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.

Scripps Research wins $4 million CIRM grant to develop novel platform for neurodevelopmental disorders

Most people are born with two functioning copies of each gene-one inherited from each parent. In many cases, having just one working copy is enough for the body to function normally.

28 Oct 2025

Les Turner Symposium on ALS brings researchers and clinicians to discuss new research findings

The 15th annual Les Turner Symposium on ALS will be held on Monday, Nov. 3, 2025 in Chicago. Hosted by the Les Turner ALS Foundation and the Les Turner ALS Center at Northwestern Medicine, this event will gather leading ALS researchers and clinicians to discuss the latest research and perspectives on the disease.

23 Oct 2025

International experts publish guidelines on the administration of gene therapy for hereditary hearing loss

Up to 60% of congenital and early-onset hearing loss is caused by genetic mutations in an inherited gene, and gene therapy has recently emerged as a potential treatment option.

23 Oct 2025

New gene-editing method can correct many disease-causing mutations in mammalian cells

Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same disorder might have a different combination of mutations.

23 Oct 2025

Researchers achieve proof of concept for generating eggs from somatic cells

Researchers at Oregon Health & Science University have accomplished a unique proof of concept to treat infertility by turning skin cells into eggs capable of producing early human embryos.

17 Oct 2025

New European trial tests subcutaneous EEG technology for people with epilepsy

A new clinical trial co-led by researchers at FutureNeuro and RCSI University of Medicine and Health Sciences is investigating how advanced brain monitoring could improve the diagnosis and management of epilepsy.

16 Oct 2025

Longevity gene offers hope for children with progeria

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' found in people who live exceptionally long lives - over 100 years old.

15 Oct 2025

Experimental gene therapy restores immune system function in children with genetic immune disorder

An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare and deadly genetic immune disorder.

15 Oct 2025

Gene therapy shows long-term success in children with rare immune disorder

Results from the largest cohort of children who received a gene therapy for a rare immunodeficiency condition have shown the long-term safety and efficacy of the curative treatment, in a study led by a UCL researcher.

15 Oct 2025

New gene therapy reverses symptoms of SYNGAP1-related disorders in mice

In an exciting scientific first, researchers at the Allen Institute successfully designed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders (SRD) in mice.

9 Oct 2025

The ISSCR releases new resource to accelerate the development of PSC-derived therapies

The International Society for Stem Cell Research (ISSCR) is proud to announce the release of Best Practices for the Development of Pluripotent Stem Cell-Derived Cellular Therapies, a comprehensive, interactive new resource designed to accelerate the translation of human pluripotent stem cell (PSC)-derived therapies into clinical trials and commercial use.

9 Oct 2025

Empowering genomics research with cloud-based innovation

Discover how Discovery Life Sciences enhances genomics research with cloud-based platforms like DNAnexus.

From DNAnexus, Inc. 8 Oct 2025

Aptamer expands into targeted radiopharmaceuticals with major top 3 pharma contract

Aptamer Group plc, the developer of next-generation synthetic binders for the life sciences industry, has announced a significant development contract with a top 3 global pharmaceutical company to engineer Optimer® binders as targeted radiopharmaceuticals for potential therapeutic applications.

8 Oct 2025

New partnership brings next-generation cell isolation solutions to researchers advancing cell and gene therapy development

Cayman Chemical, a global provider of life science research tools, and Akadeum Life Sciences, a leader in buoyancy-based cell separation technology, announced today a distribution partnership to expand access to Akadeum's next-generation cell isolation technology for cell and gene therapy development.

3 Oct 2025

Gene Therapy News and Research

Further Reading

Scripps Research wins $4 million CIRM grant to develop novel platform for neurodevelopmental disorders

Les Turner Symposium on ALS brings researchers and clinicians to discuss new research findings

International experts publish guidelines on the administration of gene therapy for hereditary hearing loss

New gene-editing method can correct many disease-causing mutations in mammalian cells

Researchers achieve proof of concept for generating eggs from somatic cells

New European trial tests subcutaneous EEG technology for people with epilepsy

Longevity gene offers hope for children with progeria

Experimental gene therapy restores immune system function in children with genetic immune disorder

Gene therapy shows long-term success in children with rare immune disorder

New gene therapy reverses symptoms of SYNGAP1-related disorders in mice

The ISSCR releases new resource to accelerate the development of PSC-derived therapies

Empowering genomics research with cloud-based innovation

Aptamer expands into targeted radiopharmaceuticals with major top 3 pharma contract

New partnership brings next-generation cell isolation solutions to researchers advancing cell and gene therapy development

Highly targeted gene therapy could revolutionize treatment for cancers linked to herpesvirus

Harnessing “DNA surgery” to develop therapeutic medicines for genetic liver and heart diseases

SynGenSys introduces NK.SET synthetic promoter library to advance natural killer cell therapies

Gut microbial imbalance linked to chronic sickle cell disease pain

Novel biotechnical vector integrates viral and synthetic features for targeted cancer therapy

FUJIFILM Biosciences introduces BalanCD HEK293 perfusion a medium to enable gene therapy production

Ultimate guide to the essential tools for the characterization of proteins, polymers and nanoparticles eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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