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

pharmaphorum

OCTOBER 12, 2021

The research has focused on Alzheimer’s associated with an apolipoprotein E4 (APOE4) gene variant known to be a major risk factor for late-onset forms of the disease. All told, five drugs emerged with a gene expression signature that the researchers believed might help neutralise the disease, but the strongest candidate was bumetanide.

Gene Expression 111

Gene Expression Drugs FDA Approval Gene 111

Scienmag

SEPTEMBER 29, 2020

By changing gene expression first, some immunotherapies may work better, models show Credit: (courtesy of Hackensack Meridian Health) September 29, 2020 – Nutley, NJ – A one-two punch of changing gene expression, then deploying immune checkpoint inhibitors, shows promise in battling one of the most treatment-resistant types of cancer in (..)

Gene Expression 52

Gene Expression Scientist Gene Clinical Trials 52

Drug Discovery World

MAY 16, 2023

FDA Orphan Drug Designation provides SiSaf with incentives such as tax credits for clinical trials, exemption from user fees, and expanded marketplace exclusivity. There are currently no approved treatments for Osteopetrosis ADO2 and no other treatments currently in clinical trials.

Gene Expression 52

Gene Expression RNA Clinical Trials Clinical Trials 52

Scienmag

AUGUST 4, 2020

Histone deacetylases (HDACs) are enzymes that help modulate gene expression by removing […].

Gene Expression 82

Gene Expression Gene Scientist Research 82

Drug Discovery World

AUGUST 22, 2023

In addition to the biomarkers analysed in the study, changes in gene expression were measured by mRNA sequencing on participants’ blood, for placebo and SFX-01 treated subjects that received 600mg once daily. It delivers the drug at highly relevant blood levels within the timeframes expected of an enteric coated formulation.”

Gene Expression 52

Gene Expression In-Vitro Gene Trials 52

Drug Discovery World

MARCH 21, 2023

In July 2022, DDW reported that molecular genetics company Mainz Biomed had enrolled its first patient in an international clinical study assessing the potential combination of gene expression (mRNA) biomarkers with a diagnostic test for colorectal cancer.

Drugs 52

Drugs Gene Expression RNA Clinical Trials 52

Drug Discovery World

AUGUST 16, 2023

A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy. “Our findings paint a complex picture – with many, dynamic factors driving how tumours respond to treatment.”

Genetics 52

Genetics Gene Hormones RNA 52

Delveinsight

JULY 24, 2021

Ovid Therapeutics is running Phase III clinical trials for its drug, Gaboxadol ( OV101 ). In addition to this, Angelman syndrome pipeline also involves gene therapy candidates such as GTX-101 which is a therapy of GeneTx Biotherapeutics.

Gene Therapy 96

Gene Therapy Gene Expression Gene RNA 96

XTalks

MARCH 13, 2024

XTALKS WEBINAR: Addressing Ophthalmology Clinical Trial Design and Effective Data Monitoring Committee Strategies On-Demand: Thursday, February 8, 2024 Register for this free webinar gain insights into statistical data strategies for ophthalmology clinical trials and learn best practices for establishing and reporting ophthalmic data.

Gene Therapy 111

Gene Therapy Gene Life Science DNA 111

Drug Discovery World

JULY 10, 2023

Artificial intelligence (AI), gene editing, synthetic biology and more, are beginning to prove their usefulness in drug design and development, taking therapies away from the one-size-fits-all approach, towards more definable targets that operate more effectively. This was done to assess the accuracy of the team’s machine-learning algorithms.

Gene Therapy 52

Gene Therapy Gene Scientist Gene Editing 52

Drug Discovery World

MARCH 3, 2023

Epigenetics discovery could lead to new class of cancer drugs 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.

Drugs 52

Drugs Gene Expression Genome Genomics 52

XTalks

MAY 31, 2022

XTALKS WEBINAR: All Means All: The Road to Inclusivity in Clinical Trials. Register for this free webinar to learn what the FDA’s new draft guidance means for diverse and inclusive trials. Clinical Trials of Vtama. Live and On-Demand: Tuesday, July 12, 2022, at 11am EDT (4pm BST/UK). Dermavant’s Future Targets.

