A year out from Leqembi’s approval for Alzheimer’s disease (AD), ongoing research coupled with artificial intelligence is advancing both radiopharmaceuticals and small-molecule drugs for AD diagnostics and treatment, speakers at the 2024 KoNECT-MOHW-MFDS conference said.

Gene editing strategies, from epigenetic engineering to cell reprogramming and genetic vaccines, are accelerating the development of new therapies that awaken the immune system to treat cancer, as presented last month in Rome at the 31st Annual Congress of the European Society of Gene and Cell Therapy (ESGCT). Some of these advances are taking advantage of the conditions of the tumor microenvironment, where cancer cells coexist with immune cells, microorganisms and blood vessels.

David Baker, Demis Hassabis and John Jumper share the 2024 Nobel Prize in Chemistry for their contributions to the science of protein structure. David Baker was awarded half the prize “for computational protein design,” according to the Royal Swedish Academy of Sciences. Hassabis and Jumper shared the other half “for protein structure prediction.”

David Baker, director of the Institute for Protein Design at the University of Washington School of Medicine, is a pioneer in protein design. His contributions have been recognized with countless awards, and now, a place among the 2024 Clarivate Citation Laureates. Baker’s lab has developed several open-source software applications for nanotechnology and biomedicine. With these methods, scientists build new proteins that bind to drug targets and block them or activate cellular signals.

A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.

A strategy inspired by deficient HIV replication could be used as a treatment to reduce viral load in patients living with HIV and help control the pandemic of the retrovirus. Scientists from the University of California San Francisco want to use HIV against itself by using a parasitic version of the pathogen.

Infection or cure? Scientists from Tel Aviv University and the University of Glasgow genetically modified the Toxoplasma gondii to bring a protein inside neurons. The novelty of using a protozoan that can travel from the gut to parasitize the CNS contrasts with the possibility of causing a disease. The scientists are already working on how to avoid it.

Patients with congenital hearing loss could benefit from a gene therapy currently in development. Although there are approaches that could reverse the process in children and young people before it becomes severe, so far, adults do not have any treatment that prevents the progressive deterioration of auditory sensory cells caused by this disease.

Scientists at the University of Washington have engineered human plasma B cells modified to express long-lasting bispecific antibodies that could be used to treat leukemia without requiring continuous dosing.