A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

While recent advances in gene therapy have offered unprecedented options for patients with hemophilia, new data presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, revealed persistent concerns regarding the durability of these treatments and their potential liver toxicity.

The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.

As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.

Generating gametes from nonreproductive tissues could help overcome infertility. Previous studies have successfully transformed stem cells into viable oocytes through cellular reprogramming. Scientists at Oregon Health & Science University (OHSU) developed a method to derive them from skin cells via somatic cell nuclear transfer (SCNT), unlocking a mechanism that blends mitosis and meiosis. Now, the researchers have taken another step forward by generating fertilizable eggs from human skin cells.

Clarivate plc has unveiled the 2025 Citation Laureates. Widely considered a predictor of the Nobel Prizes, this recognition has highlighted the discovery of biomolecular condensates in chemistry and the innate immunity signaling pathway in physiology or medicine, as well as the identification of leukemia stem cells and ghrelin, the so-called hunger hormone.

Two independent studies applied CRISPR-based genetic editing – one to treat leukemia and the other to target myeloma – to overcome the challenges faced by CAR T cells, such as exhaustion, impaired activation and fratricide, a phenomenon in which they attack each other.

Antibodies that bind to sugars on the surface of cancer cells, rather than to proteins, have not yielded satisfactory results so far due to their low binding affinity. However, scientists at the University of California, Irvine (UCI) have developed therapeutic proteins that recognize so-called tumor-associated carbohydrate antigens (TACAs) using lectins with a robust structure resembling velcro. This design is highly specific and eliminates only tumor cells, regardless of cancer type, while sparing healthy tissues.

Clarivate plc has unveiled the 2025 Citation Laureates. Widely considered a predictor of the Nobel Prizes, this recognition has highlighted the discovery of biomolecular condensates in chemistry and the innate immunity signaling pathway in physiology or medicine, as well as the identification of leukemia stem cells and ghrelin, the so-called hunger hormone.