Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

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 205

Gene Genetics Scientist Research 205

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. One diagnostic example that I discussed in my presentation is autism.

Genetics 189

Genetics Trials Clinical Trials Clinical Trials 189

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 290

Gene Therapy Gene Genetics Trials 290

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?

Genetics 202

Genetics Gene Research Clinical Trials 202

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 198

Gene Therapy Gene Genetics Clinical Research 198

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 197

Scientist Gene Genetics Research 197

Bio Pharma Dive

FEBRUARY 12, 2021

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.

Gene Therapy 290

Gene Therapy Gene Genetics Medicine 290

Pharmaceutical Technology

MAY 4, 2023

Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.

Gene Therapy 256

Gene Therapy Gene Development Manufacturing 256

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 192

Gene Genetics Protein Research 192

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

FEBRUARY 13, 2023

While several therapeutics are available for treating symptoms associated with epilepsy, researchers and patients have strongly called out the need for more holistic treatments that would address the condition as a whole. Meanwhile, others are researching the link between gene variation and different responses to treatments.

Research 264

Research Gene Gene Therapy Genome 264

Pharmaceutical Technology

FEBRUARY 15, 2023

In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.

Gene Therapy 246

Gene Therapy Gene Genome Genomics 246

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 179

Genetics Gene Scientist Research 179

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

Pharmaceutical Technology

MAY 25, 2023

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

Gene Therapy 243

Gene Therapy Gene Gene Editing Development 243

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

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.

Medicine 167

Medicine Research Gene Therapy Genetics 167

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 159

Genetic Disease Genetics Gene Editing Medicine 159

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 209

Genetic Engineering Gene Engineer Genetics 209

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

STAT News

FEBRUARY 22, 2023

Moderna is aiming to build a gene editing franchise powered by some of the same technologies used in its COVID-19 vaccines. The Cambridge biotech company announced Wednesday that it will partner with Life Edit Therapeutics to develop potentially permanent treatments for rare genetic diseases and other conditions.

Gene Editing 126

Gene Editing Gene Genetic Disease Research 126

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 190

Gene Expression Gene Genetics Research 190

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? It is important to consider the type of testing performed (e.g.,

Clinical Trials 130

Clinical Trials Clinical Trials Genetics Trials 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

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.

Genetics 114

Genetics Research Genome Genomics 114

Pharmaceutical Technology

JANUARY 10, 2023

Asklepios BioPharmaceutical has entered a research partnership and option agreement with ReCode Therapeutics for exploring its single-vector gene-editing platform. The new solution will enable complete gene insertion by delivering the gene-editing tool and DNA as mixed cargo to desired targets in one LNP.

Gene Editing 130

Gene Editing Gene Gene Therapy Genetics 130

STAT News

FEBRUARY 16, 2023

With hundreds of genes thought to be linked to obesity, the challenge is sifting through them all to determine which ones increase the risk of downstream complications like heart disease and diabetes. In a study published Thursday in Nature Genetics, researchers took the first steps in finding a potential candidate specifically in women.

Gene 98

Gene Research Genetics 98

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

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 144

Gene Editing Gene Gene Therapy In-Vivo 144

Pharmaceutical Technology

MARCH 24, 2023

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.

Genetics 130

Genetics Medicine Development In-Vivo 130

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. These insights were shared through several sessions, in presentation tracks specifically dedicated to “Community and Capacity Building” and “Becoming a Research Ready Organization.”

Gene 130

Gene Gene Editing Gene Therapy Clinical Trials 130

XTalks

SEPTEMBER 30, 2020

While it remains unclear why some people become more gravely ill than others when infected with the SARS-CoV-2 virus that causes COVID-19, researchers have found that an immune deficiency may be part of it. percent) of the critically ill patients harbored rare mutations in eight of the genes involved in interferon production.

Antibody 135

Antibody Genetics Research In-Vitro 135

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. The aim of the ISGCT is to foster and maintain strong scientific communities in this field of research.

Gene 98

Gene Research Genetics Clinical Trials 98

Pharmaceutical Technology

FEBRUARY 23, 2023

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

Gene Editing 147

Gene Editing Gene In-Vivo Gene Therapy 147

Medical Xpress

MAY 16, 2023

However, how the brain has developed over the course of evolution and which genes are responsible for the high cognitive abilities is still largely unclear. The better our understanding of the role of genes in brain development, the more likely it will be that we will be able to develop treatments for serious brain diseases.

Development 115

Development Genetics Gene Research 115

Pharmaceutical Technology

MAY 17, 2023

The deal will see the integration of Scribe’s new CRISPR by Design approach and Prevail’s expertise in developing genetic medicines for neurological disorders for specific genetic targets. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Genetics 130

Genetics Medicine In-Vivo Gene Therapy 130

Pharma Mirror

OCTOBER 26, 2021

a clinical-stage cell and gene therapy company focused on developing and commercializing disease-modifying therapies for patients suffering from rare diseases for which there is a lack of available treatment options, has formed a new research collaboration with Mayo Clinic. Castle Creek Biosciences Inc., Deyle, M.D.,

Gene Therapy 130

Gene Therapy Gene Genetics Clinical Development 130