Genetics and Trials - Clinical Research Informer

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

Worldwide Clinical Trials

DECEMBER 19, 2024

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.

Genetics

Genetics In-Vitro Drugs Clinical Trials

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

XTalks

DECEMBER 18, 2024

CAH is a group of rare genetic disorders affecting the adrenal glands, which produce essential hormones like cortisol, aldosterone and androgens. In the first trial, 122 adults received Crenessity twice daily and 60 received placebo twice daily for 24 weeks. Crenessity marks the third approved drug for Neurocrine.

Genetics

Genetics Hormones Trials Drugs

Amgen to test new way to lower heart risk with large drug trial

Bio Pharma Dive

NOVEMBER 7, 2022

Buoyed by strong mid-stage study results, the biotech will soon start a cardiovascular outcomes trial of a drug designed to target a genetic risk factor called lipoprotein(a).

Drug Trials

Drug Trials Trials Genetics Drugs

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A Healthy Lifestyle Could Offset Genetic Risk For Early Death by a Hopeful 62%

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

Genetics Gene Research Clinical Trials

Scientists Discover a Genetic Variant That Seems to Limit HIV Infection

AuroBlog - Aurous Healthcare Clinical Trials blog

AUGUST 13, 2023

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. A tiny fraction of people are naturally resistant to HIV infections, and scientists want to understand why.

Genetics

Genetics Scientist Research Clinical Trials

We’ve Discovered A Subtle Genetic Imbalance That May Drive Aging

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

Genetics Gene Scientist Research

Ionis, AstraZeneca change a key trial, fueling more questions about a genetic heart disease

Bio Pharma Dive

MAY 2, 2022

The partners increased the size and length of a large, ongoing study in transthyretin amyloidosis cardiomyopathy, a decision that has implications Alnylam Pharmaceuticals, which is testing two of its drugs against the disease.

Genetics

Genetics Trials Drugs

Obesity Clinical Trials: Combining Patient-Centric Care with Scientific Rigor

XTalks

OCTOBER 22, 2024

Given these dire statistics, the need for innovative approaches in obesity clinical trials has never been more urgent. These trials are crucial for understanding and managing obesity while improving strategies to combat this complex health issue. Patient Retention Once participants are enrolled, retaining them becomes the next hurdle.

Clinical Trials

Clinical Trials Clinical Trials Trials Clinical Research

Psychiatry Clinical Trials: Inclusive, Patient-Centric Approaches for Mental Health Research

XTalks

DECEMBER 19, 2024

Despite advancements in neuroscience and psychiatry, clinical trials for mental health conditions such as major depressive disorder (MDD), schizophrenia, bipolar disorder and generalized anxiety disorder face unique challenges. One of the main challenges lies in defining and selecting appropriate trial populations.

Clinical Trials

Clinical Trials Clinical Trials Trials Research

UniQure moves Huntington's gene therapy to next phase of key trial

Bio Pharma Dive

MAY 27, 2021

Safety monitors cleared the company to test a higher dose after reviewing data from 10 patients, providing some hopeful news after recent setbacks for experimental drugs targeting the genetic disease.

Gene Therapy

Gene Therapy Genetic Disease Gene Genetics

Genetic diagnostics promises paradigm shift towards personalized, proactive healthcare

AuroBlog - Aurous Healthcare Clinical Trials blog

FEBRUARY 18, 2024

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.

Genetics

Genetics Medicine Development Drugs

8 Frequently Asked Questions About Genetic Testing in Clinical Trials

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

Clinical Trials Clinical Trials Genetics Trials

Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw

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. This holds the potential to cure genetic diseases that have plagued families for generations.

Genetic Disease

Genetic Disease Genetics Gene Editing Medicine

Gene therapy startup emerges with green light for first-of-its-kind trial

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

Gene Therapy Gene Genetics Trials

Genetically modified herpes combats advanced cancers

Pharma Times

SEPTEMBER 21, 2022

A new genetically engineered virus has delivered a one-two punch in initial phase 1 trial

Genetics

Genetics Genetic Engineering Engineer Trials

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

NOVEMBER 12, 2024

CGTs address rare and complex diseases at the root cause with increasing use in cancer, genetic disorders, and autoimmune diseases. Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys.

