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.

CRISPR/Cas technology helped revolutionize diagnostics and gene therapy

Announcing a new article publication for BIO Integration journal. In this mini review article the authors Meiyu Qiu and Pei Li from Korea Advanced Institute of Science and Technology (KAIST), Daejeon, Republic of Korea summarize CRISPR/Cas-based Diagnostics and Gene Therapy.

26 Apr 2021

Genethon announces dosing of first patient at pediatric clinical trial platform for neuromuscular diseases

A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon.

20 Apr 2021

Gene therapy for common forms of congenital blindness shows promise to improve vision

A new gene therapy for one of the most common forms of congenital blindness was safe and improved patients' vision, according to initial data from a clinical trial led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.

19 Apr 2021

Experts to discuss advanced cancer cell and gene therapies at Summit 2021

April 29 marks a pivotal moment in cancer research when the world's brightest minds come together to discuss how to translate the success of CAR T-cell therapies for blood cancers into successful cell and gene therapies for the most complex and deadly solid tumor cancers.

15 Apr 2021

Biology of single-celled organisms reveals potential treatment target for COPD

In a series of experiments that began with amoebas -- single-celled organisms that extend podlike appendages to move around -- Johns Hopkins Medicine scientists say they have identified a genetic pathway that could be activated to help sweep out mucus from the lungs of people with chronic obstructive pulmonary disease a widespread lung ailment.

13 Apr 2021

Gene therapy may offer a new approach to preserving vision in people with retinitis pigmentosa

A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published today in eLife.

13 Apr 2021

Albumedix Expands Production Capacity at its Commercial-Scale Recombinant Human Albumin Manufacturing Plant and Opens New Research Facilities

Albumedix Ltd. ('Albumedix'), a recognized global leader in recombinant human albumin specializing in the enablement of advanced therapies, announces the completed expansion of the company’s commercial-scale manufacturing facility, alongside with the establishment of a new Technology Centre equipped with state-of-the-art laboratories at its UK-based campus in Nottingham.

From Albumedix Ltd. 13 Apr 2021

Toward a transformative therapy for sickle cell patients

For the millions of people worldwide who have sickle cell disease, there are only a few treatment options, which include risky bone marrow transplants, gene therapy or other treatments that address a subset of symptoms.

9 Apr 2021

Rice bioengineer wins NIH support for a new strategy to fight cystic fibrosis

Chemical and biomolecular engineer Xue Sherry Gao of Rice University's Brown School of Engineering has won National Institutes of Health support for a new strategy to fight cystic fibrosis.

8 Apr 2021

Researchers evaluate new drug for spinal muscular atrophy in a broader spectrum of patients

The rarity of spinal muscular atrophy (SMA) means that promising new treatments may be tested in only a limited spectrum of patients before approval. Investigators evaluated a newly approved drug, onasemnogene abeparvovec, in a broader spectrum of patients in order to obtain expanded data on its side effects profile.

6 Apr 2021

New approach to CAR T gene therapy yields efficient and longer-lasting response against HIV

A UCLA research team has shown that using a truncated form of the CD4 molecule as part of a gene therapy to combat HIV yielded superior and longer-lasting results in mouse models than previous similar therapies using the CD4 molecule.

3 Apr 2021

Fight for Sight funds research to explore gene editing as treatment for inherited retinal diseases

Fight for Sight is funding cutting-edge research at UCL, which will explore whether gene editing could be used to repair faults in genes for some inherited retinal diseases.

1 Apr 2021

Gene therapy restores vison in dogs with severe form of Leber congenital amaurosis

Gustavo Aguirre and William Beltran, veterinary ophthalmologists and vision scientists at the University of Pennsylvania School of Veterinary Medicine, have studied a wide range of different retinal blinding disorders.

31 Mar 2021

Scientists launch clinical trial of CRISPR gene correction therapy in patients with sickle cell disease

Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical trial of a CRISPR gene correction therapy in patients with sickle cell disease using the patient's own blood-forming stem cells.

30 Mar 2021

First cell-based gene therapy approved by the FDA for treating multiple myeloma

The U.S. Food and Drug Administration approved Abecma (idecabtagene vicleucel), a cell-based gene therapy to treat adult patients with multiple myeloma who have not responded to, or whose disease has returned after, at least four prior lines (different types) of therapy.

29 Mar 2021

Mogrify wins Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year at CambridgeshireLive Business Excellence Awards 2020

Mogrify has been awarded the Hewitsons Award for Innovation in Business and Price Bailey Award for Business of the Year.

From Mogrify 26 Mar 2021

Novel gene therapy reprograms glioma cells into functional neurons

Glioma is a fatal neurological disorder that has limited interventional treatment, despite extensive research over the past several decades.

24 Mar 2021

First model of early human embryo generated from skin cells

In a discovery that will revolutionise research into the causes of early miscarriage, infertility and the study of early human development - an international team of scientists led by Monash University in Melbourne, Australia has generated a model of a human embryo from skin cells.

22 Mar 2021

Alliance for Cancer Gene Therapy provides new opportunity to develop cancer cell and gene therapies

Alliance for Cancer Gene Therapy wants to change the C-word from Cancer to Cure -- building on the life changing success of CAR-T therapies for blood cancers, the organization is now focused on the urgent need to translate this success into therapies to fight more types of cancer, such as solid tumor cancers.

19 Mar 2021

Novel cancer-fighting gene therapy enters phase I clinical trial

Researchers from the MDC and Charité have developed a new gene therapy based around a special T-cell receptor that helps the immune system effectively recognize and fight cancer cells.

18 Mar 2021