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.

Innovative mixed reality training platform to be developed for pharmaceutical companies and healthcare

UK immersive technology company FourPlus, along with collaborators Holosphere and the Cell and Gene Therapy Catapult, have been awarded funding from Innovate UK to create and test a mixed reality training platform that enables customised training delivery for pharmaceutical companies and healthcare.

17 Jul 2023

Stable nanoparticles train the immune system to fight cancer

Over the past decade, researchers have sought more effective and enduring cancer treatments. Among the wide variety of immunotherapies, Stimulator of Interfron Genes activation (STING agonism) has emerged as a particularly promising approach that harnesses a patient’s immune system to combat tumors throughout the body.

17 Jul 2023

Researchers develop a chemical approach to reverse aging

In a groundbreaking study, researchers have unlocked a new frontier in the fight against aging and age-related diseases.

12 Jul 2023

Whole genome sequencing shows promise for early diagnosis of genetic disorders in newborns, infants

A new national study, led by researchers at Tufts Medical Center in Boston, has found whole genome sequencing (WGS) to be nearly twice as effective as a targeted gene sequencing test at identifying abnormalities responsible for genetic disorders in newborns and infants.

11 Jul 2023

Imperial spinout NK:IO awarded £1.6M Innovate UK New Cancer Therapeutics grant for development of pioneering NK cell therapy

NK:IO, a leader in NK cell biology applied to the development of exceptionally potent, off-the-shelf cell therapies targeting solid tumors, today announced it has been awarded £1.6M in grant funding from Innovate UK’s New Cancer Therapeutics program.

From NK:IO Limited 11 Jul 2023

New DNA repair test could revolutionize treatment for genetic disorders and cancers

A personalised test that tells how well chemotherapy, radiotherapy or gene therapy will work on individual patients is now approved for UK, US, EU and Japanese patents.

10 Jul 2023

Using a vaccine to boost the response of engineered T cells

Engineering T cells to destroy cancer cells has shown success in treating some types of cancer, such as leukemia and lymphoma. However, it hasn't worked as well for solid tumors.

5 Jul 2023

Base editing shows promise for treating sickle cell disease and beta thalassemia

Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia.

3 Jul 2023

SPG-15: A Mother's Fight for a Gene Therapy Cure, Maddi's Story

News Medical spoke to the CEO and founder of the Maddi Foundation to gain a patient perspective on how important research in gene therapy is.

3 Jul 2023

AI may hold the key to a sharper and more precise DNA scissor technology

Artificial Intelligence may be the key to more precise and effective gene therapy treatments. A new study from Aarhus University has found, that applying AI predictions of protein structures enhances the DNA scissor technology CRISPR, by making the cuts in a patient's DNA more precise.

29 Jun 2023

Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult

Albumedix Ltd., an enabler of advanced therapies and the world leader in recombinant human albumin (rAlb), announced today that they have entered into a collaboration agreement with Cell and Gene Therapy Catapult (CGT Catapult) to investigate the use of Albumedix´ proprietary albumin-based solutions for advanced therapy applications, including viral vectors manufacturing.

From Albumedix Ltd. 29 Jun 2023

Novel method to deliver drugs into inner ear restores hearing in deaf mice

An international team of researchers has developed a new method to deliver drugs into the inner ear. The discovery was possible by harnessing the natural flow of fluids in the brain and employing a little understood backdoor into the cochlea.

28 Jun 2023

Cell and gene therapy CDMO Andelyn Biosciences celebrates over 20 years of leadership and innovation with opening of new 180,000 square foot manufacturing headquarters

Andelyn Biosciences, a pioneer in the development and manufacturing of cell and gene therapies, today formally inaugurates its new manufacturing headquarters, the Andelyn Corporate Center (ACC), located in the Innovation District in Columbus, Ohio.

From Andelyn Biosciences 23 Jun 2023

World's largest gathering of NF community convenes in Arizona to advance research and care

From June 21 through June 27, the largest gathering of NF researchers, clinicians, and patients in the world will take place at the Fairmont Scottsdale Princess in Scottsdale, Arizona.

22 Jun 2023

Newborn screening for "bubble-baby" disease boosts survival of children

Screening newborns for severe combined immunodeficiency disease (SCID) significantly increases the survival of children after bone marrow transplantation, a new North American study finds.

21 Jun 2023

Newborn screening facilitates early identification of infants with severe combined immunodeficiency

Introducing widespread screening of newborns for a deadly disease called severe combined immunodeficiency, or SCID, followed by early treatment boosted the five-year survival rate of children with the disorder from 73% before the advent of screening to 87% since, researchers report.

20 Jun 2023

Phase I clinical study of TCR NK cell therapy for sarcoma expected to commence later this year

The University of Texas MD Anderson Cancer Center and Replay today announced that the Food & Drug Administration (FDA) has issued a 'safe to proceed' for the Investigational New Drug (IND) application for NY-ESO-1 TCR/IL-15 NK, an engineered T cell receptor natural killer (TCR NK) cell therapy for advanced synovial sarcoma and myxoid/round cell liposarcoma.

20 Jun 2023

Catalent adds new cryogenic capabilities at Shiga, Japan, facility to support clinical supply demand for cell and gene therapy development

Catalent today announced that it has expanded the services and capabilities at its facility in Shiga, Japan, to include the storage, kitting, and distribution of advanced therapies at ultra-low temperatures for clinical trials.

From Catalent 16 Jun 2023

New base-edited T-cells proven effective to treat lymphoblastic leukemia

Three young patients with relapsed T-cell leukemia have now been treated with base-edited T-cells, as part of a 'bench-to-bedside' collaboration between UCL and Great Ormond Street Hospital for Children (GOSH).

15 Jun 2023

Charles River Laboratories Launches Off-the-Shelf Lentiviral Vector Packaging Plasmids

Charles River Laboratories International, Inc. (NYSE: CRL) today announced the launch of its off-the-shelf lentiviral vector (LVV) packaging plasmids, providing a streamlined solution to the challenges associated with plasmid sourcing in cell and gene therapies.

From Charles River Laboratories, Inc. 13 Jun 2023