ROCKEFELLER UNIVERSITY

Seth Lederman, co-founder, CEO & Chairman of Tonix Pharmaceuticals, is a physician, scientist, and entrepreneur with extensive experience in molecular immunology and autoimmune diseases. Steve Gardner, CEO of Precision Life, has over 30 years of experience in precision medicine, drug discovery, and computational biology, and is involved in genomics and AI-enabled drug discovery.

KFF Health News highlights recent health research: RNA interference therapy shows promise for chronic hepatitis B; beta-blockers may slow Huntington's disease progression; new obesity drug combo uses leptin; HPV vaccines linked to drop in cervical cancer deaths; prior fractures increase osteoporosis risk; chikungunya's global impact; men more likely to develop COVID-19 pneumonia.

Yun Zhong, PhD, joins Wedbush Securities as SVP, Equity Research, Biotechnology, initiating coverage on ASND, BMRN, KROS, MLTX, PASG, PTGX, RZLT, and VYGR.

Precision medicine uses biomarkers like blood tests and brain scans to improve Alzheimer’s diagnosis and treatment, potentially delaying onset by up to 20 years. Dr. Howard Fillit discusses the role of biomarkers, monoclonal antibody drugs, and lifestyle management in Alzheimer’s care, emphasizing the need for combination therapy and precision medicine to address the disease comprehensively.

A three-neuron circuit connects hunger-signaling hormone to jaw movements of chewing, identified by Rockefeller University researchers. BDNF neurons in the ventromedial hypothalamus, when inhibited, lead to overeating and chewing motions without food. Activation suppresses food intake and chewing, suggesting a reflex-like control of feeding.

Vertex Pharmaceuticals developed a therapy for cystic fibrosis, challenging low drug development odds. Their R&D strategy focuses on causal human biology, using human genomics and modality agnosticism to improve success rates, exemplified by FDA approvals for CF, sickle cell disease, and beta thalassemia treatments.

Emory researchers discovered an enzyme, CU43, effective in treating IgG-mediated pathologies like myasthenia gravis, requiring 4,000 times less dose than current drugs, potentially offering a more effective treatment option with fewer side effects.

Starting a biotech company involves navigating scientific validation, regulatory requirements, and fundraising challenges. Founders must choose the right focus, leverage university collaborations, attract investments, and build a strong team. Regulatory and IP strategies are crucial, and founders should be prepared for a long, challenging journey.

Nobel Prizes in physics, chemistry, and physiology or medicine to be announced, celebrating groundbreaking work. Predicting winners is challenging due to secrecy and collaborative nature of research. Notable potential winners include the human genome project team, GLP-1 drug developers, AI pioneers AlphaFold, gut microbiome researchers, and BRCA1 gene discoverer Mary-Claire King.