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The US Food and Drug Administration (FDA) has granted Fast Track designation for Avidity Biosciences’ AOC 1020 to treat facioscapulohumeral muscular dystrophy (FSHD). AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4.
Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients. It could also be made more powerful by incorporating personal genetic information and geneexpression profiles in tissues such as the lungs, they suggest.
Brazilian research group shows that valproic acid (VPA), used to treat epilepsy since the 1960s, modulates geneexpression in tumor gene models and acts on DNA conformation and the histones in chromatin Results of recent studies involving valproic acid, used for decades as an anti-convulsant drug, show that it can interact with the conformation of (..)
The detailed complexity of these processes can be captured by creating models that combine correlates of gene and proteinexpression, providing insight into the molecular composition of tissues. Visium Spatial GeneExpression Solution. Feature barcoding using gel beads. Single Cell Data Analysis.
The US Food and Drug Administration (FDA) has granted clearance for Neurogene’s investigational new drug (IND) application for NGN-401 to treat Rett syndrome. Developed with the University of Edinburgh, the EXACT self-contained technology enables MeCP2 protein therapeutic levels while avoiding overexpression-related toxicities.
Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting geneexpression. GlobalData forecasts total sales from this drug class to exceed $1bn by 2029.
A form of blood cancer known as mantle cell lymphoma is critically dependent on a protein that coordinates geneexpression, such that blocking its activity with an experimental drug dramatically slows the growth of this lymphoma in preclinical tests, according to a study from Weill Cornell Medicine researchers.
Alnylam has reported that it aims to develop a therapy that targets the INHBE gene and reduces the overall risk of T2D and coronary heart disease for at-risk patients. Targeting geneexpression that results in increased abdominal fat and leads to obesity is a key factor for the reduction of T2D and wider metabolic syndrome.
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. It’s not the big pharma’s first foray into the transcription factor area.
UNC researchers now show that these two factors can directly associate with one another, modulating cancer-cell-specific programs of geneexpression. ” They found that EZH2 possesses two different binding patterns on chromatin in acute leukemia cells, eliciting two distinct gene-regulatory programs (Figure 1). .”
However, DelveInsight’s Angelman syndrome market analysis report estimates that several approaches focused primarily on the Gene Therapies, Topoisomerase Inhibitors, Cannabidiol, Protein Phosphatase 2A inhibitor, among others are under development to find a cure for the Angelman syndrome.
Use of the drug verapamil to treat Type 1 diabetes continues to show benefits lasting at least two years, researchers report in the journal Nature Communications. The suggestion that verapamil might serve as a potential Type 1 diabetes drug was the serendipitous discovery of study leader Anath Shalev, M.D.,
Cambridge, Massachusetts-based FogPharma has closed another impressive fundraising round, adding $178 million to the pot as it ushers its polypeptide-based drug candidates towards the clinic. Ut comes just as FogPharma is preparing to start clinical trials of alpha-helical polypeptide candidate FOG-001.
Therapeutic Developments Related to Glaucoma The introduction of new classes of glaucoma medications, such as prostaglandin analogs, nitric oxide-donating drugs and Rho kinase inhibitors, has added to the pharmacological options available for managing IOP.
Known as Zynteglo in Europe where it is approved to treat the rare blood disorder beta-thalassemia, the gene therapy has been given the all-clear by bluebird’s scientists after reviewing the case of AML that emerged in a trial of the drug in sickle cell disease. Shares in bluebird ticked up following the announcement.
Hybridization Tests – These tests utilize complementary DNA or RNA probes to detect and quantify specific genetic targets, enabling the identification of pathogens, genetic variations, and geneexpression patterns. Such test can be used for geneexpression profiling, genotyping and detecting chromosomal abnormalities.
ANGLE’s proven patent protected platforms include a circulating tumor cell (CTC) harvesting technology and a downstream analysis system for cost effective, highly multiplexed analysis of nucleic acids and proteins. The system is epitope independent and can capture all types of CTCs as well as CTC clusters in a viable form (alive).
When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.
Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. billion in funding.
Coding RNAs include messenger RNA (mRNA) and short interfering RNA (siRNA), which encode proteins and silence geneexpression, respectively. ASOs are short single-stranded nucleotides that bind to specific messenger RNAs and prevent the production of a particular protein.
To activate these instructions, cells create a copy called messenger RNA (mRNA), which exits the nucleus and directs the cell’s machinery to produce a specific protein. Despite containing the same set of genes, cells are able to differentiate due to the regulatory role of microRNAs, which control geneexpression in a precise manner.
Biotechnology, Pharma and Biopharma News – Research – Science – Lifescience ://Biotech-Biopharma-Pharma: Protein that manipulates geneexpression inside the cell nucleus could inspire new RSV drugs, vaccines.Protein that manipulates geneexpression inside the cell nucleus could inspire new RSV … Continue reading →
Further, the expression of any gene is dependent on the rate at which it is transcribed into mRNA and translated into proteins. There are various regulatory proteins or transcription factors that are responsible for affecting the transcription rate.
With the increased interest and gradual shift of investment from small molecule drugs to biologics and the establishment of several biologics manufacturing companies / biologics CMOs, more than 250 biologic therapies and vaccines have been developed, globally. Like all drugs, biologics are regulated by the FDA.
Food & Drug Administration (FDA) for Istodax ® (romidepsin), a histone deacetylase (HDAC) inhibitor, as monotherapy for the treatment of peripheral T-cell lymphoma (PTCL) in adult patients who have received at least one prior therapy. HDACs also deacetylate non-histone proteins, such as transcription factors. DRUG INTERACTIONS.
The drug, UCB0107, is designed to impede or decrease the buildup of tau proteins in the brain, which leads to nerve cell damage and death. Omega’s platform is designed to adjust geneexpression to healthy levels rather than switching genes on and off. GlaxoSmithKline, the U.S.
The drug, Yeliva (opaganib) is a first-in-class, oral spingosine kinase-2 (SK2) selective inhibitor with triple-action effect on the pathophysiological processes linked to COVID-19. The drug is a humanized IgG4 monoclonal antibody that blocks CCR5 and is being developed for COVID-19, HIV and metastatic triple-negative breast cancer.
Nat Rev Drug Discov. The obesity epidemic and food addiction: clinical similarities to drug dependence. J Psychoactive Drugs. Curr Drug Abuse Rev. Addicted to Palatable Foods: Comparing the Neurobiology of Bulimia Nervosa to that of Drug Addiction. Am J Clin Nutr. 1992;55(1):151S–154S. PMID: 1728825. Pediatr Ann.
From rare disease drug approvals to treatments involving immunotherapies and gene therapies and awarding of a Nobel Prize to the inventors of the gene-editing tool CRISPR, 2020 was a year of great activity and productivity despite the backdrop of the pandemic. Drugs, Testing and Vaccines.
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