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At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.
Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. The post Lilly and ProQR to expand genetic medicine development agreement appeared first on Pharmaceutical Technology.
Now an international team of researchers has discovered a new genetic variant in people of African ancestries that appears to restrict HIV replication after an infection sets in. Though more research is needed to confirm their findings, the […]
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.
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.
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide Clinical Trials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. The most common form of CAH, 21-hydroxylase deficiency, disrupts hormone production, leading to cortisol deficiency and an overproduction of androgens.
Indian healthcare industry is of the view that emergence of genetic diagnostics will be a paradigm shift towards personalized medicine. Through the ever-evolving landscape of healthcare, the role of genetic diagnostics offers personalized insights and proactive solutions for disease management and prevention.
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. This holds the potential to cure genetic diseases that have plagued families for generations.
Discover how Step Pharma is harnessing genetics and developing therapeutics targeting CTPS1 to advance cancer research and enhance clinical success. Learn more about their innovative approach.
Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.
In a Policy Forum, Anna Lewis and colleagues argue that, for researchers and others who want to invoke genetic ancestry, there is a scientific and ethical imperative to move away from continental ancestry categories and to instead embrace a view of genetic ancestry that reflects continuous variation and historical depth.
Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease.
Indian healthcare looks at pharmacogenomics, the study of influence of genetic factors on drug response, in patient care as comprehensive genetic tests gain ground.
In a recent webinar, Challenges of Drug Development in Early Parkinsons Disease , Tom Babic, MD, PhD , our Vice President of Scientific Solutions in Neuroscience, uses his 40+ years of experience to share invaluable insights into the current state and future directions of early Parkinsons disease (PD) research. Contact us today to learn more.
But despite continued setbacks, drug developers are hopeful that genetic technologies and a better understanding of biology will bring new options in the not-too-distant future The fatal nerve disease has few treatments.
At a time when many startups have struggled to gain cash, the biotech has raised another $135 million to back its research, a new twist on genetic medicine.
Spun out of research at Nationwide Children’s Hospital, Vironexis claims it will be the first company to start a study testing a cancer drug delivered via the adeno-associated viruses commonly used in genetic medicines.
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.
Another unique challenge in psychiatry trials is the placebo response, which is notably high in mental health research. Each patients experience with mental health conditions is influenced by unique genetic, psychological and environmental factors.
The convergence of gene therapies and clinical research 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.
Genetic testing provides patients with a diagnosis for their illness, helps patients and family members to understand risks of developing new diseases, and can be used to support clinical trial advancement. What are the benefits and limitations to using genetic testing? It is important to consider the type of testing performed (e.g.,
Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.
Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots.
Researchers from Children's Hospital of Philadelphia (CHOP) and Princeton University have discovered a novel genetic disorder associated with neurodevelopmental differences. The discovery identified the disorder in 21 families from all over the world.
Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.
Scientists at the National Institutes of Health have identified new genetic risk factors for two types of non-Alzheimer's dementia. These findings were published in Cell Genomics and detail how researchers identified large-scale DNA changes, known as structural variants, by analyzing thousands of DNA samples.
(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.
The CRISPR specialist joins some of its genetic medicine peers in trimming staff, announcing an “organizational streamlining” to focus resources on prioritiy drug programs.
Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes.
When using genetic screening to identify clinical trial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article! Read the full article!
Researchers studied cancer growth in mice. Nature, 2024) Aging brings two opposing trends in cancer risk: first, the risk climbs in our 60s and 70s, as decades of genetic mutations build up in our bodies. Zhuang et al., But then, past the age of around 80, the risk drops again and a new study […]
Unlock the potential of genetic modification with Precision Biosciences Inc's patented MTEM technology for precise editing of eukaryotic cells. Discover how this mitochondria-targeting engineered meganuclease is revolutionizing geneticresearch.
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.
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.
UT Southwestern Medical Center researchers have identified a genetic mutation that slows the growth of melanoma and potentially other cancers by harnessing the power of the immune system.
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