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.

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

Tailored gene therapy dramatically reduces childhood epilepsy seizures in trial

SCN2A‑related developmental epileptic encephalopathy (DEE) is a rare, severe form of childhood epilepsy and one of the most common causes of monogenic autism.

21 Jul 2026

New photoswitchable drugs restore light perception in blind animals

Blinding diseases caused by the degeneration of photoreceptors, such as age-related macular degeneration (AMD) and retinitis pigmentosa (RP), affect 200 million people worldwide and represent the leading causes of visual impairment and blindness.

15 Jul 2026

Experimental gene therapy reverses fragile X symptoms in mice

A gene therapy designed to replace the missing protein that causes fragile X syndrome restored several disease-relevant traits in a mouse model, according to a new study published in Gene Therapy.

14 Jul 2026

EpilepsyGTx partners with ClearPoint Neuro to support targeted delivery of lead gene therapy in epilepsy

EpilepsyGTx, a biotechnology company developing cutting-edge gene therapies to treat refractory epilepsy, and ClearPoint Neuro ("ClearPoint"), a global therapy-enabling company offering precise navigation and delivery to the brain, today announced a partnership to support the targeted delivery of EpilepsyGTx's lead gene therapy candidate, EPY201, for focal refractory epilepsy (FRE).

From EpilepsyGTx 14 Jul 2026

Niche-targeted therapies offer a fresh approach to regenerative medicine

A healthy bone marrow (BM) produces nearly all types of cells in our blood. Many blood disorders occur when hematopoietic stem cells (HSCs) in the BM malfunction.

10 Jul 2026

Off-the-shelf CAR T therapy targets treatment-resistant systemic sclerosis

Today at ISSCR 2026, new preliminary clinical data was presented evaluating an induced pluripotent stem cell (iPSC)-derived CAR T-cell therapy for patients with treatment-resistant systemic sclerosis, a rare autoimmune disease with high mortality.

9 Jul 2026

Phelan-McDermid syndrome may be more common than previously believed

New research, led by scientists from the Seaver Autism Center for Research and Treatment at Mount Sinai and published June 28 in Autism Research, has estimated that Phelan-McDermid syndrome (PMS) affects approximately 1 in 7,300 people, making it dramatically more common than previous estimates suggested.

9 Jul 2026

Autolomous and Cellular Origins expand platform integration to deliver end-to-end cell therapy manufacturing orchestration

Autolomous and Cellular Origins today announce end-to-end integration of their respective platforms, bringing full automation and digitization to the entire cell therapy manufacturing process.

From Cellular Origins 9 Jul 2026

Weill Cornell Medicine receives federal grant to study new gene therapy for Alzheimer's disease

The National Institute on Aging of the National Institutes of Health has awarded a five-year grant initially worth $8.37 million for Weill Cornell Medicine researchers to study a new gene therapy for Alzheimer's disease in people who have a high genetic risk of developing the condition.

8 Jul 2026

Novel strategy can make stem cell transplants safer

Stem cell transplantation (also called bone marrow transplantation) and gene therapy are among the most powerful curative approaches for blood diseases such as sickle cell disease, b-thalassemia, immune deficiencies, and some blood cancers.

8 Jul 2026

New gene therapy strategy uses the brain's own glymphatic transport system for drug delivery

A new study describes a gene therapy strategy that uses the brain's own glymphatic transport system to distribute engineered viral vectors throughout the brain.

8 Jul 2026

Bio-Rad launches Vericheck ddPCR Kits compatible with QX700 platform for biopharma quality control and cell and gene therapy workflows

Bio-Rad Laboratories, Inc., a global leader in life science research and clinical diagnostics products, today announced the launch of the QX700 System-compatible Vericheck ddPCR Kit portfolio for critical quality control (QC) workflows in advanced cell and gene therapy development and other biopharma applications.

From Bio-Rad Laboratories 8 Jul 2026

Gene Therapy News and Research

Further Reading

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

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

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

Emerging non-endocytic transmembrane delivery strategies for precision medicine

Compact genetic switch enables targeted gene therapy for epilepsy

Senolytic drugs rejuvenate blood stem cells in sickle cell disease

SynGenSys makes key appointments to strengthen senior commercial and scientific teams

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

Tailored gene therapy dramatically reduces childhood epilepsy seizures in trial

New photoswitchable drugs restore light perception in blind animals

Experimental gene therapy reverses fragile X symptoms in mice

EpilepsyGTx partners with ClearPoint Neuro to support targeted delivery of lead gene therapy in epilepsy

Niche-targeted therapies offer a fresh approach to regenerative medicine

Off-the-shelf CAR T therapy targets treatment-resistant systemic sclerosis

Phelan-McDermid syndrome may be more common than previously believed

Autolomous and Cellular Origins expand platform integration to deliver end-to-end cell therapy manufacturing orchestration

Weill Cornell Medicine receives federal grant to study new gene therapy for Alzheimer's disease

Novel strategy can make stem cell transplants safer

New gene therapy strategy uses the brain's own glymphatic transport system for drug delivery

Bio-Rad launches Vericheck ddPCR Kits compatible with QX700 platform for biopharma quality control and cell and gene therapy workflows

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

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