The global CGT therapeutics space is transitioning from a niche, high-complexity segment into a mainstream, high-growth therapeutic class. Driven by scientific breakthroughs, accelerated regulatory pathways, and the entry of next-generation platforms—including in vivo CRISPR gene editing, allogeneic “off-the-shelf” cell therapies, and automated smart-factory bioprocessing—the landscape is expected to cross $50 billion by 2030, reflecting >30% CAGR between 2024 and 2030. This growth aligns with the expanding global disease burden: oncology indications continue to dominate CGT development, while rare genetic, neurological, hematologic, and ophthalmologic disorders fuel diversification into new high-value segments.

Pipeline momentum is unprecedented. In 2025, oncology remains the largest therapeutic focus, with hematologic malignancies accounting majority of trials and solid-tumor programs accelerating through improved tumor-microenvironment targeting and next-generation vectors. Rare diseases continue to anchor the CGT landscape—over two-thirds of rare-disease trials target rare cancers, while new programs are broadening into genetically linked common diseases such as Alzheimer’s, cardiovascular disease, arthritis, and HIV. Approvals of CASGEVY™, Lyfgenia™, Elevidys™, Ryoncil™, Hemgenix®, Roctavian®, Vyjuvek®, and emerging CNS and ophthalmology therapies demonstrate durable evidence that ex vivo and in vivo modalities offer curative potential across multiple disease categories.

• What are the major scientific and operational inflection points reshaping the sector, and how is in-vivo gene editing rapidly progressing with vast growth potential?
• In what ways do parallel advances in automated manufacturing (e.g., Cellares Cell Shuttle™ receiving FDA AMT designation) and the rise of regional “IDMO Smart Factories” in the US, EU, and Japan signal a decisive move toward scalable, closed system, digitally orchestrated CGT production?
• Why are factors like manufacturing readiness (MRL), AMT-qualified platforms, and cloud-enabled QA becoming as important as clinical data for asset valuation, and how will they create new growth avenues?