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.

Vector BioPharma and iBET collaborate to develop high-throughput analytical methods and scalable processes for gene delivery

Collaboration set to develop and implement high-throughput analytical methods in support of Vector BioPharma’s CMC (chemistry, manufacturing and controls) development.

From Vector BioPharma 10 Jan 2023

Clinically relevant biomarker strategies in drug development

In this interview, NewsMedical speaks with Dr. Justin Devine and Dr. Nicholas Woudberg about biomarkers, their importance for drug development as well as the potential they have when it comes to machine learning.

From Synexa Life Sciences 9 Jan 2023

SynGAP Research Fund awards $180,000 grant to the University of Edinburgh Medical School's Patrick Wild Centre & Centre for Discovery Brain Sciences

SynGAP Research Fund (SRF), a 501(c)(3) public charity whose mission is to improve the quality of life for SYNGAP1 patients through the research and development of treatments, therapies and support systems, along with SynGAP Research Fund UK, today announced they have awarded a $180,000 grant to the University of Edinburgh Medical School's Patrick Wild Centre & Centre for Discovery Brain Sciences to advance the science around gene transfer to correct SYNGAP1 haploinsufficiency.

5 Jan 2023

'Stem cell plasters' could revolutionize treatment for children with congenital heart disease

Researchers at the University of Bristol funded by the British Heart Foundation (BHF) have developed 'stem cell plasters' to revolutionize the way surgeons treat children living with congenital heart disease, so they don't need as many open-heart operations.

20 Dec 2022

Many genetic disorders respond well to specialized gene therapy

When the normal DNA sequence in an individual changes or mutates it can cause disease. The mutation may involve multiple genes and may be caused by environmental factors, or there may be chromosomal defects that are harmful at birth, in infancy or environment at a later stage.

12 Dec 2022

GOSH delivers world-first base-edited cell therapy for a patient with relapsed T cell leukemia

A patient with relapsed T cell leukemia has been given base-edited T-cells in a world-first use of a base-edited cell therapy, in a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).

12 Dec 2022

Researchers seek to reduce Chikungunya virus-caused joint pain with new drug

Oregon Health & Science University researchers and partners are developing what could become the first-ever treatment against the debilitating joint pain that can last months or years after becoming infected with the emerging Chikungunya virus.

9 Dec 2022

Study looks at the possibility of promoting hair cell regeneration using cell reprograming

About 430 million people around the world experience disabling hearing loss. In the United States, approximately 37.5 million adults report some trouble hearing. Hearing loss can happen when any part of the ear or the nerves that carry information on sounds to the brain do not work in the usual way.

8 Dec 2022

Liver cancer study could help improve the safety of certain gene therapies for hemophilia

Research led by Randal J. Kaufman, Ph.D., has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases.

7 Dec 2022

Shenandoah Biotechnology introduces expanded range of CTGrade recombinant proteins manufactured following cGMP practices

Shenandoah Biotechnology Inc., a FUJIFILM Irvine Scientific company and leading manufacturer of recombinant proteins, today announced it has expanded its CTGrade portfolio of cytokines and growth factors for cell and gene therapies manufactured following cGMP guidelines.

From FUJIFILM Irvine Scientific 7 Dec 2022

Using cationic nanonmaterials to target visceral adiposity

Researchers have long been working on how to treat obesity, a serious condition that can lead to hypertension, diabetes, chronic inflammation, and cardiovascular diseases.

1 Dec 2022

New CRISPR-based tool can snip out faulty genes and replace them with new ones

Building on the CRISPR gene-editing system, MIT researchers have designed a new tool that can snip out faulty genes and replace them with new ones, in a safer and more efficient way.

24 Nov 2022

Sartorius BIA Separations and Exopharm Sign Joint Research Agreement to Develop Integrated Technology

Sartorius BIA Separations (BIA), a leading bio-chromatography company, now part of the international life science group Sartorius, and Exopharm Ltd. (ASX:EX1), a company at the forefront of transformative medicines using exosomes, or extracellular vesicles (EVs), today announced they had entered into a formal collaboration.

From Sartorius 23 Nov 2022

New family of adeno-associated viral vectors found to be better at delivering genetic cargo

Gene therapies can treat, even potentially cure, certain genetic diseases, but it is challenging to deliver the treatments to the parts of the body where they are needed.

22 Nov 2022

Creating New Targeted Antibacterial Products to Help Tackle Antimicrobial Resistance

We speak to Matthew Dunne, Director for Drug Discovery at Micreos Pharmaceuticals, about the importance of creating new targeted antibacterial products.

22 Nov 2022

Rice bioengineer receives support for brain cancer research

Kevin McHugh, an assistant professor of bioengineering at Rice University's George R. Brown School of Engineering, has earned a Distinguished Scientist Award for 2022 from The Sontag Foundation, which helps launch the careers of scientists expected to make a significant impact in the fight against brain cancer.

21 Nov 2022

Scientists explore links between survival motor neuron protein and sarcopenia

Adopting some of the strategies behind successfully treating the childhood disease spinal muscular atrophy may enable development of therapies to curb the muscle decline that accompanies aging, new research suggests.

15 Nov 2022

IVI and Lemonex sign MOU to join forces on vaccine development

The International Vaccine Institute announced on November 10 it signed a memorandum of understanding (MOU) with Lemonex, a company specializing in RNA gene therapy development, and agreed to seek cooperation in research and development.

10 Nov 2022

Tissue nanotransfection shows promise as a treatment for traumatic muscle loss

Technology developed by researchers at the Indiana University School of Medicine that can change skin tissue into blood vessels and nerve cells has also shown promise as a treatment for traumatic muscle loss.

9 Nov 2022

What is the role of adenoviruses in human health?

With advances in molecular biology informing recent developments in vaccinology, gene therapy, and other fields of medicine, human adenoviruses (HAdVs) have been of primary importance in medical research. A recent review looks at the promises of HAdVs, their potential dangers, and future challenges.