-19 patients. Related: Low T Cell Counts Observed in COVID-19 Patients. The analysis revealed that 23 (3.5

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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

JANUARY 11, 2024

The team, led by University College London (UCL), King’s College London and Moderna scientists, found that mRNA could be used to correct a rare genetic liver disease known as argininosuccinic aciduria in a mouse model. mRNA contains instructions that direct the cells to make proteins.

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Research Gene Therapy RNA Gene 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

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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Trials Clinical Trials Clinical Trials Protein 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

BioTech 365

FEBRUARY 25, 2021

It transfers genetic information, translates it into proteins or supports gene regulation. … Continue reading →

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RNA Genetics Protein Regulation 40

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

Drug Discovery World

NOVEMBER 27, 2023

In addition, it has stimulated increased collaboration among drug developers, technology providers, supply chain organisations, and regulators. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics. Biotechnol Bioeng 2012;118:2326. Budge JD, Knight TJ, Povey J, et al.

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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

Roots Analysis

JANUARY 14, 2024

Like all drugs, biologics are regulated by the FDA. Biologics activate certain proteins or cells in your immune system to create specific responses to targets, while many conventional systemic drugs activate the entire immune system in a more generalized manner. The most widely used bacterial host system is Escherichia coli (E.

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Contract Manufacturing Manufacturing Protein Bacteria 40

Drug Discovery World

AUGUST 16, 2023

Decoding ‘junk DNA’ The Human Genome Project and subsequent studies discovered that most of our DNA (approximately 98%) does not actually code for proteins, with humans having approximately 20,000 tox 25,000 protein-coding genes. This leads to potentially severe, debilitating and unbearable toxicities for patients.

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RNA Genome Genomics DNA 52

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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Gene Therapy Gene Manufacturing Production 52

Drug Discovery World

APRIL 26, 2023

A team of scientists have discovered that modification of a gene in the brain can reduce anxiety levels, offering an exciting novel drug target for anxiety disorders. They focused on miRNAs, which regulate multiple target proteins controlling the cellular processes in the amygdala.

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XTalks

MARCH 13, 2024

There are also studies examining gene therapy-based techniques to repair or replace genetic defects contributing to this disease. The Glaucoma Foundation has funded studies focusing on two specific areas within gene therapy. Secondly, other funded research is exploring differential gene expression regulated by DNA methylation.

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Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

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FDA Approval Gene Clinical Trials Clinical Trials 52

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

pharmaphorum

NOVEMBER 18, 2021

Danish drugmaker Novo Nordisk must like what it has seen in its two-year-old alliance with gene-silencing specialist Dicerna Pharma – it has just agreed to acquire the biotech for $3.3 Once in the blood, the GalXC molecules travel to the liver, where they enter hepatocyte cells and can switch protein production on or off.

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The Pharma Data

AUGUST 13, 2020

Mutations in the DMD gene can lead to an absence of the muscle cell protein dystrophin. The drug was trialled in two studies of a total of 32 male participants with genetically confirmed DMD. In one of these studies of 16 patients, Viltepso generated an average increase in dystrophin levels from 0.6%

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FDA Approval Genetics Protein Regulation 52

The Pharma Data

JANUARY 27, 2021

FDA Also Grants Two Orphan Drug Designations for the Company’s Gene Therapy Programs for Gaucher Disease and Mucolipidosis –. Food and Drug Administration (FDA) granted six rare pediatric disease designations (RPDDs) for various programs within its development pipeline for LSDs, including four recombinant enzyme and two gene therapy programs.

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Gene Therapy Gene Protein Life Science 40

The Pharma Data

NOVEMBER 22, 2020

As the Pfizer and BioNTech COVID-19 vaccine goes to regulators and the Moderna vaccine approaches the end of its Phase III trials, AstraZeneca and the University of Oxford announced high-level results from an interim analysis of their COVID-19 vaccine, AZD1222. It contains the genetic materials of the spike protein.

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Vaccination Vaccine Protein Genetics 52

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Dr. Bishop: Rett syndrome is a genetic disorder caused by a mutation in the MECP2 gene.

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FDA Approval Genetics Trials Drugs 97

Delveinsight

SEPTEMBER 23, 2021

Boehringer stated that the Abexxa’s generation aims at cancer-specific proteins situated throughout the mobile versus proteins at the mobile membrane broadening the selection of possible cancer antigen objectives. 858’s drug candidates include proteins, which regulate RNA and immune response.

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RNA Gene Therapy Gene Protein 61

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.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

JANUARY 26, 2023

Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations. As with many cancers, CLL and SLL are more difficult to treat in the relapse/refractory setting.

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FDA Approval Life Science Clinical Trials Clinical Trials 97