Pharmaceutical Technology

MAY 25, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) aims to launch a pilot genetic biobank that will gather patient data to associate drug-related adverse events to their genetic makeup. According to the press release, the MHRA will be the first drug safety regulator in the world to pilot such a biobank.

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Biobanking Genetics Genome Project Genome 130

Scienmag

JULY 31, 2021

Research from Wellesley College shows that despite being a clonal insect species, weevils use gene regulation to adapt to new food sources and pass down epigenetic changes to future generations Without the benefits of evolutionary genetic variation that accompany meiotic reproduction, how does an asexual invasive species adapt over time to a new environment (..)

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Genetics Gene Regulation Research 111

Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Credit: St. Jude Children’s Research Hospital St.

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Gene Expression Regulation Gene DNA 97

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.

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Genetics Gene Expression DNA Antibody 130

Pharmaceutical Technology

OCTOBER 25, 2022

There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).

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Gene Therapy Gene Development Genetics 264

Medical Xpress

FEBRUARY 13, 2023

The findings of the genome exploration study, published in The Journal of Clinical Investigation, provide insight into how heartbeats are regulated and could impact diagnosis and risk prediction for a variety of common arrhythmias.

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Regulation Genetics Research Genome 75

pharmaphorum

OCTOBER 2, 2020

European regulators have started a first ‘rolling review’ of a COVID-19 vaccine, which is being developed by AstraZeneca in collaboration with the University of Oxford. The post European regulators begin rolling review of AZ’s COVID-19 vaccine appeared first on.

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FDA Law Blog

NOVEMBER 10, 2024

We note that the only Phase 3 study funded in the FY2024 announcement is the first-ever late-stage clinical trial for the indication of microcystic lymphatic malformations , a serious, rare genetic skin disease with no FDA-approved therapies. Relative to other areas of medicine (e.g.,

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Production Clinical Development Development Regulation 69

STAT News

NOVEMBER 15, 2022

Antitrust regulators in Spain have fined a drugmaker $10.6 At issue is a medicine called CDCA that is used to treat people with a rare genetic metabolic disease known as cerebrotendinous xanthomatosis, or CTX. There was a low-cost version available for decades. Continue to STAT+ to read the full story…

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Regulation Drugs Genetics Medicine 98

Medical Xpress

FEBRUARY 23, 2023

We do know that an overactive innate immune system is causing severe COVID-19 disease, but it is unclear how this is regulated. But why is this the case? Unfortunately, we do not know exactly.

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Regulation Genetics Research 74

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. Casanova’s lab is now searching for the genetic driver behind the autoantibodies.

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Worldwide Clinical Trials

NOVEMBER 12, 2024

CGTs address rare and complex diseases at the root cause with increasing use in cancer, genetic disorders, and autoimmune diseases. The process, from patient coordination through manufacturing and administration, is intricate, time-sensitive, and highly regulated. Below, we discuss some of these challenges in cell therapy trials.

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Gene Therapy Clinical Supply Trials Gene 182

Pharmaceutical Technology

MAY 4, 2023

The approval allows KALYDECO to be used in infants who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to the therapy, on the basis of clinical and/or in vitro assay results.

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In-Vitro Genetic Disease Medicine Genetics 246

XTalks

OCTOBER 8, 2024

The 2024 Nobel Prize in Physiology or Medicine has been awarded to American scientists Victor Ambros and Gary Ruvkun for their groundbreaking discovery of microRNA (or miRNA) and its role in post-transcriptional gene regulation. This control plays a critical role in development and cellular function.

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Regulation Genetics Scientist RNA 52

Pharmaceutical Technology

MARCH 2, 2023

We are grateful to Friedreich’s ataxia patients, investigators, US regulators, and our scientists and employees who made this approval possible. “As

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FDA Approval Trials Genetics Drugs 264

Scienmag

NOVEMBER 13, 2020

The results provide an interesting example of co-evolution of a circRNA, and its host-encoded protein product, that regulate each other’s […].

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RNA Regulation Protein Production 93

Medical Xpress

MAY 4, 2023

The team, led by CERA's principal investigator of cellular reprogramming, Associate Professor Raymond Wong, have for the first time found that the genes TMEM97 and POLDIP2 play a role in regulating oxidative stress—a part of aging in the macula.

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Genetics Gene Regulation Research 97

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

pharmaphorum

SEPTEMBER 24, 2024

After various twists and turns in the regulator path, Ipsen has secured EU approval for Kayfanda, its treatment for severe itching (pruritus) in patients with the rare liver disease Alagille syndrome (ALGS).Kayfanda

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Genetics Regulation Drugs 118

Medical Xpress

FEBRUARY 21, 2025

Now, researchers at the University of Copenhagen and the Max Planck Institute of Biochemistry have revealed how these proteins are regulated during childhood and adolescence, providing a vital foundation for understanding disease mechanisms and developing better diagnostic tools.

