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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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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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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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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Pharmaceutical Technology

JANUARY 26, 2023

Cell and gene therapies are predicted to be the future of medical treatment by providing the body with the means to repair itself and recover from a range of serious conditions and severe diseases. Often, cell and gene therapies are spoken of as miracle treatments, yet this progress has hardly occurred overnight.

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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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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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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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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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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. Shares in bluebird ticked up following the announcement.

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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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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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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.

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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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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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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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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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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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STAT News

AUGUST 11, 2022

STAT’s Jason Mast joins us to discuss Jim Wilson, a pioneering scientist synonymous with the rise of gene therapy who staffers say presided over a toxic, abusive workplace. We cover all that and more this week on “The Readout LOUD,” STAT’s biotech podcast.

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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

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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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

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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Worldwide Clinical Trials

JANUARY 24, 2024

There are many treatments in the pipeline for genetic diseases, including several cell and gene therapies that present significant treatment potential. As of July 2023, the American Society of Cell + Gene Therapy stated there were 3,905 therapies in development.

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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

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Pharmaceutical Technology

SEPTEMBER 29, 2022

Sanofi Research global head and chief scientific officer Frank Nestle said: “At Sanofi, we are pushing the boundaries of science by developing a diverse range of next-generation therapies based on natural killer (NK) cells, which could have broad applications across solid tumours and blood cancers.

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Scienmag

NOVEMBER 5, 2020

Scientists have used gene therapy to regenerate damaged nerve fibres in the eye, in a discovery that could aid the development of new treatments for glaucoma, one of the leading causes of blindness worldwide.

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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.

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BioSpace

MAY 27, 2021

To develop and commercialize them with maximum efficiency, the FDA, scientists and developers must innovate, according to panelists speaking during the Presidential Plenary on Commercialization at the International Society of Cell & Gene Therapy on May 26.

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The Pharma Data

MAY 26, 2023

Catalent’s expert workforce of approximately 18,000 includes more than 3,000 scientists and technicians. Its flexible manufacturing platforms at over 50 global sites supply around 80 billion doses of nearly 8,000 products annually. Source link: [link]

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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.

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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 […].

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