Scienmag

NOVEMBER 25, 2020

Credit: Professor Jane Farrar and Dr Daniel Maloney, Trinity College Dublin Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a progressive loss of vision and affects thousands of people across the globe.

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FDA Law Blog

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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STAT News

FEBRUARY 7, 2023

Kathy High, a scientist and executive who led the development of the first gene therapy approved in the U.S., has left Bayer subsidiary AskBio two years after joining.

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STAT News

AUGUST 8, 2022

Scientist Jim Wilson is synonymous with gene therapy — and for good reason. His work has generated millions of dollars for his employer, the University of Pennsylvania, and made Wilson a towering figure in the gene therapy world, with the wealth and fame to match.

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Medical Xpress

APRIL 19, 2023

Scientists at Trinity College Dublin today announced a significant development toward a new therapeutic treatment of glaucoma.

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BioPharma Reporter

OCTOBER 22, 2020

Scientists have used gene therapy and a newly developed light-sensing protein to restore sight in mice: with the technology to start clinical trials in the US later this year.

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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. Based on extensive experience, our leading circRNA scientists have invented a clearly differentiated vector system to generate circRNA in target tissues.

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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.

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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. Life Edit is the North Carolina subsidiary of ElevateBio, a cell and gene therapy manufacturing firm in Waltham. Continue to STAT+ to read the full story…

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Medical Xpress

MAY 2, 2023

Scientists at UMass Chan Medical School have developed a technology to deliver gene therapy directly to lung tissue through intranasal administration, a development that could potentially create a new class of treatments for lung disease.

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Pharmaceutical Technology

APRIL 21, 2023

Moderna and IBM scientists will also use an AI foundation model known as MoLFormer, to predict the physical properties of a molecule and understand the characteristics of potential mRNA medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Scienmag

DECEMBER 8, 2020

Findings add safety, efficacy support for gene therapy developed at OHSU A decade after the birth of the first primates born with the aid of a gene therapy technique designed to prevent inherited mitochondrial disease, a careful study of the monkeys and their offspring reveals no adverse health effects.

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BioPharma Reporter

MARCH 22, 2024

Claudia Zylberberg is a scientist and founder, board member and former CEO of Akron Biotech, a company that makes materials and technologies to support advanced therapy development. We spoke to her about her career, personal triumphs and candidacy for President of the International Society for Cell and Gene Therapy (ISCT),

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Medical Xpress

MARCH 18, 2023

University of Utah Health scientists have corrected abnormal heart rhythms in mice by restoring healthy levels of a protein that heart cells need to establish connections with one another.

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Pharmaceutical Technology

DECEMBER 5, 2022

The hub will connect Queensland scientists with the teams at Sanofi’s mRNA Centre of Excellence in the US and France, putting them at the front of international vaccine development and biomedical research. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.

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Scienmag

DECEMBER 9, 2021

Scientists have discovered that gene therapy and the diabetes drug metformin may be potential treatments for late-onset retinal degeneration (L-ORD), a rare, blinding eye disease. The findings are published in Communications Biology.

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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.

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pharmaphorum

SEPTEMBER 26, 2022

The past twelve months have culminated in an unprecedented level of excitement, investment, and clinical progress within the gene therapy field. This year’s agenda includes a novel track designed for quality control and process development groups working in gene therapy. Download the full event guide to find out more.

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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. bluebird bio’s Philip Gregory.

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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).

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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 […].

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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.

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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.

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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.

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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.

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases.

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The Pharma Data

OCTOBER 18, 2020

In late August, Pfizer announced it was investing an additional $500 million into its state-of-the-art gene therapy manufacturing facility in Sanford, North Carolina. Ricci told BioSpace the company has been “investing heavily in gene therapy and in the Raleigh-Durham Research Triangle Park area.”

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Scienmag

OCTOBER 15, 2021

Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. Credit: Courtesy of […].

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Pharmaceutical Technology

MAY 17, 2023

A group of scientists designed BravoAAV and ProntoLVV at AGC Biologics’ site in Milan, Italy. AGC Biologics’ global cell and gene team has so far provided support to four commercial viral vector products, three cell therapies, as well as more than 30 cell and gene therapy studies across the US and Europe.

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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.

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The Pharma Data

SEPTEMBER 21, 2020

. A biotech company spun out of the University of Edinburgh will utilise research to develop new gene therapies to target different types of cancer. The funding will be used to develop a pipeline of ‘highly selective’ gene therapies targeting cancer stem cells in a variety of solid tumour types.

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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 […].

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Scienmag

AUGUST 18, 2020

In Nature Communications, Fred Hutch scientists demonstrate in a mouse model how gene therapy could cure an infection that afflicts billions of people Infectious disease researchers at Fred Hutchinson Cancer Research Center have used a gene editing approach to remove latent herpes simplex virus 1, or HSV-1, also known as oral herpes.

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XTalks

DECEMBER 14, 2022

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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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. As of now, the US Food and Drug Administration (FDA) has approved 22 gene and cell therapy products.

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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) (..)

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STAT News

NOVEMBER 4, 2022

A man with muscular dystrophy who was first in line to receive an experimental gene editing therapy tailor made to treat the cause of his rare form of the disease has died. Terry had long been too old to participate in clinical trials of experimental therapies for the disease, which are often geared towards young boys.

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