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The convergence of gene therapies and clinicalresearch is pushing the boundaries of what’s possible in ophthalmic care, offering hope for more effective treatments and potential cures for a range of vision-threatening conditions.
(Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows. Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth.
Scientists have found an extremely subtle twist in the genetics of aging cells, one that seems to make them increasingly less functional as time goes on.
A unique genetic mutation in two siblings – that has never been seen in anyone else – has been discovered by UK researchers at the University of Exeter, pointing the way towards new treatment options for type 1 diabetes. The mutation is in the gene for a protein called programmed death-ligand 1 (PD-L1), and a […]
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.
We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics. In […]
New research from ETH Zürich in Switzerland could see future wearable devices (with perhaps a few implants and a touch of genetic engineering) boost our health directly. Fitness trackers help you stay healthy by keeping count of your steps and monitoring your heart rate, driving you on to hit those cardio goals.
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.
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.
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.
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 clinicalresearch.
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.
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.”
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 […]
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 clinicalresearch, many biomarkers, such as neurofilament, can be measured within the CSF using a lumbar puncture.
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.
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.
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.
This approach contrasts with the typical phenotype-first approach to clinicalresearch, which starts with clinical findings. The study was published in the American Journal of Human Genetics.
The use of engineered genetic materials in clinical trials is rapidly expanding, with potential applications for genetic vaccines, gene-modified cellular therapies, and gene therapies. A key part of the IBC’s evaluation is assessing the risks posed by the engineered genetic materials.
Generative artificial intelligence (AI) techniques are increasingly being applied in clinicalresearch 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.
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?
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.
Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Eagleton recently spoke on a webinar with his colleagues from Medpace about lessons learned from successful approaches from rare disease and gene therapy product approvals.
This research community is exploring diverse therapeutic avenues and creative thinking, including gene therapies, cell therapies, immunotherapies, novel drug candidates, and the repurposing of drugs from other indications.
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 clinicalresearch for its A+LCGuard lung cancer screening kit by mid-2025 underscoring its commitment to rapid, affordable and reliable diagnostics.
As Nick Spittal states in this Advarra press release, membership in the Gene Therapy Ready (GTR) site network “allows Velocity to start studies over a month faster and provides a meaningful credential and important validation that increases sponsors’ confidence in our specialized capabilities to conduct complex clinicalresearch safely.”
In a new study published in Circulation: Genomics & Precision Medicine, Mayo Clinicresearchers designed and developed the first suppression-replacement KCNH2 gene therapy for correcting both long QT syndrome (LQTS) and short QT syndrome (SQTS).
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 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.
A clinicalresearch team from the LKS Faculty of Medicine, the University of Hong Kong (HKUMed), and international geneticresearchers led a global research study using multi-omics analysis and identified a novel gene, DDX39B, for a rare disease.
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.
This revolution is enabling the growth of innovative biomarker-based precision medicine and cell and gene therapy, transforming both clinicalresearch and post-market care. For instance, in oncology, patients with specific genetic mutations (e.g.,
A recent clinicalresearch paper highlights the importance of targeting of PPIs in stem cells of cancer. A PPI network was constructed from nine colon stem cell markers in order to identify potential genetic targets in patients. What does this mean for clinicalresearch? Charlotte Di Salvo.
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.
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.
Recombinant DNA technologies and genetically modified biological agents are being adapted for a wide scope of therapeutic applications, and their use is becoming increasingly common in clinical trials. Synthetic mRNA-based therapeutics used in clinicalresearch today also fall into this self-limiting class of recombinant therapeutics.
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.
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.
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.
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 clinicalresearch platform that gathers heart health data. The aim of the study is to further understand the genes that cause cardiovascular disease.
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.
“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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