University of Pennsylvania

New results show promise in treating Leber congenital amaurosis 1 (LCA1) with ATSN-101, an AAV5 gene therapy. Some patients experienced a 10,000-fold improvement in vision, with rapid improvements lasting at least 12 months. Published in *The Lancet*, the study highlights the potential of gene therapy for inherited retinal diseases.

New data from two clinical trials in 55 patients show nadunolimab's potential in treating patients who have progressed on pembrolizumab, with a median survival of 19.7 months and a disease control rate of 60%. The trials, CANFOUR and CIRIFOUR, highlight nadunolimab's efficacy in targeting immune suppressive cells in the tumor microenvironment.

Cantargia reports strong antitumor effects and encouraging median survival times in nadunolimab combination therapy trials involving 55 cancer patients previously treated with pembrolizumab. The data suggest nadunolimab's unique role in targeting immunosuppressive cells in the tumor microenvironment, with results to be presented at ESMO Congress 2024.

Newly updated COVID-19 vaccines, including mRNA vaccines from Moderna and Pfizer-BioNTech and the Novavax protein-based vaccine, are available across the U.S. The CDC recommends everyone age 6 months and older receive the updated vaccine, which targets the KP.2 strain of COVID-19. Vaccines offer protection against severe illness, hospitalization, and death, and help prevent long COVID. Individuals at higher risk, such as the elderly and immunocompromised, should get vaccinated promptly. The vaccine is free for those with insurance and through certain programs for uninsured individuals and children.

Researchers worldwide will gather in Philadelphia to discuss psychedelics, exploring their potential as modern treatments for PTSD, depression, and neurological disorders. The second annual PhilaDelic conference at the University of Pennsylvania, themed 'Paradigm Shifts in Psychedelic Studies,' aims to advance the field by sharing ideas and information.

A study led by Christos Davatzikos identifies 5 forms of cerebral atrophy linked to aging, detectable via machine learning. These patterns, influenced by biological traits and habits, predict dementia risk. Other research highlights inflammation's role in cognitive decline, suggesting exercise and tailored treatments as countermeasures. Overall, these studies aim to refine aging measurement and improve preventive actions.

UF scientists developed a gene therapy restoring vision in LCA1 patients, with a 10,000-fold light sensitivity improvement and successful navigation in a maze. The treatment showed a clean safety profile, paving the way for phase 3 trials and potential commercialization.

CCR West celebrates its 40th anniversary with a focus on CAR T-cell therapy and precision medicine, featuring cutting-edge research and presentations by leading experts in rheumatology.