Worldwide Clinical Trials

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

Gene Editing 189

Gene Editing Gene Gene Therapy In-Vivo 189

AuroBlog - Aurous Healthcare Clinical Trials blog

MARCH 17, 2024

Similar genetic changes in two different types of brain cells may contribute to cognitive impairment in schizophrenia and aging. US researchers examined gene expression in more than a million brain cells collected post-mortem from 191 donors.

Gene Expression 242

Gene Expression Gene Genetics Research 242

Pharma Mirror

MAY 7, 2021

The leading Australian CRO for biotechs and Frost & Sullivan Asia-Pacific CRO Market Leadership Award winner, Avance Clinical, has expanded its gene technology clinical trial services to meet the increasing global demand predicted to reach $17.4 billion by 2023.

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Clinical Trials Clinical Trials Gene Marketing 130

Worldwide Clinical Trials

JULY 29, 2024

Evolving sentiments recognize obesity as a medical condition and lifestyle choice as well as a chronic disease with genetic, psychological, cultural, and environmental causes (Figure 1). DEXA (Dual-Energy X-ray Absorptiometry) is the optimal method for accurately measuring body composition in clinical research.

Clinical Research 130

Clinical Research Research Clinical Trials Clinical Trials 130

AuroBlog - Aurous Healthcare Clinical Trials blog

JULY 19, 2023

Genetic inheritance is a physical link that tethers our birth parents; one-half of two sets of chromosomes meshing together to form our own unique set of molecular instructions for life.

Gene 187

Gene Genetics Clinical Trials Clinical Trials 187

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics. The current research areas of interest in India include precision medicine which treatments based on individual genetic profiles to improve […]

Medicine 189

Medicine Research Gene Therapy Genetics 189

Worldwide Clinical Trials

OCTOBER 24, 2023

Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”

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Gene Gene Editing Gene Therapy Clinical Trials 130

Pharmaceutical Technology

OCTOBER 18, 2022

CSF biomarkers have been a popular area of interest in neurodegenerative disease research with previous studies related to Alzheimer’s and Multiple Sclerosis exploring the potential of their use. In PD clinical research, many biomarkers, such as neurofilament, can be measured within the CSF using a lumbar puncture.

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Protein Genetics Gene Clinical Research 264

XTalks

MAY 23, 2024

One of the more intriguing developments in cancer research in recent years is the growing understanding of clonal hematopoiesis, a phenomenon where blood cells expand from a single clone due to genetic mutations. Clonal hematopoiesis increases in prevalence with age and can precede hematologic malignancies.

Genetics 114

Genetics Research Genomics Genome 114

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science 130

Life Science Genetic Disease Genomics Genome 130

XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

Gene Therapy 98

Gene Therapy Clinical Trials Clinical Trials Gene 98

Medical Xpress

JANUARY 5, 2023

This approach contrasts with the typical phenotype-first approach to clinical research, which starts with clinical findings. The study was published in the American Journal of Human Genetics.

Genomics 75

Genomics Genome Genetics Genotype 75

XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

Clinical Trials 88

Clinical Trials Clinical Trials Trials Gene Therapy 88

Cloudbyz

JUNE 26, 2023

Generative artificial intelligence (AI) techniques are increasingly being applied in clinical research to enhance various aspects of medical data analysis, diagnosis, treatment planning, and drug discovery. These notes have been used to advance research in NLP and clinical decision support systems.

Clinical Research 97

Clinical Research Research Clinical Trials Clinical Trials 97

Olympian Clinical Research

SEPTEMBER 27, 2023

While the exact cause of vitiligo remains a subject of ongoing research, genetics has emerged as a significant factor in understanding its onset and progression. In this deep dive, we’ll explore the genetic links associated with vitiligo and address the burning question: Is vitiligo hereditary?

Genetics 52

Genetics Gene Clinical Research Research 52

Worldwide Clinical Trials

MARCH 20, 2024

One way to ensure customized disease management is to utilize pharmacogenomics (PGx) in your clinical trial. As the study of how an individual’s genes affect their response to drugs, pharmacogenomics is a critical factor in developing effective and safe drugs and dosages in clinical trials.

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Clinical Trials Clinical Trials Trials Genetics 130

XTalks

APRIL 17, 2025

Approximately 80% of these diseases have a genetic basis , and many manifest in childhood , often with significant challenges in achieving a timely and accurate diagnosis. This happens due to reduced activity of tissue-nonspecific alkaline phosphatase (TNAP), an enzyme encoded by the ALPL gene.

Clinical Research 52

Clinical Research Research Clinical Trials Clinical Trials 52

Medical Xpress

NOVEMBER 21, 2022

In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinic researchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).

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Gene Therapy Gene Genomics Genome 73

Advarra

AUGUST 8, 2023

Gene therapy research is booming in the clinical setting. In this blog, we summarize the growth, risks, and regulatory requirements for gene therapy research. We also discuss how a centralized biosafety review process can benefit this type of research.

