Camargo

JULY 27, 2021

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

AuroBlog - Aurous Healthcare Clinical Trials blog

MAY 7, 2024

When research tells us that our grandparents’ diets can affect our health decades later, it can be hard to shake the feeling that genetics spells out the trajectory of our wellbeing. But how much of our health is actually determined by our genes, and what effect does our lifestyle have, for better or worse? Can […]

Genetics 245

Genetics Gene Research Clinical Trials 245

Bio Pharma Dive

SEPTEMBER 12, 2024

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.

Gene Therapy 281

Gene Therapy Gene Genetics Trials 281

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 9, 2024

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.

Gene Therapy 244

Gene Therapy Gene Genetics Clinical Research 244

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 22, 2024

(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.

Scientist 244

Scientist Gene Genetics Research 244

Worldwide Clinical Trials

NOVEMBER 12, 2024

By Amy Raymond, PhD, PMP, Executive Director, Therapeutic Strategy Lead, Rare Disease Cell and gene therapies (CGTs) include cutting-edge approaches that offer the hope of a healthier, happier, and better tomorrow for a wide range of patient populations. Operationalizing these trials requires proactive and flawless management at every stage.

Gene Therapy 182

Gene Therapy Clinical Supply Trials Gene 182

AuroBlog - Aurous Healthcare Clinical Trials blog

DECEMBER 21, 2022

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.

Genetics 239

Genetics Gene Scientist Research 239

AuroBlog - Aurous Healthcare Clinical Trials blog

APRIL 29, 2024

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 […]

Gene 243

Gene Genetics Protein Research 243

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

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 […]

Genetic Disease 189

Genetic Disease Genetics Gene Editing Medicine 189

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. Recent years have seen a steadily growing number of approvals for cell and gene therapies, which has spurred on the community to continue innovating in this space.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

Pharmaceutical Technology

JUNE 13, 2023

Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. The asset, now transferred to Beacon Therapeutics, is currently in Phase II clinical trials.

Gene Therapy 246

Gene Therapy Gene Trials Clinical Trials 246

Pharmaceutical Technology

MAY 24, 2023

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases.

Clinical Trials 246

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain 246

Worldwide Clinical Trials

DECEMBER 12, 2022

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?

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 130

Pharmaceutical Technology

JANUARY 5, 2023

Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

Gene Therapy 237

Gene Therapy Gene Development Engineer 237

Pharma Mirror

APRIL 28, 2021

Catalent will provide process development and CGMP manufacturing of AavantiBio’s adeno-associated viral (AAV) vector-based therapeutic candidate for use in clinical trials in the U.S. and Europe. Catalent will further support process optimization and look to reduce material.

Gene Therapy 130

Gene Therapy Genetic Disease Genetics Gene 130

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

XTalks

JANUARY 6, 2025

CF is a progressive genetic disease caused by defective CFTR proteins, which are crucial for regulating salt and water movement in cells. The CFTR gene itself is complex, with over 2,000 known mutations. Without functional CFTR, thick mucus builds up in organs like the lungs, leading to chronic infections and lung damage.

Gene Therapy 105

Gene Therapy Protein Genetic Disease Gene 105

AuroBlog - Aurous Healthcare Clinical Trials blog

AUGUST 7, 2023

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.

Genetic Engineering 246

Genetic Engineering Gene Engineer Genetics 246

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 The post Avance Clinical Expands Gene Technology Clinical Trial Services to Meet $17.4

Clinical Trials 130

Clinical Trials Clinical Trials Gene Marketing 130

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia A custom-made, one-dose gene therapy, Zynteglo is indicated for such patients who need red blood cells (RBCs) transfusions on a regular basis.

Gene Therapy 246

Gene Therapy Gene Genotype In-Vivo 246

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

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.

Clinical Trials 130

Clinical Trials Clinical Trials Trials Genetics 130

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.

Gene Editing 145

Gene Editing Gene Gene Therapy In-Vivo 145

XTalks

FEBRUARY 4, 2025

Related: FDA Approves New Use for Ozempic to Reduce the Risks of Kidney Disease Maze stands out in the biotech space with its Compass platform, which analyzes genetic data to identify disease-linked variants. For complex diseases like chronic kidney disease (CKD), where genetics play a significant role, such a platform holds promise.

Clinical Trials 66

Clinical Trials Clinical Trials Trials Genetics 66

pharmaphorum

JUNE 15, 2021

When Biogen acquired Nightstar Therapeutics in 2019, it added a pair inherited retinal disorder (IRD) gene therapies that it hoped would accelerate a push into the fast-evolving category. .

Gene Therapy 105

Gene Therapy Gene Trials Genetics 105

Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.

RNA 245

RNA Gene Expression Gene Gene Therapy 245

pharmaphorum

FEBRUARY 11, 2022

Data from the first patients enrolled into Regenxbio’s trial of its gene therapy for rare inherited disease mucopolysaccharidosis type I (MPS I) – also known as Hurler syndrome – has shown the first signs of clinical activity. The post Regenxbio expands MPS I gene therapy trial after first look at data appeared first on.

Gene Therapy 111

Gene Therapy Gene Trials FDA Approval 111

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. Roughly 80% of rare diseases are due to a known genetic driver. Over the years, I have attended several Global Genes Summits.

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

pharmaphorum

AUGUST 23, 2022

France’s BrainVectis, a subsidiary of Bayer’s Asklepios BioPharma (AskBio) unit, has been given the green light by regulators in France to start dosing patients with its gene therapy candidate for devastating neurodegenerative disorder Huntington’s disease.

Gene Therapy 105

Gene Therapy Gene Trials Protein 105

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

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

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

Development 246

Development Gene Therapy Protein Gene 246

Pharmaceutical Technology

MAY 25, 2023

Pushing back an initial deadline, the US Food and Drug Administration (FDA) has proposed a new regulatory action date of 22 June, by which time the agency will assess the logistics of a possible approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene therapy.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

STAT News

JULY 11, 2022

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

Gene 111

Gene Gene Therapy Genetic Engineering Engineer 111

pharmaphorum

OCTOBER 25, 2022

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates. ” The post Astellas makes another gene therapy play, takes stake in Taysha appeared first on.

Gene Therapy 104

Gene Therapy Gene Genetic Disease Genetics 104

XTalks

APRIL 24, 2023

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.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95