Gene Therapy

Gene Therapy Clinical Supply Trials Gene

Indian healthcare looks at pharmacogenomics in patient care as comprehensive genetic tests gain ground

AuroBlog - Aurous Healthcare Clinical Trials blog

NOVEMBER 14, 2023

Indian healthcare looks at pharmacogenomics, the study of influence of genetic factors on drug response, in patient care as comprehensive genetic tests gain ground.

Genetics

Genetics Drugs Clinical Trials Clinical Trials

Biogen shelves ALS drug after early-stage trial failure

Bio Pharma Dive

MARCH 28, 2022

The study setback raises questions over how well a type of genetic medicine can work in adults with central nervous system disorders like ALS.

Genetics

Genetics Trials Medicine Drugs

Verve gets FDA green light to expand base editing trial into US

Bio Pharma Dive

MARCH 24, 2025

The company expects next quarter to read out initial data from its Heart-2 trial, which is testing a one-time treatment for people with genetically elevated cholesterol.

Trials

Trials Genetics

Eloxx repackages ELX-02 data in cystic fibrosis as it eyes pivotal trial

Pharmaceutical Technology

JUNE 15, 2023

Eloxx has revealed its lead candidate ELX-02 improved predicted forced expiratory volume (ppFEV1) in patients with Class 1 cystic fibrosis (CF) in a new analysis of a Phase II trial that missed its efficacy endpoints. The regimen in the trial was a one-week monotherapy period (1.5

Trials

Trials Genetics Protein Marketing

AHA 2024: Plozasiran demonstrates promising Phase III results in FCS adults

Pharmaceutical Technology

NOVEMBER 18, 2024

During AHA 2024, data were presented from the Phase III trial of plozasiran targeting adults with genetically or clinically diagnosed FCS,

Genetics

Genetics Trials

Integrating Bioanalytical Automation, Biomarkers, Pharmacogenomics, & Cost-Effective Strategies

Worldwide Clinical Trials

JANUARY 6, 2025

Through the implementation of full automation in bioanalytical testing PK, immunogenicity, and biomarker assays we streamline trials, reduce costs, save time, and maintain the highest data quality standards. Biomarkers support adaptive trial designs, allowing modifications based on interim results to optimize study outcomes.

Clinical Trials

Clinical Trials Clinical Trials Trials Genetics

STAT+: Emerging class of genetically targeted drugs induce remissions in leukemia patients

STAT News

DECEMBER 10, 2022

NEW ORLEANS — An emerging class of genetically targeted drugs is inducing remissions in about one-third of patients with advanced leukemia, according to updates Saturday from separate clinical trials. Continue to STAT+ to read the full story…

Genetics

Genetics Drugs Clinical Trials Clinical Trials

To Share or Not to Share Failed Genetic Screening Results with Patients

Worldwide Clinical Trials

FEBRUARY 6, 2023

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!

Genetics

Genetics Clinical Trials Clinical Trials Genotype

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

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

Clinical Trials Clinical Trials Gene Therapy Clinical Supply Chain

Alkeus raises funds to launch Stargardt disease therapy gildeuretinol

Pharmaceutical Technology

JUNE 6, 2023

Alkeus Pharmaceuticals has raised $150m in Series B financing to support the registration and launch of gildeuretinol (ALK-001) to treat Stargardt disease, a genetic cause of blindness in children and young adults. The genetic cause is well characterised, but to date there is no effective treatment.

Genetics

Genetics Life Science Trials Clinical Development

Fulgent Genetics acquires therapeutics development company Fulgent Pharma

Pharmaceutical Technology

NOVEMBER 8, 2022

Fulgent Genetics has acquired clinical-stage therapeutics development firm Fulgent Pharma for a total deal price of nearly $100m. According to the deal, the purchase price, contingent on adjustments, has to be paid by Fulgent Genetics as a combination of cash on hand and its shares of common stock.

