Worldwide Clinical Trials

JANUARY 24, 2024

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.

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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.

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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.

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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. The global nature of cell and gene therapy trials adds another layer of complexity.

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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?

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Clinical Trials Clinical Trials Genetics Trials 130

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.

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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 […]

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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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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.

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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

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.

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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.

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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.

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Genetic Engineering Gene Engineer Genetics 246

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.

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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.

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Gene Expression Gene Genetics Research 242

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.

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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.

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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.

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Clinical Trials Clinical Trials Trials Genetics 66

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.

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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.

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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 […]

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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.

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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.

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Pharmaceutical Technology

APRIL 13, 2023

Following the increased use of telemedicine during the Covid-19 pandemic, the potential of digital technologies in communication, data collection, and analysis has become increasingly realised by patients, healthcare systems, and clinical trial sponsors.

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Pharmaceutical Technology

MAY 24, 2023

It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene.

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pharmaphorum

MARCH 10, 2022

18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. The post Scenic Bio raises $31m for genetic modifier platform appeared first on.

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Pharmaceutical Technology

JUNE 29, 2022

A CD19-directed genetically modified autologous T cell immunotherapy, Yescarta is indicated for FL patients who have received three or more previous lines of systemic therapy. The latest approval is based on findings from the global, single-arm Phase II ZUMA-5 clinical trial in 122 relapsed or refractory FL patients.

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Gene Therapy Clinical Trials Clinical Trials Genetics 278

Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies. The Purpose of INTERACT Meetings.

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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.

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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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Gene Therapy Gene Packaging Packaging 130

Pharmaceutical Technology

JUNE 15, 2023

Verve Therapeutics and Eli Lilly and Company have entered an exclusive research partnership to advance the former’s preclinical stage in vivo gene editing programme targeting lipoprotein(a) (Lp(a)) to treat atherosclerotic cardiovascular disease (ASCVD). Lilly will provide funding for the Phase I clinical trials.

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Pharmaceutical Technology

NOVEMBER 7, 2022

A lead product candidate of the company, SNK01 is presently being analysed as a single agent and along with other agents, including checkpoint inhibitors and cell engagers, in trials to treat advanced refractory solid tumours. . Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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.

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Genetics Research Genome Genomics 114

Pharmaceutical Technology

DECEMBER 12, 2022

An investigational late-stage product candidate, CART-ddBCMA is being analysed in the Phase II iMMagine-1 clinical trial at present. It leverages the new D-Domain binder of Arcellx and contains autologous T cells genetically modified for targeting multiple myeloma. By Cytiva Thematic.

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Development Gene Therapy Clinical Trials Clinical Trials 264

XTalks

FEBRUARY 2, 2022

The Israeli Society of Gene and Cell Therapy (ISGCT) is hosting its upcoming annual meeting in-person this year to encourage networking and the sharing of information about genetic and cellular therapies. This 2022 meeting will also have a free online panel discussion about gene delivery technologies for therapeutic applications here.

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XTalks

OCTOBER 26, 2021

After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Scienmag

APRIL 28, 2021

Routine genetic screening for all children with cardiomyopathy could make a powerful difference in outcomes and even survival Credit: Douglas Levere/University at Buffalo BUFFALO, N.Y.

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