Recent research sheds light on molecular targets and novel treatments across oncology and rare diseases. UCLA scientists identified a protein IGF2BP3 that rewires leukemia metabolism and RNA regulation, offering therapeutic potential. Bristol Myers Squibb patented TLR7 agonists for cancer treatment. Lonza's Synaffix partnered with Qurient Therapeutics to develop a dual-payload antibody-drug conjugate (ADC), aiming to improve solid tumor therapies. Additionally, Youthbio Therapeutics advanced Alzheimer’s gene therapy candidate YB-002 following FDA feedback, underscoring continuing efforts in rare disease treatments.