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Protein Genetics Development Regulation 79

BioPharma Reporter

DECEMBER 13, 2022

Annogen has begun a research project with Pfizer to functionally test tens of thousands of disease-related non-coding sequence variants for their effect on gene regulation.

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Genetics Research Gene Regulation 89

Medical Xpress

JANUARY 18, 2023

A new research paper titled "Genetic deficiency and pharmacological modulation of RORα regulate laser-induced choroidal neovascularization" has been published in Aging.

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Genetics Regulation Research 40

Scienmag

JULY 19, 2022

A key genetic mutation that occurs early on in cancer alters RNA “dark matter” and causes the release of previously unknown RNA biomarkers for cancer early detection, a new study by UC Santa Cruz researchers published in the journal Cell Reports shows. Kim A key genetic mutation that occurs early on in […]. Credit: Daniel H.

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RNA Regulation Gene Genetics 64

Pharmaceutical Technology

JUNE 22, 2022

It works by down-regulating fibroblast growth factor receptor 3 (FGFR3) signalling and subsequently boosting endochondral bone formation. CNP was discovered as a natural regulator of bone growth in Japan in 1990 so we are especially proud to be able to offer a therapeutic choice there.”.

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Pharmaceutical Technology

APRIL 26, 2023

This marks the first-ever designation for genetically modified gamma-delta T cell therapies. INB-400, an autologous, genetically engineered gamma-delta T cell therapy, is the company’s DeltEx chemotherapy-resistant autologous and allogeneic drug-resistant immunotherapy (DRI) technology.

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Pharma Mirror

MAY 7, 2021

Avance Clinical is accredited as a gene technology CRO under the Office of the Gene Technology Regulator (OGTR). This accreditation recognizes that Avance Clinical has the CRO resources and internal processes in place to effectively manage pre-clinical and clinical trials for products involving genetically modified organisms (GMOs).

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XTalks

FEBRUARY 21, 2025

Current Share Price: PWS is a complex, genetically determined neurodevelopmental disorder that affects multiple organ systems. And hyperphagia is common not only to PWS but also to hypothalamic obesity, binge-eating disorder and certain rare genetic disorders like Bardet-Biedl syndrome.

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Genetics Hormones Gene Expression Gene 59

Scienmag

MAY 5, 2021

Researchers have mapped the gene activity of osteocytes to improve their understanding of skeletal disease Credit: Garvan Institute of Medical Research Research led by the Garvan Institute of Medical Research has for the first time mapped the unique genetic profile of the skeleton’s ‘master regulator’ cells, known as osteocytes.

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Gene Genetics Regulation Research 98

Scienmag

JANUARY 22, 2021

Cryo-electron microscopy study allows researchers to visualize structural changes in an E.

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RNA Production Regulation Bacteria 85

Medical Xpress

APRIL 7, 2023

A team of scientists at the Medical University of South Carolina (MUSC) has identified a stress-regulated gene that plays a role in the link between long-term stress and a common type of depressive behavior in mice.

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Genetics Gene Scientist Regulation 75

pharmaphorum

AUGUST 4, 2024

An FDA advisory committee has recommended approval of Zevra Therapeutics' arimoclomol for the ultra-rare disorder Niemann-Pick disease, three years after the regulator rejected the drug.The Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted 11 to five that the data support the approval of the drug for Niemann-Pick disease type C (NPC), a (..)

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Genetics Regulation Drugs 85

XTalks

DECEMBER 9, 2024

This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible. Programs like the Rare Disease Endpoint Advancement Pilot encourage collaboration with the FDA to establish novel surrogate endpoints.

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Clinical Trials Clinical Trials Compliance Trials 105

Scienmag

MAY 12, 2021

Mutations can disrupt protein binding through a “burr effect” thus interfering with the regulation of cell growth Credit: Kümmel team/Oeckinghaus team Tuberous Sclerosis Complex (TSC) affects between one and two of every 10,000 new-born babies.

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Genetic Disease Research Genetics Protein 94

STAT News

JULY 26, 2022

The Food and Drug Administration has agreed to review a treatment for a rare, genetically defined form of ALS developed by Biogen, even though results from a clinical trial failed to show a definitive benefit for patients. Biogen said Tuesday that U.S.

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Clinical Trials Clinical Trials Trials Genetics 98

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 Genomics Genome 62

Scienmag

JULY 7, 2022

MADISON — Two of the most common genetic changes that cause cells to become cancerous, which were previously thought to be separate and regulated by different cellular signals, are working in concert, according to new research from the University of Wisconsin–Madison.

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Genetics Regulation Drugs Research 79

XTalks

NOVEMBER 15, 2024

Drug resistance in DEEs often stems from genetic and molecular factors unique to each syndrome, limiting standard treatments’ effectiveness. Bexicaserin, an investigational oral therapy, works by activating specific brain receptors, notably the 5-HT2C receptor, which helps regulate neurological functions.

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Trials Clinical Trials Clinical Trials Genetics 97