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New initiative aims to scale individualized therapies for rare diseases

Critical Path Institute (C-Path) today announced the launch of One to Millions, a global, multi-stakeholder public-private initiative to enable scalable development of advanced therapies for highly individualized conditions.

26 Mar 2026

The new microchip electrophoresis system MultiNA II MCE-301- High sensitivity detection, improved analysis function and operability

Shimadzu has recently released the MultiNA II MCE-301 microchip electrophoresis system. By automating the entire process of electrophoresis, which takes more than two hours by hand (creation of gels, dispensing reagents, dispensing sample, separation, dyeing, cleaning of equipment), the company significantly improves the efficiency of genetic analysis operations.

From Shimadzu Europa GmbH 26 Mar 2026

Tiny gene mutations shape cancer behavior and immune response

Cancer is often thought of as a single disease. Yet even tumors that arise in the same organ can follow very different genetic paths.

19 Mar 2026

Newly identified protein regulates lysosome positioning and cellular metabolism

Lysosomes are the control centers for the metabolism of cells and tissues, including the brain. They break down defective proteins and other macromolecules into their basic building blocks.

18 Mar 2026

Vitamin B2 metabolism helps cancer cells resist ferroptosis

A lack of vitamin B2 makes tumour cells more susceptible to a unique form of cell death. This was discovered by researchers at the Rudolf Virchow Centre at the University of Würzburg.

13 Mar 2026

Protrusion-derived extracellular vesicles boost functional cargo transfer

Extracellular vesicles (EVs) are tiny membrane-bound particles released by cells to transport proteins and other molecules to neighboring cells. Because of this natural delivery ability, EVs have attracted growing interest as potential vehicles for therapeutic protein and genome-editing enzyme delivery.

13 Feb 2026

Scientists create new map showing how cancer gene mutations influence tumor growth

Scientists have created a complete map showing how hundreds of possible mutations in a key cancer gene influence tumor growth.

2 Feb 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

New method can help better assess precision genome editing technology

Scientists and physicians can better assess precision genome editing technology using a new method made public today by St. Jude Children's Research Hospital. Significant amounts of time and resources spent improving CRISPR gene editing technology focus on identifying small off-target sites that pose a safety risk, which is also technically challenging.

2 Jan 2026

Reframing the role of MCL1 in cancer signaling and metabolism

A study by the Mildred Scheel Early Career Center group led by Dr. Mohamed Elgendy at the TUD Faculty of Medicine provides fundamental insights into cancer biology.

22 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

Timing of genetic mutation dictates childhood leukemia aggressiveness

A team of researchers at the Icahn School of Medicine at Mount Sinai has uncovered why children with the same leukemia-causing gene mutation can have dramatically different outcomes: it depends on when in development the mutation first occurs.

8 Dec 2025

Study reveals impact of viral combinations on infant respiratory illness severity

A new study led by researchers at National Jewish Health has revealed that, while a wide range of viruses can cause lower respiratory tract illnesses (LRIs) in infants, certain viruses and viral combinations dramatically increase the risk of severe disease.

24 Oct 2025

New gene-editing method can correct many disease-causing mutations in mammalian cells

Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person's genome, often with enough variation that even two individuals who share the same disorder might have a different combination of mutations.

23 Oct 2025

Harnessing “DNA surgery” to develop therapeutic medicines for genetic liver and heart diseases

Genome editing is increasingly recognized as a leading frontier in biomedical research and an essential driver of healthcare innovation.

30 Sep 2025

New screening method boosts efficiency and accuracy of CAST genome editing

Natural systems such as CRISPR-associated transposons (CASTs) offer a targetable, one-step way to edit genomes.

23 Sep 2025

CRISPR-based therapeutic approach designed to treat fatal pediatric disease

Multisystemic smooth muscle dysfunction syndrome (MSMDS) is a rare condition associated with stroke, aortic dissection (tearing) and death in childhood.

11 Sep 2025

Nanostructures boost CRISPR delivery for genetic medicine

With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense promise to revolutionize medicine. But until scientists can deliver its gene-editing machinery safely and efficiently into relevant cells and tissues, that promise will remain out of reach.

2 Sep 2025

Clonogenic hepatocytes identified as engines of liver development

In a new study published in the Journal of Hepatology, researchers from the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) show that only 15–20% of hepatocytes in newborn mice - dubbed clonogenic hepatocytes - are responsible for generating over 90% of the adult liver mass.

29 Aug 2025