In vaccine development, one might think that targeting multiple epitopes increases the likelihood of improving outcomes. However, when several immunogens are administered together, the immune system does not always generate antibodies against all of them. Two parallel studies have overcome this challenge by using multiple simultaneous immunogens against HIV, effectively triggering various types of broadly neutralizing antibody (bnAb) precursors in two different preclinical animal models.
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.
Nabla Bio Inc. has established a new multiyear research collaboration with Takeda Pharmaceutical Co. Ltd. to advance AI-driven design of protein therapeutics.
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.
The 2025 Nobel Prize in Chemistry “is a story full of holes, but with enormous capacity to absorb all your attention,” Heiner Linke told reporters. “And other things.” Linke is Chair of the Nobel Committee for Chemistry. On Oct. 8, 2025, the committee announced that it has awarded the 2025 Nobel Prize in Chemistry to Susumu Kitagawa, Richard Robson and Omar Yaghi “for the development of metal-organic frameworks” (MOFs).
Activation of cannabinoid receptors in the peripheral nervous system may help treat inflammatory and gastrointestinal disorders as well as pain, and several full agonists have been reported but they present safety concerns. Partial agonists, such as those that activate receptors to 20%-50% of full activity, may be safer, yet so far such agonists have emerged serendipitously.
Pharma companies are collaborating to boost the power of artificial intelligence (AI) in drug discovery by allowing access to proprietary structural data to train a large language model. Each of the partners is contributing data from several thousand experimentally determined protein:ligand interactions, creating one of the most diverse datasets and the richest chemistry assembled to date for model training.