Scienmag

OCTOBER 26, 2020

New genetic sensors, developed by scientists at University of Warwick and Keele University, could function as a lab test device and even as a live monitoring system inside living cells The innovative system can detect when a specific gene in a cell is active – instead of only detecting its presence The technology is based […].

Genetics 75

Genetics Scientist Gene Development 75

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 98

Genetics Drugs Genome Project Biobanking 98

Scienmag

MAY 31, 2021

NIH- and USU- led study links ALS to a fat manufacturing gene and maps out a genetic therapy Credit: Courtesy of the NIH/NINDS.

Genetics 56

Genetics Scientist Gene Manufacturing 56

Scienmag

JUNE 9, 2021

Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

Gene Therapy 53

Gene Therapy Gene Scientist Medicine 53

Scienmag

JUNE 29, 2021

Scientists and doctors at University College London Great Ormond Street Institute of Child Health (UCL GOS ICH) and Great Ormond Street Hospital (GOSH) have given hope of a gene therapy cure to children with a rare degenerative brain disorder called Dopamine Transporter Deficiency Syndrome (DTDS).

Gene Therapy 58

Gene Therapy Gene Doctor Doctors 58

pharmaphorum

MARCH 10, 2021

bluebird bio is to ask regulators to restart clinical studies of its LentiGlobin for sickle cell disease, after an investigation concluded that a case of acute myeloid leukaemia (AML) was “very unlikely” to be caused by the gene therapy. Shares in bluebird ticked up following the announcement.

Gene Therapy 59

Gene Therapy Gene Trials Gene Expression 59

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

Gene Therapy 52

Gene Therapy Gene Manufacturing Production 52

Pharmaceutical Technology

APRIL 5, 2023

But while companies continue studying allogeneic CAR-T therapies, including for their coveted use in solid tumours, such advancements remain challenging. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. AZ: Cell and gene therapies often come with a high price.

Gene Therapy 147

Gene Therapy Gene Engineer Production 147

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

Gene Therapy 52

Gene Therapy Gene Production Clinical Trials 52

XTalks

NOVEMBER 3, 2023

After spending almost an entire day deliberating the safety of Vertex Pharmaceuticals’ and CRISPR Therapeutics’ CRISPR-based gene therapy exa-cel for sickle cell disease, a US Food and Drug Administration (FDA) advisory panel appears to be satisfied with what it saw. CRISPR works as genetic scissors to edit parts of the genome.

Gene Therapy 59

Gene Therapy Gene Gene Editing In-Vivo 59

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. Credit: St. Jude Children’s Research Hospital St.

Gene Expression 97

Gene Expression Regulation Gene DNA 97

Pharmaceutical Technology

SEPTEMBER 29, 2022

Scribe Therapeutics and Sanofi have signed a strategic partnership to expedite the development of breakthrough clustered regularly interspaced short palindromic repeats (CRISPR)-based cell therapies for cancer. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

Development 130

Development In-Vivo Genome Genomics 130

Scienmag

MARCH 11, 2021

The CRISPR-based gene editor, C-to-G Base Editor (CGBE), opens up treatment avenues for up to 40 per cent of genetic disorders caused by single-nucleotide mutations Credit: Agency for Science, Technology and Research (A*STAR), Genome Institute of Singapore (GIS) A team of researchers from the Agency for Science, Technology and Research’s (A*STAR) (..)

Gene 74

Gene Scientist Genome Genomics 74

The Pharma Data

FEBRUARY 22, 2022

Eli Lilly and Company (NYSE: LLY) today announced the launch of the Lilly Institute for Genetic Medicine and an investment of approximately $700 million to establish a state-of-the-art facility at a new site in the Boston Seaport. Adams, vice president of genetic medicine at Lilly and co-director of the Institute.

Genetics 52

Genetics Medicine RNA Scientist 52

Scienmag

FEBRUARY 11, 2021

Study in The CRISPR Journal reports the app advances CRISPR gene therapy R&D; 18-year-old software savant helped lead the app’s development Wilmington, DE, Feb. The app reveals potentially risky DNA […].

Gene 73

Gene Gene Therapy DNA Scientist 73

Delveinsight

DECEMBER 17, 2020

Following that hypothesis, scientists designed oral inhibitors, which could target mitochondrial DNA (mtDNA). Atsena raises USD 55 Million financings for blindness gene therapy. Luxturna has been authorized for LCA caused by mutations in the RPE65 gene, and another inherited retinal disease called Retinitis Pigmentosa.

Gene Therapy 96

Gene Therapy Gene DNA Genetics 96

Delveinsight

FEBRUARY 11, 2021

The scientists analyzed samples from patients with the most common pancreatic cancer, pancreatic ductal adenocarcinoma (PDAC). Avrobio gene therapy eradicates toxic substrate in Fabry patient. Median overall survival in those patients was 5.8 months, whereas patients without inflammation before treatment survived on average 12.3

Gene Therapy 52

Gene Therapy Gene Immune Response In-Vivo 52

Scienmag

MAY 28, 2021

Genetics toolkit targets less researched Culex mosquitoes, which transmit West Nile virus and avian malaria Credit: Gantz Lab, UC San Diego Since the onset of the CRISPR genetic editing revolution, scientists have been working to leverage the technology in the development of gene drives that target pathogen-spreading mosquitoes such as Anopheles and (..)

Containment 69

Containment Genetics Research Gene 69

Scienmag

FEBRUARY 23, 2021

CRISPR gene editing has transformed research, but it is not perfect, and can sometimes target unintended genes; to watch CRISPR enzymes respond to different genes, Leipzig University researchers developed a new method using DNA origami and were able to me Credit: Image courtesy of Julene Madariaga Marcos.

