Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

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Pharmaceutical Technology

DECEMBER 22, 2023

Discover Amicus Therapeutics' groundbreaking gene therapy vectors and methods of use. Enhance protein expression and cellular targeting for effective treatment of genetic disorders. Explore the patent claim now!

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Gene Therapy Protein Gene Genetics 100

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

Pharmaceutical Technology

OCTOBER 18, 2022

Last week, Nature magazine published a paper detailing an in-situ study of Parkinson’s disease (PD), showing protein structural changes in cerebrospinal fluid (CSF) between healthy individuals and Parkinson’s patients. Genetic biomarkers are being investigated to aid in the development of precision medicine for PD.

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Protein Genetics Gene Clinical Research 264

Pharmaceutical Technology

FEBRUARY 13, 2023

Several biotech companies and researchers are now exploring medical devices and gene therapies to address not just common forms of epilepsy, but also rare conditions such as Dravet Syndrome. Meanwhile, others are researching the link between gene variation and different responses to treatments.

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pharmaphorum

JANUARY 29, 2021

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. Zinc fingers. The exact mechanism depends on the disease in question.

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Gene Editing Gene Gene Therapy In-Vivo 145

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. OVs can be attenuated natural viruses or recombinant viruses.

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Protein Gene Genetics Gene Therapy 130

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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Gene Therapy Genetics Protein Gene 130

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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Pharmaceutical Technology

JUNE 4, 2023

Innovation S-curve for the pharmaceutical industry Transcription factors for AAV is a key innovation area in pharmaceutical Adeno-associated virus (AAV) vectors are widely used for gene therapy. There are two main genes in the AAV genome, rep and cap, which encode nine different proteins.

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Gene Gene Therapy Genetics DNA 262

Pharmaceutical Technology

JUNE 4, 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: Transcription factors for genetically modified cells. However, not all innovations are equal and nor do they follow a constant upward trend.

Genetics 130

Genetics Gene Expression DNA Antibody 130

Scienmag

APRIL 15, 2021

Team of Japanese and European scientists identify a novel genetic mitochondrial disorder by analyzing DNA samples from three distinct families Credit: Fujita Health University DNA ligase proteins, which facilitate the formation of bonds between separate strands of DNA, play critical roles in the replication and maintenance of DNA.

Genetics 105

Genetics DNA Genome Genomics 105

Scienmag

MAY 7, 2021

Credit: UPMC PITTSBURGH, May 7, 2021 – In a paper published today in Nature Communications, an international group of collaborators led by researchers at UPMC Children’s Hospital of Pittsburgh have identified a genetic cause of a rare neurological disorder marked by developmental delay and loss of coordination, or ataxia.

Genetics 95

Genetics Genetic Disease RNA Protein 95

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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Gene Regulation Protein Gene Editing 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.

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Gene Splicing Gene DNA Recombinant DNA Technology 130

Worldwide Clinical Trials

AUGUST 14, 2024

Historically, these indications have challenged the one-size-fits-all treatment approach due to patient variability, such as genetic differences in drug metabolism and underlying health conditions. This resistance may occur due to genetic mutations, epigenetic changes, or a tumor’s microenvironment.

Genetics 195

Genetics Protein Gene Genotype 195

Pharmaceutical Technology

APRIL 20, 2023

In addition to new regulatory functionalities, this feature may result in stronger and more durable protein expression. Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy RNA Gene Development 240

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

XTalks

JUNE 20, 2022

Hereditary ATTR has an autosomal dominant pattern of inheritance and is caused by point mutations in the transthyretin ( TTR ) gene that codes for the transthyretin transport protein found in the plasma and cerebrospinal fluid.

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FDA Approval Protein Genetics RNA 98

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.

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Gene Therapy Gene Gene Editing Sales 111

BioSpace

SEPTEMBER 15, 2020

The company is genetically programming RNA not just to deliver a gene of interest, but to control the location, timing and intensity of therapeutic protein expression using mRNA-encoded logic circuits.

