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 168

Gene Editing Gene Gene Therapy In-Vivo 168

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.

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Gene Therapy Gene Genome Genomics 246

STAT News

OCTOBER 3, 2022

A Swedish scientist won the 2022 Nobel Prize in medicine or physiology on Monday for his groundbreaking research into the evolutionary history of humankind. Pääbo unlocked scientists’ understanding of how genes from these extinct relatives have been passed down to present-day humans.

Medicine 142

Medicine Contamination Research DNA 142

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.

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Gene Therapy Gene Gene Editing Manufacturing 243

Drug Discovery World

FEBRUARY 16, 2023

Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.

Genetics 52

Genetics Medicine Gene Editing In-Vivo 52

Drug Discovery World

OCTOBER 5, 2023

A research team at the Francis Crick Institute and Great Ormond Street Hospital (GOSH)/UCL Great Ormond Street Institute of Child Health have identified potential treatments for children with rare genetic conditions. The researchers have shown that problems with calcium underlie these progressive diseases.

Genetics 59

Genetics Research Gene Doctors 59

Scienmag

SEPTEMBER 29, 2021

Describing a previously unknown genetic condition that affects children, researchers at University of California San Diego School of Medicine and Rady Children’s Institute for Genomic Medicine say they also found a potential method to prevent the gene mutation by administering a drug during pregnancy.

Genetics 84

Genetics Research Genome Genomics 84

Drug Discovery World

AUGUST 7, 2023

Evox Therapeutics has agreed a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn Mount Sinai) in New York, NY, US. The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease.

Gene Therapy 52

Gene Therapy Gene Research In-Vivo 52

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.

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Gene Therapy Gene Development Engineer 237

BioSpace

JUNE 23, 2022

The Boston University School of Medicine has unraveled details about a known genetic component for the development of Alzheimer’s disease (AD): the APOE4 gene.

Genetics 88

Genetics Gene Research Medicine 88

Medical Xpress

APRIL 11, 2023

University of Virginia School of Medicine researchers have identified a gene that plays a crucial role in determining the risk for heart attacks, deadly aneurysms, coronary artery disease and other dangerous vascular conditions.

Gene 93

Gene Research Medicine Genetics 93

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 278

Gene Therapy Gene Genetics Medicine 278

Drug Discovery World

MARCH 20, 2023

Researchers have identified two previously unknown genes linked to schizophrenia and newly implicated a third gene as carrying risk for both schizophrenia and autism. The investigators found the two risk genes, SRRM2 and AKAP11, by comparing the gene sequences of people with schizophrenia to those of healthy controls.

Gene 52

Gene Research Genetics Gene Sequencing 52

Drug Discovery World

APRIL 9, 2024

A study by researchers at the UCLA Health Jonsson Comprehensive Cancer Center in the US suggests that injecting engineered dendritic cells directly into cancerous lung tumours can help promote a stronger immune response. Additionally, they observed that this therapy helped to establish a long-lasting immune response against the cancer.

Genetic Engineering 52

Genetic Engineering Engineer Genetics Immune Response 52

XTalks

MARCH 28, 2024

Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

Scienmag

MAY 6, 2021

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Jude Children’s Research Hospital St. Credit: St.

Gene Expression 97

Gene Expression Regulation Gene DNA 97

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

DECEMBER 21, 2022

Using the CRISPR-Cas9 gene editing system, UT Southwestern researchers corrected mutations responsible for a common inherited heart condition called dilated cardiomyopathy (DCM) in human cells and a mouse model of the disease.

Gene Therapy 98

Gene Therapy Gene Editing Gene Research 98

STAT News

DECEMBER 13, 2022

Medicines that reshape or tamp down immune responses may be life-changing for patients with cancer and autoimmune disorders, but in some cases they can awaken a dormant virus and unleash a deadly brain disease. A new study suggests that the root of the problem is buried in our genetic code.

Genetics 105

Genetics Immune Response Gene Drugs 105

Worldwide Clinical Trials

MARCH 20, 2024

In the increasingly advancing world of medicine, personalized care is a key factor that can progress your drug development efforts. 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

Scienmag

NOVEMBER 3, 2020

Credit: Encarnacion/UConn Photo The advent of genome science has given researchers an unprecedented ability to understand the root causes of a host of conditions.

Genetics 71

Genetics Genome Genomics Research 71

Scienmag

JUNE 20, 2021

Transport system of essential materials in brain cells disrupted in certain genetic developmental disorders. Credit: Dr. Riazuddin University of Maryland School of Medicine (UMSOM) researchers identified a new gene that may be linked to certain neurodevelopmental disorders and intellectual disabilities.