FDA Approval 98

FDA Approval Clinical Trials Clinical Trials Trials 98

pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.

Gene Therapy 52

Gene Therapy Gene Gene Sequencing Gene Expression 52

The Pharma Data

JULY 12, 2021

The AI approach helps model a disease using a map of successive changes in gene expression at the onset and during the progression of the disease. The underlying algorithms ensure that the identified gene expression patterns are ‘invariant’ regardless of different disease cohorts. Nature Communications, 2021.

Drugs 52

Drugs Gene Expression Big Data Gene 52

pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

Trials 59

Trials Gene Silencing RNA Gene Expression 59

XTalks

JUNE 17, 2022

It blocks the activity of JAK1 and JAK2 to interfere with the JAK-STAT signaling pathway to dampen the production of inflammatory cytokines through the modulation of gene expression. Olumiant is a Janus kinase (JAK) inhibitor that was first discovered by Incyte and then licensed to Eli Lilly.

FDA Approval 97

FDA Approval Gene Expression Cosmetics Trials 97

pharmaphorum

NOVEMBER 22, 2022

Ut comes just as FogPharma is preparing to start clinical trials of alpha-helical polypeptide candidate FOG-001. The Series D comes a little over 18 months after the six-year-old biotech’s third-round financing netted it around $107 million, taking its total raised to date north of $360 million.

Gene Expression 52

Gene Expression Packaging Packaging Clinical Trials 52

Drug Discovery World

DECEMBER 12, 2022

According to the publication, “A small clinical trial has shown that researchers can use CRISPR gene editing to alter immune cells so that they will recognise mutated proteins specific to a person’s tumours. Those cells can then be safely set loose in the body to find and destroy their target. . “It

Drugs 52

Drugs Gene Editing Genome Genomics 52

Drug Discovery World

FEBRUARY 13, 2023

RNA transcript expression levels can provide insight into underlying biology – eg. which genes are upregulated, and which ones are downregulated – which is very useful when studying different disease states. DDW: Can RNA profiling be used past the point of early development to monitor how drugs perform within clinical trials?

RNA 52

RNA Drugs Gene Expression Gene 52

The Pharma Data

MARCH 3, 2022

This finding stemmed from more than two decades of her basic research into a gene in pancreatic islets called TXNIP. In 2014, Shalev’s UAB research lab reported that verapamil completely reversed diabetes in animal models, and she announced plans to test the effects of the drug in a human clinical trial.

Insulin 52

Insulin Gene Expression Gene Protein 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.

Gene 40

Gene Gene Therapy Gene Expression Regulation 40

Camargo

JULY 8, 2021

The UCLA researchers measured monoamine oxidase A (MAOA) gene expression in tumors from cancer patients and found that high MAOA expression was associated with shorter survival times and turned to a mouse model to investigate these observations with promising results.

Development 130

Development Bioequivalency Gene Expression Drugs 130

Drug Discovery World

OCTOBER 3, 2022

The researchers performed flow-cytometry EMT characterisation of CTCs as compared to EMT gene expression in tumour tissue and investigated expression correlation with respect to clinical outcomes. The expression of EpCAM and N-cadherin were utilised to characterise CTC status as epithelial or EMT.

Gene Expression 52

Gene Expression Genome Genomics Clinical Trials 52

pharmaphorum

JANUARY 10, 2023

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. In April 2022, the company also announced it was halting the development of three gene therapy candidates for Duchenne muscular dystrophy (DMD).

Gene Therapy 119

Gene Therapy Gene Licensing Licensing 119

Drug Discovery World

APRIL 16, 2023

The clinical benefit was observed regardless of the tumour mutational burden (TMB) status, according to results from the Phase IIb KEYNOTE-942 clinical trial presented at the AACR Annual Meeting 2023, held April 14-19. The association between this treatment approach and TMB will be further explored in upcoming studies.

