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.

Researchers identify chromatin accessibility changes driving stem cell transformation in MDS

Over the past few decades, advances in hematology have illuminated how a delicate balance between stem cell self-renewal and differentiation sustains healthy blood formation.

12 Dec 2025

$EpilepsyGTx raises $33 million Series A to develop single dose gene therapy for focal refractory epilepsy$

EpilepsyGTx raises $33 million Series A to develop single dose gene therapy for focal refractory epilepsy

EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing to advance its lead program EPY201 through Phase 1/2a clinical trials.

10 Dec 2025

VALANX Biotech establishes Board of Directors

VALANX Biotech (VALANX), a biotech company developing novel antibody-drug-conjugate (ADC) assets enabled by its GoldenSite™ site-specific protein conjugation technology, today announced the formation of its Board of Directors.

10 Dec 2025

Genome-edited immune cell therapy shows promise for treating aggressive blood cancer

A groundbreaking new treatment using genome-edited immune cells, developed by scientists at UCL (University College London) and Great Ormond Street Hospital (GOSH), has shown promising results in helping children and adults fight a rare and aggressive form of blood cancer called T-cell acute lymphoblastic leukemia (T-ALL).

8 Dec 2025

Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia

The first study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing demand for gene therapies in the coming years.

7 Dec 2025

Exa-cel shows complete success in early pediatric trials for sickle cell disease and beta-thalassemia

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12.

6 Dec 2025

CRISPR-based Cellgorithm technology ushers in a new era of cell programming

Syntax Bio, a synthetic biology company programming the next generation of cell therapies, today announced the publication of new research in Science Advances detailing the company's CRISPR-based Cellgorithm technology, which lays the groundwork for programmable control of gene activity in human stem cells and offers an alternative to the slow, variable manual processes researchers use today.

5 Dec 2025

Extending the reach of Amperia™: Simplifying his-tagged protein quantification across expression and development workflows

Discover how Abselion’s Amperia™ platform delivers fast, reproducible His-tagged protein quantification with minimal prep, even from crude lysates.

From Abselion 27 Nov 2025

Eli Lilly’s Adverum move: The future of ocular gene therapy at the 4th Ophthalmic Drug Delivery Summit

The race to develop safer, longer lasting, and less invasive treatments is accelerating, and at the heart of that challenge lies drug delivery.

From Hanson Wade Group - 4th Ophthalmic Drug Delivery Summit 26 Nov 2025

Scientists reverse aging in blood stem cells by restoring lysosome function

Researchers at the Icahn School of Medicine at Mount Sinai have discovered how to reverse aging in blood-forming stem cells in mice by correcting defects in the stem cell's lysosomes.

24 Nov 2025

New discovery could reshape how scientists think about Alzheimer’s treatment

Rutgers neuroscientist Peng Jiang was visiting his hometown of Qianshan, a city in China's Anhui province, when a neighbor came to his parents' house with a story that would stay with him.

24 Nov 2025

Astrocyte-driven approach shows potential to reverse cognitive decline in Alzheimer’s

Researchers at Baylor College of Medicine have discovered a natural mechanism that clears existing amyloid plaques in the brains of mouse models of Alzheimer's disease and preserves cognitive function.

21 Nov 2025

Muscle-driven gene therapy shows promise for protecting memory in Alzheimer’s

Alzheimer's disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment is available.

18 Nov 2025

Fondazione Telethon achieves milestone with Waskyra approval for Wiskott-Aldrich Syndrome

Fondazione Telethon announces the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending marketing authorization in the European Union for Waskyra, an ex vivo gene therapy for Wiskott-Aldrich Syndrome (WAS), a rare and life-threatening primary immunodeficiency.

14 Nov 2025

Terasaki Institute and Caltech join forces on groundbreaking embryo formation study

The Terasaki Institute for Biomedical Innovation (TIBI) is pleased to announce its collaboration with the California Institute of Technology (Caltech) on a newly awarded $2.8 million Discovery Stage Research (DISC0) grant from the California Institute for Regenerative Medicine (CIRM).

14 Nov 2025

Celleo Biotech uses INTEGRA's PIPETBOY GENIUS to improve reproducibility and efficiency

Celleo Biotech is using the PIPETBOY GENIUS serological pipet controller from INTEGRA Biosciences to streamline the development and manufacturing of novel cell and gene therapies.

From INTEGRA Biosciences 13 Nov 2025

New precision therapies reshape the future of pancreatitis treatment

Pancreatitis remains a major global health burden, encompassing acute pancreatitis (AP), chronic pancreatitis (CP), and autoimmune pancreatitis (AIP).

11 Nov 2025

Study advances the possibility of age-based pancreatic cancer treatment

While some risk factors for cancer can be mitigated through lifestyle choices such as diet or exercise, aging is not one of them. In the case of pancreatic cancer, it also can limit treatment options if a patient is too frail to be safely treated with surgery or other alternatives.

10 Nov 2025

One-time CRISPR therapy safely lowers cholesterol and triglycerides in early trial

In a 15-patient, Phase 1 one, first-in-human trial, a one-time, CRISPR-Cas9 gene-editing therapy safely reduced LDL cholesterol and triglycerides in people with difficult-to-treat lipid disorders, according to a preliminary late-breaking science presentation today at the American Heart Association's Scientific Sessions 2025.

10 Nov 2025

Uppsala researchers develop Trojan horse approach for medulloblastoma treatment

The malignant pediatric brain tumor medulloblastoma is driven by proteins deep within the cancer cells, for which there are often no effective drugs available.

10 Nov 2025

Gene Therapy News and Research

Further Reading

Researchers identify chromatin accessibility changes driving stem cell transformation in MDS

EpilepsyGTx raises $33 million Series A to develop single dose gene therapy for focal refractory epilepsy

VALANX Biotech establishes Board of Directors

Genome-edited immune cell therapy shows promise for treating aggressive blood cancer

Challenges and opportunities in delivering gene therapies for sickle cell disease and beta thalassemia

Exa-cel shows complete success in early pediatric trials for sickle cell disease and beta-thalassemia

CRISPR-based Cellgorithm technology ushers in a new era of cell programming

Extending the reach of Amperia™: Simplifying his-tagged protein quantification across expression and development workflows

Eli Lilly’s Adverum move: The future of ocular gene therapy at the 4th Ophthalmic Drug Delivery Summit

Scientists reverse aging in blood stem cells by restoring lysosome function

New discovery could reshape how scientists think about Alzheimer’s treatment

Astrocyte-driven approach shows potential to reverse cognitive decline in Alzheimer’s

Muscle-driven gene therapy shows promise for protecting memory in Alzheimer’s

Fondazione Telethon achieves milestone with Waskyra approval for Wiskott-Aldrich Syndrome

Terasaki Institute and Caltech join forces on groundbreaking embryo formation study

Celleo Biotech uses INTEGRA's PIPETBOY GENIUS to improve reproducibility and efficiency

New precision therapies reshape the future of pancreatitis treatment

Study advances the possibility of age-based pancreatic cancer treatment

One-time CRISPR therapy safely lowers cholesterol and triglycerides in early trial

Uppsala researchers develop Trojan horse approach for medulloblastoma treatment

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