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.

Penn Medicine researchers identify new therapeutic target for Alzheimer's disease

A gene encoding a protein linked to tau production-;tripartite motif protein 11 (TRIM11)-;was found to suppress deterioration in small animal models of neurodegenerative diseases similar to Alzheimer's disease (AD), while improving cognitive and motor abilities, according to new research from the Perelman School of Medicine at the University of Pennsylvania.

27 Jul 2023

New gene editing approach could revolutionize the treatment of blood disorders

In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.

27 Jul 2023

NK:IO raises £1.2M, bringing total Seed funding to £5.1M, to advance pioneering NK cell therapy

New investment of £1.2M brings total Seed funding to £5.1M including Innovate UK grants of £1.9M.

From NK:IO Limited 25 Jul 2023

BioIVT to highlight its integral role in liquid biopsy research at AACC conference

BioIVT, a global research partner and biospecimen solutions provider for drug and diagnostic development, today announced that it will highlight the integral role it is playing in liquid biopsy research at the American Association for Clinical Chemistry Annual Scientific Meeting and Clinical Lab Expo.

21 Jul 2023

Unchained Labs goes all in on Lipid Nanoparticles, acquires Blacktrace Holdings

Unchained Labs, the life sciences company that’s all about getting biologics and gene therapy researchers the right tools for the job, acquired Blacktrace Holdings today.

From Unchained Labs 21 Jul 2023

Neuroscientists discover new brain cells that help correct navigational errors

During routine navigation in daily life, our brains use spatial mapping and memory to guide us from point A to point B. Just as routine: making a mistake in navigation that requires a course correction.

19 Jul 2023

Gene Therapy Contract Development and Manufacturing: Challenges and Future Perspectives

In this interview conducted at the Cell and Gene Therapy Summit Tour London, we speak to Ramin Baghirzade, Global Head of Commercial at Charles River for Gene Therapy, about the Gene Therapy CDMO solutions that Charles River Laboratories offers and more.

From Charles River Laboratories, Inc. 18 Jul 2023

New agreement to facilitate genetic diagnosis of hereditary retinal dystrophies

An agreement between the University of Barcelona spin-off DBGen Ocular Genomics and the company Novartis España will enable the genetic identification of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) in affected patients.

17 Jul 2023

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

Gene Therapy News and Research

Further Reading

Penn Medicine researchers identify new therapeutic target for Alzheimer's disease

New gene editing approach could revolutionize the treatment of blood disorders

NK:IO raises £1.2M, bringing total Seed funding to £5.1M, to advance pioneering NK cell therapy

BioIVT to highlight its integral role in liquid biopsy research at AACC conference

Unchained Labs goes all in on Lipid Nanoparticles, acquires Blacktrace Holdings

Neuroscientists discover new brain cells that help correct navigational errors

Gene Therapy Contract Development and Manufacturing: Challenges and Future Perspectives

New agreement to facilitate genetic diagnosis of hereditary retinal dystrophies

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

Stable nanoparticles train the immune system to fight cancer

Researchers develop a chemical approach to reverse aging

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

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

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

Using a vaccine to boost the response of engineered T cells

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

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

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

Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult

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

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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