Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

MIP Discovery closes £7M Series A financing to drive commercialization in cell and gene therapy space

Company’s synthetic affinity reagents are antibody alternatives designed to advance the speed, safety, and efficiency of manufacturing at scale, making advanced therapies more accessible to patients.

From MIP Discovery 20 Feb 2024

Researchers develop novel nanomedicine loaded with genetic material to fight HIV

Society learned about the value of mRNA during the COVID-19 pandemic when we saw scientists and medical professionals harness its power to deliver a vaccine for the virus within a year.

20 Feb 2024

City of Hope cures oldest person of blood cancer and achieves HIV remission

City of Hope, one of the largest cancer research and treatment organizations in the United States, treated the oldest person to be cured of a blood cancer and then achieve remission for HIV after receiving a blood stem cell transplant from a donor with a rare genetic mutation.

15 Feb 2024

Gene therapy reverses advanced heart failure in animal study

Heart failure remains the leading cause of mortality in the U.S. During a heart attack blood stops flowing into the heart.

13 Feb 2024

Gene editing gets a boost with new CRISPR-COPIES

CRISPR/Cas systems have undergone tremendous advancement in the past decade. These precise genome editing tools have applications ranging from transgenic crop development to gene therapy and beyond.

12 Feb 2024

GoFundMe has become a health care utility

GoFundMe started as a crowdfunding site for underwriting "ideas and dreams," and, as GoFundMe's co-founders, Andrew Ballester and Brad Damphousse, once put it, "for life's important moments."

12 Feb 2024

Never-before-documented genetic disorder causes rare lung disease

The macrophage is one of the body's most important inhabitants. Meaning "big eater" in Greek, this immune cell consumes and digests problematic elements from microbes and cancer cells to dust and debris.

8 Feb 2024

First gene therapy trial aims to restore hearing in children

The aim of this clinical trial, which has just received approval in France, is to assess the safety and efficacy of a new gene therapy drug in children aged between 6 and 31 months with profound hearing loss.

5 Feb 2024

Revvity showcases cutting-edge solutions at SLAS2024

Revvity presents its continued commitment to expanding the boundaries of human potential through science.

From Revvity 5 Feb 2024

Breakthrough recombinase technology ushers in precise, adaptable gene editing

A team of researchers at the Carl Gustav Carus Faculty of Medicine, TUD Dresden University of Technology, led by Prof. Frank Buchholz, has achieved a major breakthrough in genome editing technology.

1 Feb 2024

Gene therapy could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy

A new study in mice shows that replacement of a dysfunctional gene could prolong survival in some people with arrhythmogenic right ventricular cardiomyopathy (ARVC), a rare inherited disorder in which the muscular walls of the heart progressively weaken and put patients at risk of dangerous irregular heartbeats.

30 Jan 2024

Asimov launches LV Edge Packaging System to optimize lentivirus production

New off-the-shelf system reduces the cost and risk of lentiviral (LV) production for cell and gene therapy developers.

From Asimov 29 Jan 2024

New study of daylight vision using canine model offers critical insight into restoring cone function

In the retinas of human eyes, the cones are photoreceptor cells responsible for color vision, daylight vision, and the perception of small details.

27 Jan 2024

Prime editing takes gene therapy to the next level

This study is led by Prof. Xianqun Fan (Department of Ophthalmology, Shanghai Jiao Tong University School of Medicine Affiliated Ninth People's Hospital).

27 Jan 2024

Study shows effectiveness of gene therapy in treating congenital deafness

A new study co-led by investigators from Mass Eye and Ear, a member of Mass General Brigham, demonstrated the effectiveness of a gene therapy towards restoring hearing function for children suffering from hereditary deafness.

24 Jan 2024

Core Biogenesis and Nucleus Biologics announce strategic collaboration for manufacturing and distribution of cost effective and sustainable recombinant proteins

In a joint effort to provide lower cost and more defined reagents and media to the cell and gene therapy market, Nucleus Biologics, The Cell Performance Company™, a leading provider of custom cell culture media solutions for the cell and gene therapy industry, has signed a manufacturing, distribution, and collaboration agreement with Core Biogenesis, an innovator in the field of recombinant human proteins expressed in plants.

From Nucleus Biologics 24 Jan 2024

3D nanomatrix vascular sheet model for high-throughput atherosclerosis drug testing

A groundbreaking 3D, three-layer nanomatrix vascular sheet that possesses multiple features of atherosclerosis has been applied for developing a high-throughput functional assay of drug candidates to treat this disease, University of Alabama at Birmingham researchers report in the journal Biomaterials.

12 Jan 2024

mRNA therapy shows promise for curing children's rare liver disease

By exploiting the technology used in Covid-19 vaccines, a team led by UCL, King's College London and Moderna scientists has created an effective therapy for a rare disease, in a study in mice, demonstrating the technology's potential therapeutic use in people.

10 Jan 2024

Marking a vital milestone for Vertex and Charles River

Charles River’s Memphis facility approved to manufacture Vertex’s CASGEVY™ therapy, the world’s first gene-edited therapy targeting severe sickle cell disease

From Charles River 9 Jan 2024

Cellular Origins, a TTP Company, establishes Advisory Board of global industry leaders

Renowned experts from across the CGT and robotics industries, Professor John Campbell, Dr Matthew Li, Dr Paul Roberts, and Geoffrey Hodge appointed to provide strategic and technical perspectives.

From Cellular Origins 9 Jan 2024