BAYLOR COLLEGE OF MEDICINE

Baylor College of Medicine's Dr. David Szafron explains that bloating, a feeling of trapped gas, can be triggered by food, medications, gut bacteria imbalances, and more. Common food triggers include lactose and fructose. Regular bloating may require a food diary to identify triggers, while severe symptoms like weight loss or vomiting warrant a doctor's visit.

The FDA granted accelerated approval for Kebilidi, a gene therapy for AADC deficiency, a rare genetic disorder causing severe disability. Developed by PTC Therapeutics, Kebilidi is the first U.S. gene therapy directly administered to the brain, delivered via a stereotactic surgical procedure. It showed efficacy in a trial with 13 pediatric patients, with 8 meeting new gross motor milestones, compared to none in an untreated cohort. Common adverse reactions include dyskinesia and procedural complications.

ClearPoint Neuro receives FDA De Novo marketing authorization for its SmartFlow Neuro Cannula, the first device approved for direct brain gene therapy delivery, specifically for AADC deficiency treatment with PTC Therapeutics' KEBILIDI™, marking the first commercially available neuro gene therapy in the U.S.

Tong Huo discusses CAR-T therapy's advantages, next-gen improvements, gene-editing enhancements, and potential applications beyond oncology, emphasizing Sino Biological's contributions.

FDA granted orphan drug designation to Capsida’s CAP-002, a gene therapy for STXBP1-related developmental and epileptic encephalopathy. CAP-002 aims to correct disease pathology via a single infusion, with an IND expected in H1 2025. Capsida also developing CAP-003 for Parkinson disease with GBA mutations, showing significant GCase protein and activity increases in NHP studies.

The Phase III VANCE trial, a major T cell therapy clinical trial for solid tumours, showed improved survival in nasopharyngeal cancer patients treated with chemotherapy and EBV-specific cytotoxic T cells. The study highlights the need for further optimization of T cell therapies.

The VANCE trial, the largest T cell therapy clinical trial for solid tumors, demonstrates Singapore's capability in large-scale global cell therapy. Published in 'Annals on Oncology' (2024), it shows no overall survival improvement but highlights potential for subgroup refinement and biomarker identification.

Largest T cell therapy trial for solid tumours, led by Singapore, showed no overall survival benefit but better outcomes in US, Singapore, and Taiwan sites. Identifying biomarkers could enhance therapy success.

The VANCE trial, a multinational phase III study led by Singapore, concludes that T cell therapy for solid tumours is safe but requires refinement to improve outcomes. The trial, published in Annals on Oncology, involved 330 patients across 23 sites, showing no overall survival benefit but potential in specific subgroups. Future efforts aim to identify biomarkers for personalized T cell therapy.

Stock analysts predict significant shifts in biopharma due to Trump's election and Robert F. Kennedy Jr.'s potential role in healthcare, focusing on vaccine regulation and FDA leadership changes. Moderna's stock declines despite Q3 earnings, reflecting investor concerns over vaccine sentiment. Smaller biotechs may benefit from a more favorable M&A environment under Trump's administration.