Scienmag

SEPTEMBER 28, 2020

Brazilian research group shows that valproic acid (VPA), used to treat epilepsy since the 1960s, modulates gene expression 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 (..)

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Pharmaceutical Technology

JUNE 13, 2023

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 gene expression. GlobalData forecasts total sales from this drug class to exceed $1bn by 2029.

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The Pharma Data

JULY 12, 2021

online issue of Nature Communications, researchers at University of California San Diego School of Medicine describe a new approach that uses machine learning to hunt for disease targets and then predicts whether a drug is likely to receive FDA approval. the success rates in drug discovery?are

Drugs 52

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pharmaphorum

OCTOBER 19, 2022

The dark genome – a loose term that covers non-coding regions of the genome that are capable of regulating the expression of genes, previously rather inaccurately referred to as junk DNA – is increasingly being explored by biopharma companies for new drug targets. Dr Danuta Jeziorska.

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Roots Analysis

AUGUST 12, 2024

These diagnostic solutions are essential for detecting and monitoring diseases, identifying genetic abnormalities, and guiding personalized treatment plans. Molecular diagnostic solutions are pivotal across various medical fields, including oncological disorders, infectious diseases, genetic testing, and personalized medicine.

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XTalks

MAY 31, 2022

The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. It has been estimated that around eight million people in the US and 125 million people in the world have been affected by psoriasis. Clinical Trials of Vtama.

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XTalks

MARCH 13, 2024

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.

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pharmaphorum

MARCH 10, 2021

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.

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Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. For instance, GTX-102 , an antisense therapy silencing the gene expression by binding to the RNA molecule behind the regulation of its expression, has also secured USFDA Fast Track Designation.

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The Pharma Data

NOVEMBER 8, 2020

The program, developed in collaboration with Regeneron Pharmaceuticals, uses a proprietary, cell-selective promoter to precisely control gene expression in cochlear hair cells. Chief Executive Officer of Decibel. “We We are grateful to this group of outstanding investors for choosing to partner with us to advance this vision.”.

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The Pharma Data

JANUARY 19, 2021

Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced that it has priced an underwritten public offering of 4,000,000 shares of its common stock at a public offering price of $11.00 CAMBRIDGE, Mass.,

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Camargo

JULY 27, 2021

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.

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The Pharma Data

NOVEMBER 9, 2020

Food and Drug Administration Breakthrough Therapy Designation granted to tezepelumab in Sept. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. Food and Drug Administration for the products. EMJ Allergy Immunol. 2018; 3: 60-9.

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XTalks

OCTOBER 8, 2024

Today, it’s known that the human genome encodes over a thousand microRNAs, which play vital roles in how organisms grow, differentiate and operate, uncovering a new layer of complexity in genetic regulation. Their ability to modulate gene expression at the RNA level provides a novel therapeutic approach for conditions.

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Regulation Genetics Scientist RNA 52

pharmaphorum

JANUARY 29, 2021

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.

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Roots Analysis

OCTOBER 22, 2023

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

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Roots Analysis

AUGUST 31, 2023

It is worth noting that the transcription factor is responsible for recruiting RNA polymerase to bind to the gene and produce messenger RNA, which is then translated into the protein. An additional level of genetic control is provided by gene switches that are located upstream of the promoter region.

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Roots Analysis

JANUARY 14, 2024

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.

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The Pharma Data

JULY 8, 2021

Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted Priority Review for tezepelumab in the treatment of asthma. 1) The Prescription Drug User Fee Act goal date for a decision by the FDA is during the first quarter of 2022. US Food and Drug Administration. Priority Review.

Allergies 52

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The Pharma Data

MAY 15, 2021

All too often, it is lifestyle factors – the things we do day to day – that determines whether a particular gene switches on to create actual illness. The scientific study of how to undo genetic illnesses – like HCT – is called epigenetics. It’s because certain genes act one way on some people… and another way on other people.

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The Pharma Data

DECEMBER 27, 2020

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.

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The Pharma Data

MAY 13, 2021

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.

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XTalks

DECEMBER 24, 2020

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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