Genetics

Genetics Development Genome Genomics

This Rare Gene Variant Seems to Protect You From Alzheimer’s Disease

AuroBlog - Aurous Healthcare Clinical Trials blog

JUNE 25, 2024

A woman in Colombia with rare genetic resistance to Alzheimer’s disease may have more company than scientists thought.

Gene

Gene Genetics Scientist Research

How Pharmacogenomics Can Benefit Your Clinical Trial

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

Clinical Trials Clinical Trials Trials Genetics

Ophthalmologists eye gene therapies for vision-threatening conditions as NGS facilitates precision diagnosis

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 9, 2024

According to Dr Anup Rawool, associate director, medical genetics and head, scientific and medical affairs, MedGenome, molecular genetic testing, particularly next-generation sequencing […]

Gene Therapy

Gene Therapy Gene Genetics Clinical Research

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

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. In clinical trials, Alyftrek demonstrated non-inferiority to Trikafta, showing similar improvements in lung function and a reduction in sweat chloride levels.

Gene Therapy

Gene Therapy Protein Genetic Disease Gene

Maze Therapeutics Introduces IPO with Two CKD Candidates in Clinical Trials

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

Clinical Trials Clinical Trials Trials Genetics

A box of 200 mosquitoes did the vaccinating in this malaria trial. That's not a joke!

NPR Health - Shots

SEPTEMBER 22, 2022

Volunteers for the trial put an arm over a box with hundreds of mosquitoes carrying a genetically modified malaria parasite. Here's why they did it that way — and why the trial holds promise. Image credit: Annette M Seilie)

Trials

Trials Genetics Vaccination Vaccine

BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

XTalks

NOVEMBER 26, 2024

The trial demonstrated a 42 percent reduction in combined cardiovascular death and recurrent hospitalizations at Month 30, with cardiovascular-related hospitalizations reduced by half. BridgeBio has been highly active in the genetic diseases space. Participants were randomized to receive acoramidis or placebo over 30 months.

Genetic Disease

Genetic Disease Genetics Protein Clinical Development

FDA Refines Accelerated Approval Pathway with New Draft Guidance

XTalks

DECEMBER 9, 2024

The new framework allows the FDA to require confirmatory trials to be underway before or shortly after approval, potentially reducing the time to confirm a therapys clinical impact from several years to as little as two or three years.

Clinical Trials

Clinical Trials Clinical Trials Compliance Trials

Innovation in Autoimmune and Bone Health Research: Driving Progress for Better Patient Outcomes

Worldwide Clinical Trials

OCTOBER 14, 2024

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. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

Research

Research Clinical Trials Clinical Trials Immune Response

eGenesis scoops $191m for genetically modified pig kidney transplant trial

Pharmaceutical Technology

SEPTEMBER 5, 2024

eGenesis conducted the first-in-human porcine kidney transplant earlier this year, though the patient died two months later.

Genetics

Genetics Trials

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

XTalks

MARCH 19, 2025

Elevidys is the first FDA-approved gene therapy for DMD a rare genetic disorder characterized by progressive muscle degeneration. To date, over 800 patients have received the therapy in clinical trials and real-world settings. It will update Elevidys prescribing information and adjust monitoring protocols as necessary. million.

Gene Therapy

Gene Therapy Gene Genetics Clinical Trials

Reneo cuts 70% of staff following Phase III trial failure

Pharmaceutical Technology

DECEMBER 15, 2023

Reneo’s mavodelpar failed to meet its endpoints in the Phase III trial in primary mitochondrial myopathies, a rare genetic disorder.

Trials

Trials Genetics

Innovating in Autoimmune and Bone Health Research: Driving Progress for Better Patient Outcomes

Worldwide Clinical Trials

OCTOBER 14, 2024

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. One of the biggest hurdles in developing new therapies is recruiting diverse patients for clinical trials.

Research

Research Clinical Trials Clinical Trials Immune Response

Breakthrough: Rare Gene Mutation Offers Clues to Preventing Type 1 Diabetes

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

Gene Genetics Protein Research

Scientists Just Identified Hundreds of Genes That Could Cause Cancer

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 22, 2024

Cancer is typically triggered by some kind of change to our genetic coding that interferes with a cell’s ability to manage its growth. (Richard Jones/Science Photo Library/Getty Images) Scientists have discovered hundreds of genes that could potentially promote cancer, new research shows.