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Gene RNA Genetics Protein 86

Medical Xpress

MARCH 18, 2023

University of Utah Health scientists have corrected abnormal heart rhythms in mice by restoring healthy levels of a protein that heart cells need to establish connections with one another.

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Gene Therapy Protein Genetics Gene 109

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. There are several different types of interferons in mammals, which include: IFN-? We said, ‘bingo!’”

Antibody 135

Antibody Genetics Research In-Vitro 135

XTalks

JUNE 8, 2021

Novartis’ Zolgensma (onasemnogene abeparvovec) gene therapy has been making significant strides as of late, including dosing of the first Spinal Muscular Atrophy (SMA) patient with the treatment in the UK last week. SMA is caused by mutations in the survival motor neuron 1 ( SMN1) gene, which along with the SMN2 gene, encode the SMN protein.

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Gene Therapy Gene Protein Trials 110

pharmaphorum

DECEMBER 2, 2021

Astellas has signed another bolt-on deal to build its gene therapy pipeline, agreeing a deal with Dyno Therapeutics to tap into its adeno-associated virus (AAV) vector platform for delivering genes to skeletal and cardiac muscle. Those safety issues have hit Astellas’ aspirations in gene therapy directly.

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Gene Therapy Gene Manufacturing Clinical Trials 97

Pharmaceutical Technology

JUNE 29, 2022

The next wave of medicine is well on course to be cell and gene-based. Specialty enzymes are proteins that can act as very specific biocatalysts to accelerate reactions and produce the desired target molecule in pharmaceuticals,” explains Kristoffer Laursen, Head of R&D at Novo Nordisk Pharmatech.

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Drugs Gene Therapy Production Manufacturing 295

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

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FDA Approval Genetics Gene Therapy Gene 105

Scienmag

OCTOBER 27, 2021

The rapid scientific advancements that followed the mapping of the human genome have revealed just how staggeringly complex the world of genetics is. We now know that proteins are not just the products of genes, but that they also interact with genes, influencing and regulating the rhythm of their expression.

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Gene Genome Genomics Genetics 72

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

Scienmag

SEPTEMBER 7, 2020

Credit: Dr Jie Zheng An innovative genetic study of blood protein levels, led by researchers in the MRC Integrative Epidemiology Unit (MRC-IEU) at the University of Bristol, has demonstrated how genetic data can be used to support drug target prioritisation by identifying the causal effects of proteins on diseases.

Genetics 56

Genetics Protein Drugs Development 56

Pharmaceutical Technology

APRIL 28, 2023

OCU410ST is an adeno-associated virus (AAV) serotype 5 capsid protein, which comprises a gene construct that encodes human retinoic acid receptor (RAR)-related orphan receptor alpha. Utilising an AAV delivery platform, OCU410ST enables the delivery of the RAR-related orphan receptor A (RORA) gene to the retina.

Drugs 130

Drugs Gene Protein Genetics 130

Pharmaceutical Technology

SEPTEMBER 22, 2022

Avrobio has received rare pediatric disease designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AVR-RD-04, designed to treat cystinosis. AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein.

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Gene Therapy Gene Protein Clinical Trials 147

Scienmag

APRIL 27, 2022

CHAPEL HILL, NC – Inside embryonic cells, specific proteins control the rate at which genetic information is transcribed from DNA to messenger RNA – a crucial regulatory step before proteins are created. Those specific “regulatory” proteins are called transcription factors, and they do their thing by binding to […].

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Scientist Gene RNA Protein 83

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

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. Subject to Microbial Growth.

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Protein Contamination Allergies Bacteria 98

STAT News

SEPTEMBER 16, 2022

Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells.

DNA 142

DNA Gene Editing In-Vivo Genetics 142

pharmaphorum

AUGUST 1, 2022

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to making it available in sometime around the middle of 2023. The post Sarepta says early filing for DMD gene therapy is back on appeared first on.

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Gene Therapy Gene Genetics Trials 105