Vaccination 52

Vaccination Vaccine Immune Response Gene Expression 52

The Pharma Data

OCTOBER 15, 2020

Immuno-oncology and CAR T cells energized the field of regenerative medicine, but for cell and gene to deliver on their promises, new, disruptive technologies and new modes of operation are needed. Other cell and gene companies are advancing solutions, too. Often, there is no regulatory precedence so there are holes to be addressed.

Manufacturing 52

Manufacturing Gene Therapy Gene Gene Expression 52

Roots Analysis

NOVEMBER 3, 2023

NAD + is involved in a wide range of cellular processes, including energy metabolism, DNA repair, and gene expression. Several factors have contributed to the failure of NAMPT inhibitors in clinical trials, including poor pharmacokinetic properties, limited efficacy, and toxicity issues.

Development 40

Development Drugs Trials FDA Approval 40

Drug Discovery World

FEBRUARY 10, 2023

Magnus Persson is a physician and scientist with a background in both academia and industry, working for the likes of Sanofi on clinical trials activities and across venture capital firms in the US. He is now a founding partner of EIR Ventures, a venture capital firm investing in innovative companies across the Nordics.

Life Science 52

Life Science Drugs Gene Therapy Manufacturing 52

Pharmaceutical Technology

JANUARY 24, 2023

The investigational adeno-associated virus (AAV) gene therapy candidate NGN-401 is claimed to be the first to deliver the full-length human MECP2 gene using the company’s Expression Attenuation via Construct Tuning (EXACT) gene regulation technology.

Gene Therapy 208

Gene Therapy Gene Gene Expression Regulation 208

Drug Discovery World

MARCH 26, 2024

Mutations in genes encoding urea cycle enzymes result in insufficient levels of these important proteins. CMP-CPS-001 is an antisense oligonucleotide (ASO) designed to amplify CPS1 mRNA by harnessing fundamental cellular gene expression control mechanisms.

Trials 52

Trials Gene Expression Protein Gene 52

Drug Discovery World

DECEMBER 15, 2022

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

Gene Editing 52

Gene Editing Gene In-Vivo Genome 52

pharmaphorum

JUNE 3, 2021

Further evidence of its potential efficacy as a drug candidate was also established based on diminished hypothalamic inflammatory gene expression. Demonstrating a robust safety profile can be considered a major milestone because previous melanocortin-based drug candidates had significant cardiovascular side-effects in clinical trials.

Gene Expression 40

Gene Expression Drugs Research Medicine 40

The Pharma Data

DECEMBER 14, 2020

Oncocyte has also launched DetermaIO , a gene expression test that identifies patients more likely to respond to checkpoint inhibitor immunotherapies, for research and pharma clinical trial use.

Licensing 52

Licensing Licensing Marketing Gene Expression 52

Drug Discovery World

FEBRUARY 6, 2023

Typically, the data obtained from such studies is large and high dimensional with thousands of molecules being measured for each individual sample requiring special statistical, data science, and bioinformatics skills to integrate the information and correlate it with the clinical outcome of interest.

Big Data 52

Big Data RNA Bioinformatics Vaccination 52

Drug Discovery World

JANUARY 24, 2024

Can you elaborate on the clinical timeline for these small molecules? Fibrocor is on track to complete all IND-enabling work to support IND filing and initiation of clinical trials in 2024. The gene expression data were generated by RNAseq on biopsies stored at -78°C. PW: FIB992 is our lead compound.

Research 57

Research Gene Expression Drugs Development 57

Drug Discovery World

APRIL 30, 2024

At the start of 2024, the first patients in a clinical trial investigating an mRNA cancer therapy were dosed. The Mobilize trial is investigating the mRNA-4359 therapy and its potential for treating melanoma, lung cancer and other solid tumour cancers. However, these therapies can cause severe adverse effects.

RNA 52

RNA Protein Vaccination Vaccine 52