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.

Review highlights emerging treatments for glioblastoma multiforme

A new research perspective was published in Volume 13 of Oncoscience on September 17, 2026, titled "Emerging pharmacological agents for glioblastoma multiforme: An update on active clinical trials (2025–2026)."

25 Sep 2026

C-Path awards $1.7 million to advance five drug development projects

Critical Path Institute (C-Path) today announced that its Translational Therapeutics Accelerator has awarded $1,699,500 through five Bridging Research and Innovation in Drug Development Grants (BRIDGe).

22 Sep 2026

Chronic DNA damage response may contribute to biological aging

A new review was published in Volume 18 of Aging on August 31, 2026, titled "The guardian paradox: DNA damage response overactivation as an integrative driver of aging – a tumor suppressor–negative regulator framework."

21 Sep 2026

Adenine base editing slows neurodegeneration in hereditary motor neuron disease

A team of researchers led by Professor Haruhisa Inoue (Center for iPS Cell Research and Application, Kyoto University) and Professor Yuishin Izumi (Tokushima University) demonstrated that adenine base editing can correct a disease-causing genetic mutation and alleviate neurodegeneration in hereditary motor and sensory neuropathy with proximal dominant involvement (HMSN-P), providing proof of concept for a precision gene therapy for inherited motor neuron diseases.

18 Sep 2026

Otolaryngologists gather to present latest advances in ear, nose, and throat care

The latest treatments and research advances in ear, nose, and throat care will take center stage in Los Angeles October 17-20, when more than 5,000 of the world's leading otolaryngologists gather for the 2026 Annual Meeting & OTO EXPO of the American Academy of Otolaryngology–Head and Neck Surgery Foundation.

18 Sep 2026

New prime assembly method enables long and precise DNA insertions

Genomic editing holds great potential yet continues to have its limitations. Most current methods either rely on untargeted gene delivery or short DNA edits that need to be individualized to each patient.

16 Sep 2026

ACROBiosystems deepens its France presence with a dedicated hub at The HIVE Innovation Center, embedding into the Campus Grand Parc ecosystem

Kadans Science Partner is pleased to welcome ACROBiosystems to The HIVE Innovation Center, its laboratory and office building dedicated to the life sciences, situated on the Campus Grand Parc in Villejuif.

From ACROBiosystems 16 Sep 2026

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

Gene Therapy News and Research

Further Reading

Review highlights emerging treatments for glioblastoma multiforme

C-Path awards $1.7 million to advance five drug development projects

Chronic DNA damage response may contribute to biological aging

Adenine base editing slows neurodegeneration in hereditary motor neuron disease

Otolaryngologists gather to present latest advances in ear, nose, and throat care

New prime assembly method enables long and precise DNA insertions

ACROBiosystems deepens its France presence with a dedicated hub at The HIVE Innovation Center, embedding into the Campus Grand Parc ecosystem

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

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

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

Red blood cells inspire next-generation therapeutic nanocarriers

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

Engineered red blood cell vesicles improve gene therapy delivery

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

Personalized T-cell therapy eliminates advanced kidney tumor in teenager

Targeting single gene reverses developmental deficits in adult mice

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

New combination therapy could permanently clear HIV in newborns

Multi-omics research uncovers new therapeutic targets for Fabry disease

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

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

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

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