Scientist

Scientist Gene Genetics Research

The Risk of Cancer Fades as We Get Older, And We May Finally Know Wh

AuroBlog - Aurous Healthcare Clinical Trials blog

DECEMBER 25, 2024

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. Researchers studied cancer growth in mice. Zhuang et al., But then, past the age of around 80, the risk drops again and a new study […]

Genetics

Genetics Research Clinical Trials Clinical Trials

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

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 The open-label, single-arm, 24-month Phase III trials enrolled 41 subjects of the age four to 34 years with non-?0/?0 0 genotypes.

Gene Therapy

Gene Therapy Gene Genotype In-Vivo

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

Neurocrine Biosciences’ Crenessity Approved as First New Treatment in Decades for Rare Genetic Disorder CAH

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Amgen to test new way to lower heart risk with large drug trial

Webinars

A Healthy Lifestyle Could Offset Genetic Risk For Early Death by a Hopeful 62%

Scientists Discover a Genetic Variant That Seems to Limit HIV Infection

We’ve Discovered A Subtle Genetic Imbalance That May Drive Aging

Ionis, AstraZeneca change a key trial, fueling more questions about a genetic heart disease

Obesity Clinical Trials: Combining Patient-Centric Care with Scientific Rigor

Psychiatry Clinical Trials: Inclusive, Patient-Centric Approaches for Mental Health Research

UniQure moves Huntington's gene therapy to next phase of key trial

Genetic diagnostics promises paradigm shift towards personalized, proactive healthcare

8 Frequently Asked Questions About Genetic Testing in Clinical Trials

Biology opens up new frontiers in medicine to cure genetic diseases: Kiran Mazumdar Shaw

Gene therapy startup emerges with green light for first-of-its-kind trial

Genetically modified herpes combats advanced cancers

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Indian healthcare looks at pharmacogenomics in patient care as comprehensive genetic tests gain ground

Biogen shelves ALS drug after early-stage trial failure

Verve gets FDA green light to expand base editing trial into US

Eloxx repackages ELX-02 data in cystic fibrosis as it eyes pivotal trial

AHA 2024: Plozasiran demonstrates promising Phase III results in FCS adults

Integrating Bioanalytical Automation, Biomarkers, Pharmacogenomics, & Cost-Effective Strategies

STAT+: Emerging class of genetically targeted drugs induce remissions in leukemia patients

To Share or Not to Share Failed Genetic Screening Results with Patients

Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective

Alkeus raises funds to launch Stargardt disease therapy gildeuretinol

Fulgent Genetics acquires therapeutics development company Fulgent Pharma

This Rare Gene Variant Seems to Protect You From Alzheimer’s Disease

How Pharmacogenomics Can Benefit Your Clinical Trial

Ophthalmologists eye gene therapies for vision-threatening conditions as NGS facilitates precision diagnosis

Alyftrek: Vertex Expands Cystic Fibrosis Treatment with Triple-Action Combo

Maze Therapeutics Introduces IPO with Two CKD Candidates in Clinical Trials

A box of 200 mosquitoes did the vaccinating in this malaria trial. That's not a joke!

BridgeBio’s Attruby (Acoramidis): A New, More Potent TTR Stabilizer for ATTR-CM

FDA Refines Accelerated Approval Pathway with New Draft Guidance

Innovation in Autoimmune and Bone Health Research: Driving Progress for Better Patient Outcomes

eGenesis scoops $191m for genetically modified pig kidney transplant trial

Sarepta’s Gene Therapy Elevidys Under Scrutiny After Patient Death

Reneo cuts 70% of staff following Phase III trial failure

Innovating in Autoimmune and Bone Health Research: Driving Progress for Better Patient Outcomes

Breakthrough: Rare Gene Mutation Offers Clues to Preventing Type 1 Diabetes

Scientists Just Identified Hundreds of Genes That Could Cause Cancer

The Risk of Cancer Fades as We Get Older, And We May Finally Know Wh

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

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