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.

Balance depends on preserving inner hair cells in the ear

Led by Mathieu Beraneck, researchers at the University of Paris Cité/CNRS and the University of Barcelona explored the strength of the relationship between a type of inner hair cell in the ear and balance.

9 Feb 2026

SynGenSys introduces Liver.SET synthetic promoter library for liver-specific gene expression for in vivo gene therapies

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 launch of its Liver.SET™ library of liver-specific synthetic promoters.

5 Feb 2026

New workflow boosts nuclear delivery for safer gene therapy

Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells. However, to be effective, the new gene must make it into a cell's nucleus.

5 Feb 2026

Exploring Targeted Protein Degradation as a New Strategy to Tackle Antimicrobial Resistance

Targeted protein degradation presents a promising strategy to address antimicrobial resistance, focusing on innovative approaches for gram-negative bacteria.

28 Jan 2026

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for the first time.

27 Jan 2026

Gene therapy targeting connexin 43 shows promise across inherited heart diseases

University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse model can dramatically improve heart function and extend survival in several inherited forms of arrhythmogenic cardiomyopathy (ACM)

26 Jan 2026

Unraveling liver injury mechanisms in familial hypobetalipoproteinemia

Familial Hypobetalipoproteinemia (FHBL), caused by variants in the apolipoprotein B (APOB) gene, is a rare autosomal co-dominant monogenic disorder characterized by lifelong low plasma levels of total cholesterol, low-density lipoprotein cholesterol (LDL-C), and APOB.

24 Jan 2026

Trojan horse immunotherapy opens tumors to immune system attack

Scientists at the Icahn School of Medicine at Mount Sinai have developed an experimental immunotherapy that takes an unconventional approach to metastatic cancer: instead of going after cancer cells directly, it targets the cells that protect them.

22 Jan 2026

New partnership aims to expand global access to sickle cell gene therapy

Caring Cross, a 501(c)(3) non-profit dedicated to improving access to advanced therapies, and Boston Children's Hospital today announced a collaboration to provide a sustainable, affordable pathway for patients to access stem cell gene therapies for the treatment of Sickle Cell Disease (SCD).

22 Jan 2026

Medicaid tries new approach with sickle cell: Companies get paid only if costly gene therapies work

Serenity Cole enjoyed Christmas last month relaxing with her family near her St. Louis home, making crafts and visiting friends.

21 Jan 2026

ELRIG announces Drs Stephen Ward and Annarita Miccio as keynote speakers for Cell and Gene Therapy 2026

ELRIG, a not-for-profit, volunteer-led organization dedicated to the global drug discovery community, today announced the keynote speakers for Cell and Gene Therapy 2026. Drs Stephen Ward (Cell and Gene Therapy Catapult) and Annarita Miccio (Imagine Institute) will lead the inaugural two-day event taking place at Hinxton Hall in Cambridge, UK from 9-10 March.

20 Jan 2026

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Genetic disorders occur due to alterations in the primary genetic material, deoxyribonucleic acid (DNA), of an organism. Transthyretin amyloidosis (ATTR) is a progressive disorder involving amyloid deposits of misfolded transthyretin (TTR) proteins.

14 Jan 2026

Disease-responsive nanoparticles enable ghrelin mRNA therapy in osteoarthritis

Nitin Joshi, PhD, and Jingjing Gao, PhD, of the Department of Anesthesiology at Mass General Brigham, are the co-senior authors of a paper published in Nature Nanotechnology, "A disease-severity-responsive nanoparticle enables potent ghrelin mRNA therapy in osteoarthritis." Mahima Dewani, PhD, is the lead author of this study.

14 Jan 2026

Novel immunotherapy targeting pancreatic cancer shows promising results in phase 1/2 trial

A recent publication in Nature Medicine describes a novel immunotherapy targeting pancreatic cancer that has shown promising results in a first in-human phase 1/2 trial.

13 Jan 2026

APOE gene: A major driver in Alzheimer's disease

Potentially more than 90% of Alzheimer's disease cases would not occur without the contribution of a single gene (APOE), according to a new analysis led by UCL researchers.

9 Jan 2026

CIRM awards $7.4 million to advance stem cell-based gene therapy for Friedreich's ataxia

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for Friedreich's ataxia, a rare inherited neurodegenerative disease that causes progressive loss of coordination, muscle strength, heart function and overall mobility.

8 Jan 2026

Gene therapy breakthrough could provide hope for millions living with chronic pain

A preclinical study uncovered a new gene therapy that targets pain centers in the brain while eliminating the risk of addiction from narcotics treatments, a breakthrough which could provide hope for the more than 50 million Americans living with chronic pain.

7 Jan 2026

Gene Therapy News and Research

Further Reading

Balance depends on preserving inner hair cells in the ear

SynGenSys introduces Liver.SET synthetic promoter library for liver-specific gene expression for in vivo gene therapies

New workflow boosts nuclear delivery for safer gene therapy

Exploring Targeted Protein Degradation as a New Strategy to Tackle Antimicrobial Resistance

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene therapy targeting connexin 43 shows promise across inherited heart diseases

Unraveling liver injury mechanisms in familial hypobetalipoproteinemia

Trojan horse immunotherapy opens tumors to immune system attack

New partnership aims to expand global access to sickle cell gene therapy

Medicaid tries new approach with sickle cell: Companies get paid only if costly gene therapies work

ELRIG announces Drs Stephen Ward and Annarita Miccio as keynote speakers for Cell and Gene Therapy 2026

CRISPR-Cas3: A safer gene-editing tool shows promise for transthyretin amyloidosis treatment

Disease-responsive nanoparticles enable ghrelin mRNA therapy in osteoarthritis

Novel immunotherapy targeting pancreatic cancer shows promising results in phase 1/2 trial

APOE gene: A major driver in Alzheimer's disease

CIRM awards $7.4 million to advance stem cell-based gene therapy for Friedreich's ataxia

Gene therapy breakthrough could provide hope for millions living with chronic pain

TAAR1 mutation impairs brain signaling in schizophrenia

Advances in hemophilia gene therapy bring hope for pediatric patients

Junk' DNA may hold new clues to Alzheimer’s disease

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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