Gene Therapy 52

Gene Therapy Gene Research Genetics 52

Advarra

JUNE 12, 2023

Gene therapy research is exciting and full of promise, but because of the risks involved, it’s also highly regulated, requiring an institutional biosafety committee (IBC) to provide additional oversight and risk assessment. The IBC also reviews to confirm a comprehensive risk mitigation plan is in place prior to starting the research.

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Genetic Engineering Gene Therapy Genetics Engineer 98

XTalks

MARCH 17, 2025

Looking ahead, the company plans to expand into North American and European markets by preparing localized production and regulatory filings, and it aims to initiate clinical research for its A+LCGuard lung cancer screening kit by mid-2025 underscoring its commitment to rapid, affordable and reliable diagnostics.

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Production Manufacturing DNA Genetics 71

Medical Xpress

FEBRUARY 21, 2025

A clinical research team from the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), and international genetic researchers led a global research study using multi-omics analysis and identified a novel gene, DDX39B, for a rare disease.

Gene 40

Gene Research Genetics Clinical Research 40

pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells.

Genomics 105

Genomics Genome Clinical Trials Clinical Trials 105

Cloudbyz

JULY 31, 2024

This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinical research and post-market care. For instance, in oncology, patients with specific genetic mutations (e.g.,

Gene Therapy 52

Gene Therapy Gene Medicine In-Vitro 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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Gene Therapy Gene Manufacturing Production 52

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. It’s a very exciting time in gene therapy.

Gene Therapy 52

Gene Therapy Gene Clinical Trials Clinical Trials 52

Roots Analysis

MARCH 24, 2022

Genomics is the study of genetic material within a cell, and transcriptomics is the study of gene activity in different cells. This approach is often employed to determine the effect of genetic modifications or therapeutics in pharmacokinetic studies of drug metabolism. Untargeted Metabolomics. Our Social Media Platform.

Clinical Research 52

Clinical Research Development Research Drug Delivery Systems 52

pharmaphorum

NOVEMBER 27, 2022

Testing would also help identify patients who may be eligible to enrol into clinical trials of precision cancer drugs, according to the group, which has just published a white paper making its case and is presenting the idea to the Scottish Cancer Conference in Edinburgh today.

Genomics 104

Genomics Genome Genetics Medicine 104

XTalks

SEPTEMBER 22, 2020

Fitbit isn’t the only tech company developing heart health apps; in late 2019, Apple Watch introduced an app known as Invitae Discover which serves as a clinical research platform that gathers heart health data. The aim of the study is to further understand the genes that cause cardiovascular disease.

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Gene Doctor Doctors Clinical Trials 107

Roots Analysis

AUGUST 29, 2023

Over the years, cell and gene therapies (CGT) and other advanced therapy medicinal products (ATMPs) have managed to capture the interest of both drug developers and healthcare investors. Their popularity can be attributed to various factors, such as minimal side effects, clinical efficacy and their personalized nature.

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Gene Therapy Gene Research Manufacturing 40

XTalks

FEBRUARY 24, 2023

“Many rare disease communities are seeing gene therapies as potential game changers, since most rare diseases are genetically driven,” Dr. Raymond says. Hence, one conversation that needs to take place is between the sponsors developing gene therapies and the payers who in effect “gate” patient access to these novel treatments.

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Development Clinical Trials Clinical Trials Gene Therapy 98

My Local Study

JANUARY 22, 2024

Fragile X Syndrome (FXS) is a rare genetic condition that affects individuals in varying degrees, impacting cognitive and social development. Caused by a mutation in the FMR1 gene, FXS is characterized by a range of symptoms, and no known cure.

Genetics 52

Genetics Gene Development Clinical Research 52

pharmaphorum

MAY 18, 2022

To support the wide range of patients this complex disease impacts, who suffer from a diverse range of symptoms, it is critical that research into this area continues. “At Charl believes we are witnessing the ‘decade of the brain’ with significant leaps in scientific understanding of disease pathways and genetic correlations.

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Gene Therapy Gene Genetics Development 92

WCG Clinical

AUGUST 27, 2024

The NIH Guidelines also stipulate that at least two members of each IBC come from the surrounding community and be unaffiliated with the institution where the research is occurring apart from their membership on the IBC. Many of those original concerns of the 1970s and 1980s surrounding genetic engineering have lessened over time.

Genetic Engineering 52

Genetic Engineering Engineer Compliance Genetics 52

XTalks

SEPTEMBER 17, 2020

While clinical researchers occasionally need to identify and validate a new surrogate endpoint for a given trial, choosing endpoints that have been previously used as the basis of NDAs and BLAs can clear the path to eventual approval. Mutations in the gene encoding ?-Gal Prevalence: ABCG5 gene mutation: 1 in 2.6

Trials 98

Trials Gene Hormones Clinical Trials 98

WCG Clinical

NOVEMBER 17, 2023

It also establishes the role of the Institutional Biosafety Committee (IBC) in providing local oversight of such research. At the time this change was implemented, methods used to deliver rsNA to cells or research participants typically involved the use of viral vectors that were subject to the NIH Guidelines.

Genomics 52

Genomics Genome Gene Editing Genetic Engineering 52