April 29 - Day 2Wednesday, 29 April 2026 - CT (China Time, GMT+08:00)

CREATE Medicines has developed a repeat-dose compatible in vivo CAR platform that selectively programs T, NK, and myeloid cells. Using this platform, CREATE achieved the first clinical response with an in vivo CAR in solid tumors.

This session will share the clinical and development playbook behind building and scaling a differentiated in vivo CAR platform from concept to human PoC.

• Development of robust LNP delivery systems
• Optimising LNP engineering to minimize off site delivery
• Overcoming scalability and reproducibility challenges
• Critical parameters influencing LNP manufacturing efficiency, consistency, and scalability
• Optimising LNP formulation design
• Analytical control strategy for LNPs
• Manufacturing innovation: Reducing cold-chain dependence & improving temperature stability
• Supply chain considerations for commercial viability
• Case Study: Successful LNP Scale-Up and steps towards in vivo cell therapies

Advanced cell, gene, and RNA therapies in Asia increasingly require a global‑first development strategy. This session examines how early scientific, regulatory, manufacturing, and access decisions before first‑in‑human studies impact global approval, reimbursement, and long‑term value. Attendees will gain a practical framework to design portable evidence, scalable supply chains, and globally viable development plans

A single gene Knock-out SPPL3 deletion remodels glycosylation in primary T cells, suppressing TCR-driven alloreactivity and host rejection while preserving anti-CD19 CAR-T effector function against tumours. In a Phase I investigator-initiated trial (IIT) involving B-NHL patients, this glycan-shielded approach demonstrated safety and durable persistence with potent tumour control. Preclinical proof of concept was also successfully established for solid tumour applications.

As the limitations of CAR-T therapies in solid tumors become increasingly evident, a new wave of innovation is emerging from the myeloid cell compartment. This session spotlights Signadori Bio, a Sofinnova-backed biotech born from a strategic partnership with Gustave Roussy, Europe’s leading cancer center. The company is pioneering a gene-modified monocyte-derived macrophage in vivo platform designed to remodel the tumor microenvironment and overcome resistance mechanisms in solid cancers. Attendees will gain insight into the scientific rationale, translational strategy, and in vivo delivery roadmap behind Signadori Bio’s lead program. This case study offers a compelling look at how next-generation in vivo platforms are being built from the ground up to address the unmet needs in oncology.

Systemic administration of recombinant adeno-associated virus (rAAV) vectors carrying therapeutic payloads with broad distribution in the central nervous system (CNS) provides promising treatments for many neurological diseases. Some engineered AAV variants have been demonstrated to penetrate blood-brain barrier (BBB) in mice but not in non-human primates. We developed a Receptor-targeting AAV (RtAAV®) capsid engineering platform for efficient CNS transduction and cross-species translatability.

• Genetic medicine in China
• Gene therapy by AnlongBio
• siRNA development by AnlongBio

• Lessons learnt on CMC reviews, inspections and common challenges for submissions
• What to watch out for?
• Practical experience of fast track pathways, priority reviews and initiatives to shorten CTA review timelines
• Case studies