Genetics 66

Genetics Gene Medicine Research 66

pharmaphorum

JANUARY 24, 2023

Ben Hargreaves finds that the vast amount of genetic data that exists today could help provide a faster, more targeted way of developing new drug candidates. The logical extension to this kind of approach is treating individual patients, with their individual genetic makeup.

Genetics 81

Genetics Drugs Genome Project Biobanking 81

Pharmaceutical Technology

JUNE 16, 2023

The Abu Dhabi Department of Health (DoH) in the UAE has made a declaration of collaboration with Mass General Brigham’s (MGB) International Center for Genetic Disease (iCGD) to advance life sciences.

Life Science 130

Life Science Genetic Disease Genome Genomics 130

Medical Xpress

DECEMBER 14, 2022

Researchers at the University of Miami Miller School of Medicine, McGill University and other institutions have found that a well-concealed genetic variation in the gene FGF14, called a DNA tandem repeat expansion, causes a common form of late-onset cerebellar ataxia, a brain disorder that interferes with coordinated movement.

Genetics 75

Genetics DNA Gene Medicine 75

World of DTC Marketing

JUNE 5, 2021

Obesity is a multifactorial disease in which environmental conditions and several genes play an important role in developing this disease. Research published by the World Health Organization found that a rise in fast food sales correlated to a rise in body mass index, and Americans are notorious for their fast-food consumption ?

Medicine 193

Medicine Gene Genetics Research 193

Medical Xpress

MAY 16, 2023

That was the case when Thilini Gamage was to carry out one of the studies in her doctoral work with Professor Eirik Frengen at the Institute of Clinical Medicine, University of Oslo. They study gene variation and mutations that cause rare genetic diseases. A mutation is a permanent change in the genetic material.

In-Vitro 98

In-Vitro In-Vivo Genetic Disease Genetics 98

XTalks

JANUARY 26, 2024

Technological advancements are revolutionizing cancer research and therapies. Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. Clinical trials have demonstrated the potential of CRISPR/Cas9 in this field.

Research 118

Research Gene Editing Gene Genetics 118

Medical Xpress

DECEMBER 7, 2022

Penn State College of Medicine researchers have co-led a large genetic study that identified more than 2,300 genes predicting alcohol and tobacco use after analyzing data from more than 3.4 They found that a majority of these genes were similar among people with European, African, American and Asian ancestries.

Gene 75

Gene Genetics Medicine Research 75

Medical Xpress

APRIL 17, 2023

Genes are full of clues about a person's health. A recent UC Davis Health study published in BMC Medicine suggests that this may be possible, thanks to time-sensitive gene analysis that allows for faster stroke diagnosis and treatment. They might also show the way for stroke recovery.

Gene 75

Gene Research Medicine Genetics 75

The Pharma Data

FEBRUARY 22, 2022

State-of-the-art facility will headquarter research efforts in Boston and New York to accelerate the development of RNA and DNA-based medicines. Adams, vice president of genetic medicine at Lilly and co-director of the Institute. ” The Institute will be headquartered in 334,000 sq.

Genetics 52

Genetics Medicine RNA Scientist 52

Medical Xpress

MAY 9, 2023

Researchers at Baylor College of Medicine have linked specific variants or mutations of the gene myocardin-related transcription factor B (MRTFB) with a novel neurodevelopmental disorder.

Gene 97

Gene Medicine Research Genetics 97

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

Scienmag

FEBRUARY 10, 2021

Data demonstrates new increased diversity in genetic studies and provides new insights into population-specific diseases Researchers at the University of Maryland School of Medicine (UMSOM) and their colleagues published a new analysis today in the journal Nature from genetic sequencing data of more than 53,000 individuals, primarily from minority (..)

Genetics 71

Genetics Research Medicine Bioinformatics 71

Medical Xpress

MARCH 21, 2023

An international study led by the Molecular Physiology Laboratory at the UPF Department of Medicine and Life Sciences (MELIS) identifies new genes that modulate the toxicity of the protein β-amyloid, responsible for causing Alzheimer's disease.

Protein 98

Protein Gene Life Science Research 98

Scienmag

NOVEMBER 5, 2021

It is essential for cells to control precisely which of the many genes of their genetic material they use. Researchers of Karlsruhe Institute of Technology (KIT), Friedrich-Alexander-Universität Erlangen-Nuremberg (FAU), and Max Planck Center for Physics and Medicine (MPZPM) have now found that the […].

Gene 82

Gene Genetics Medicine Research 82

Medical Xpress

DECEMBER 12, 2022

An international research team led by Dr. Ana Guadaño at the Alberto Sols Biomedical Research Institute (IIBM, a combined CSIC-UAM center) and involving the Complutense University of Madrid (UCM), used CRISPR gene editing techniques to incorporate into mice a mutation of the MCT8 protein responsible for transporting thyroid hormones to the interior (..)

Gene Editing 75

Gene Editing Genetics Hormones Medicine 75