ESGCT 2025 at a glance
Ahead of ESGCT 2025, LucidQuest outlines the high-impact developments expected in gene and cell therapy and the expanding influence of AI across the field.
📅 Build your schedule around the topics that interest you. 📥 Download the ESGCT 2025_Preview_by_LucidQuest
Dive deeper
Key Topics From Scientific ESGCT 2025 Presentations
Liver-Directed & Metabolic Gene Therapies
- OTC-HOPE, ReGenHeart II, LY-M003, GNT-012-CRIG; anticipated LTFU and refined AAVs for Wilson’s, OTC, Crigler–Najjar safety–efficacy.
Ocular & Sensory System Gene Therapies
- Laru-zova DAWN, PRODYGY RdCVF, CTx001 for AMD expected; AAVs for DFNB1A, Stargardt refine subretinal vs intravitreal delivery.
Central Nervous System & Neurodegenerative Disorders
- NGN-401 for Rett, MECP2 editing; Alzheimer’s, Wolfram, Angelman emphasize CNS biodistribution and one-time dosing.
Hematopoietic Stem Cell & Inherited Hematologic Disorders
- OTL-203, ARTEGENE I/II, RAG1-SCID show engraftment; MPS IVA, α-mannosidosis models improve VCN control and conditioning.
Neuromuscular & Musculoskeletal Disorders
- GEN6050X base editor, Optidys dual-AAV, LMNA-CMD lead; TN-401 and α-Klotho strategies improve muscle; GRMD durability guides dosing.
Cardiovascular, Renal & Metabolic Disorders
- CBN-1100, NVC-001, FGF21; AAV9 cardiomyopathy and renal programs enable precision dosing and first-in-class interventions.
Oncology, CAR-T & Oncolytic Virus Therapies
- AloCELYVIR Ib, TILT-123, HER2 sdAb-CAR-T; AI-enhanced CARs and dual-payload OVs heighten selectivity and persistence.
Emerging Technologies & Non-Viral Delivery
- FiCAT, Megabulb DNA, UCOE; LNPs, EVs, programmable RNA enable modular, repeat-dose gene and mRNA therapeutics.
Manufacturing, Platforms & Analytical Innovation
- iCellis500, SamuxMP, Recombumin; capsid analytics, digital PCR, GMP automation drive scalable, compliant production.
Regulatory, Access & Clinical Development
- Join4ATMP, Holoclar IV, EMA–FDA harmonization; equitable inclusion, safety registries, adaptive licensing accelerate access.
Artificial Intelligence and Machine Learning at ESGCT 2025
AI-Enabled Vector Engineering
- Data-driven capsid/LNP design to improve tropism, immune evasion, yield.
Precision Promoter & Payload Design:
- PromoterGPT to speed cell-type promoters, tighten expression control, minimize off-target transcription.
AI-Integrated Manufacturing & Analytics:
- Holographic imaging + LLM knowledge-graphs for predictive bioreactor control and real-time QA.
AI-Optimized Delivery Platforms:
- AI-guided LNP/polymer nanoparticles for siRNA/mRNA (e.g., macrophage modulation, CFTR restoration).
Generative AI in Capsid Innovation:
- GMU037 predicted dual-fitness capsids with superior ophthalmic transduction and high-yield production.