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.

EpilepsyGTx and Viralgen collaborate to advance AAV gene therapy program for epilepsy ahead of first-in-human trial

EpilepsyGTx, a biotechnology company developing gene therapies for refractory epilepsy, and Viralgen, a leading contract development and manufacturing organization (CDMO) specializing in recombinant adeno-associated virus (AAV) gene therapies, today announced a partnership to support production of EpilepsyGTx's lead investigational gene therapy program targeting patients with focal refractory epilepsy (FRE), EPY201, ahead of first-in-human clinical trials.

From EpilepsyGTx 9 Sep 2026

Blocking a naturally occurring molecule reverses deadly lung disease in preclinical models

Researchers blocked a naturally occurring molecule to reverse pulmonary arterial hypertension in preclinical models, a finding that could point to a new treatment approach for people with a rare and often fatal lung disease.

2 Sep 2026

Unexpected discovery points to a possible route for childhood brain tumor treatments

When researchers at Lund University investigated how the molecule UM171 works, a molecule used to make blood stem cells multiply, they made an unexpected connection.

27 Aug 2026

Red blood cells inspire next-generation therapeutic nanocarriers

Red blood cells serve as the foundation for nanocarriers that show promise in a new study as effective and efficient vehicles for gene therapy, tumor targeting and other medical treatments.

19 Aug 2026

Penn State team lays groundwork to 3D print spheroids for regenerating bone tissue

An interdisciplinary team of engineers and chemists at Penn State has laid the groundwork to 3D print spheroids - tiny clusters of living cells - capable of regenerating bone tissue in response to severe trauma or infections.

18 Aug 2026

Engineered red blood cell vesicles improve gene therapy delivery

Red blood cells serve as the foundation for nanocarriers that show promise in a new study as effective and efficient vehicles for gene therapy, tumor targeting and other medical treatments.

17 Aug 2026

SynGenSys introduces expanded CHO.SET synthetic promoter library for enhanced biomanufacturing

SynGenSys, a biotechnology company designing synthetic gene promoter systems to address critical bottlenecks in biopharma manufacturing and enhance cell and gene therapy development, today announced the introduction of CHO.SET® 2.0. The expanded library of novel and patentable synthetic promoters is designed to improve protein production in CHO (Chinese Hamster Ovary) cells.

From SynGenSys Ltd. 13 Aug 2026

Personalized T-cell therapy eliminates advanced kidney tumor in teenager

Researchers and physicians at the Hopp Children's Cancer Center Heidelberg (KiTZ) and other leading cancer centers report an exceptional treatment success in an adolescent with an advanced, therapy-resistant kidney tumor.

12 Aug 2026

Targeting single gene reverses developmental deficits in adult mice

Some of the symptoms of neurodevelopmental disorders (NDDs) such as autism spectrum disorder and epilepsy may result from dysfunction of certain neurons in the brain and their ability to change their properties in response to experiences-a process called experience-dependent plasticity.

12 Aug 2026

Early-life stress rewires brain epigenetics to heighten sensitivity to future stress

. Early-life stress increased SETD7 and H3K4me1 in the ventral tegmental area of mice, creating an epigenetically primed state that heightened later transcriptional, neuronal, and behavioral responses to stress. Reducing Setd7 during development lowered H3K4me1 and weakened these stress-sensitizing effects, identifying a mechanism that may help explain how early adversity leaves lasting biological effects.

11 Aug 2026

New combination therapy could permanently clear HIV in newborns

Every year, more than 120,000 newborns worldwide contract HIV, a global health burden that requires lifelong treatment for millions of people - assuming they have access and can afford it.

10 Aug 2026

Multi-omics research uncovers new therapeutic targets for Fabry disease

A comprehensive review is shedding new light on Fabry disease, a rare inherited disorder that can progressively damage multiple organs and significantly affect quality of life.

6 Aug 2026

RNA-based therapeutic strategy targets brain pathway behind cancer-associated cachexia

A new path has opened toward stopping cancer-associated cachexia, a devastating complication that gradually wastes patients away.

2 Aug 2026

New OpenABE gene editor improves editing efficiency by up to 36 times

A joint research team led by Professor Daesik Kim (Department of Medicine, Sungkyunkwan University), along with Professor Yong-Sub Kim (University of Ulsan College of Medicine) and Professor Jae-Hyun Park (Sungkyunkwan University School of Medicine), has announced the development of 'OpenABE' (Open Adenine Base Editor).

30 Jul 2026

New strategy combines cancer immunotherapy and gene therapy in a single nanoparticle

Cancer cells survive by hiding from the immune system's surveillance. A KAIST research team has developed a new anticancer platform that makes cancer cells send out their own danger signal—prompting immune cells to attack—while simultaneously delivering gene therapy.

28 Jul 2026

Emerging non-endocytic transmembrane delivery strategies for precision medicine

Conventional endocytosis-mediated delivery is limited by poor endosomal escape efficiency, making intracellular delivery of therapeutic biomacromolecules a long-standing challenge in modern biomedical research.

24 Jul 2026

Compact genetic switch enables targeted gene therapy for epilepsy

Epilepsy affects over 50 million people worldwide, making it one of the most common neurological disorders.

23 Jul 2026

Senolytic drugs rejuvenate blood stem cells in sickle cell disease

Sickle cell disease causes premature aging of blood stem cells, which scientists may be able to address with a special class of drugs, according to a new study from St. Jude Children's Research Hospital.

22 Jul 2026

SynGenSys makes key appointments to strengthen senior commercial and scientific teams

SynGenSys, a biotechnology company designing synthetic gene promoter systems to address critical bottlenecks in biopharma manufacturing and enhance cell and gene therapy development, today announced the expansion of its senior commercial and scientific teams with the appointments of several heads of departments.

From SynGenSys Ltd. 22 Jul 2026

New ARPA-H grant aims to advance genomic medicines for rare vascular diseases

Massachusetts General Hospital, a founding member of the Mass General Brigham healthcare system, has been awarded an Advanced Research Projects Agency for Health (ARPA-H) contract to develop advanced personalized genomic medicines to treat rare genetic vascular diseases.

21 Jul 2026