DNA 56

DNA Gene Gene Editing Scientist 56

XTalks

JANUARY 26, 2024

This technology, which allows for precise editing of DNA at specific locations, has been a major focus in the field due to its potential to directly target and modify cancer-causing genes. By editing these genes, researchers can effectively neutralize their cancer-promoting effects.

Research 119

Research Gene Editing Gene Genetics 119

XTalks

DECEMBER 14, 2022

Developed by MIT researchers Jonathan Gootenberg and Omar Abudayyeh, PASTE (Programmable Addition via Site-specific Targeting Elements) gene editing technology can insert genes as long as 36,000 DNA base pairs to liver cells in mice as well as several types of human cells.

Gene Editing 98

Gene Editing DNA Gene Gene Sequencing 98

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

Scienmag

MARCH 4, 2021

Findings explain how plants use RNA to defend against fungal invaders Credit: Nicole Ward Gauthier/University of Kentucky New research reveals an essential step in scientists’ quest to create targeted, more eco-friendly fungicides that protect food crops. However, their pivotal roles […].

Protein 95

Protein RNA Scientist Manufacturing 95

Scienmag

APRIL 12, 2021

Credit: CNIO Glioblastomas are the most common and most aggressive brain tumours. Their survival rate has barely increased over the last 50 years, indicating an urgent need to develop new therapeutic strategies.

Scientist 70

Scientist Development Research Gene Therapy 70

XTalks

SEPTEMBER 27, 2023

Poseida is a clinical-stage biopharmaceutical firm that utilizes its unique non-viral gene engineering methods to develop innovative cell and gene therapies. Scientists working at Poseida Therapeutics.

Life Science 59

Life Science Genetic Engineering Engineer Gene Therapy 59

pharmaphorum

JULY 20, 2022

DSV’s involvement will provide pre-seed capital for the new ventures, which will be incubated within Cancer Research Horizon’s wet labs with support from scientists among its researcher network. billion ($2.75 billion) since it was formed in the early 2000s.

Research 84

Research Bioinformatics Gene Therapy Medicine 84

pharmaphorum

MAY 18, 2022

At UCB, we are trying to understand the whole spectrum of triggers causing epileptic seizures, from genetic factors, brain injury to stroke, cancer or other traumas,” Charl states. Charl believes we are witnessing the ‘decade of the brain’ with significant leaps in scientific understanding of disease pathways and genetic correlations.

Gene Therapy 92

Gene Therapy Gene Genetics Development 92

Delveinsight

AUGUST 20, 2020

FDA rejects BioMarin’s gene therapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A gene therapy valoctocogene roxaparvovec. Now, the scientists at the Salk Institute have found a potential solution to those problems.

Gene Therapy 52

Gene Therapy Research Gene Antibody 52

XTalks

AUGUST 7, 2024

She began her career as a research scientist, working for more than eight years in this role before transitioning into product management and marketing. In this episode, Ayesha spoke with Lawreen Asuncion, a patient advocate who works to raise awareness for the rare disease Usher syndrome.

Life Science 52

Life Science Scientist Production Research 52

Scienmag

OCTOBER 13, 2020

A new study summarizes over 30 years of clinical experience in the treatment and management of glutaric acidemia type 1 (GA1) A new study summarizes over 30 years of clinical experience in the treatment and management of glutaric acidemia type 1 (GA1), a rare and potentially devastating metabolic disorder caused by variants in the GCDH […].

Development 55

Development Gene Therapy Gene Scientist 55

Pharmaceutical Technology

FEBRUARY 3, 2023

With scientists fervently developing mRNA vaccines, nucleic acid therapeutics, and viral vector-based gene therapies, clinicians are set to have a growing number of tools available to treat a wide range of conditions, from infectious diseases to genetic disorders and more.

Genome 244

Genome Genomics Medicine Gene Therapy 244

Pharmaceutical Technology

APRIL 20, 2023

Circio aims to develop new circRNA medicines initially for cancer, then plans to expand rapidly into vaccines and gene therapy. The circVec platform is a modular genetic cassette that provides instructions for the generation of multifunctional circRNAs. Topic sponsors are not involved in the creation of editorial content.

Gene Therapy 240

Gene Therapy RNA Gene Development 240

Delveinsight

DECEMBER 3, 2020

Janssen buys a gene therapy asset. Janssen buys a new gene therapy aimed at reversing a severe disease, from Hemera Biosciences. There are presently no medical treatments, which can regenerate retinal cells that have atrophied, though gene therapies and stem cell therapies are the leading R&D hopes in this arena.

Gene Therapy 52

Gene Therapy Gene Antibody Research 52

STAT News

FEBRUARY 22, 2023

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. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham.

Gene Editing 126

Gene Editing Gene Genetic Disease Research 126

XTalks

MAY 4, 2021

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.

DNA 98

DNA Gene Gene Silencing Genome 98

STAT News

NOVEMBER 4, 2022

Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys. The treatment used a new version of CRISPR to turn on a backup copy of the dystrophin gene that mysteriously lies dormant in our muscles. Follow him on Twitter  @RLCscienceboss.

Gene Editing 105

Gene Editing Gene Gene Therapy Scientist 105

Scienmag

JULY 14, 2021

Researchers have found a way to enhance radiation therapy using novel iodine nanoparticles Credit: Mindy Takamiya/Kyoto University iCeMS Cancer cell death is triggered within three days when X-rays are shone onto tumor tissue containing iodine-carrying nanoparticles. The findings, by scientists at Kyoto […].

DNA 97

DNA Containment Scientist Research 97