Respiratory Cell & Gene Therapy: Why Progress Lags — and What Could Change Next

Investment into cell and gene therapies (CGTs) has contracted sharply in recent years, dropping from $35.9bn in 2021 to $23.3bn in 2024 (GlobalData, 2025). Two major factors explain this pullback: low clinical success rates and persistent manufacturing bottlenecks that drive approved CGTs to very high prices — for example, about $462,000 for the CAR-T product Tecartus and roughly $4.25m for Lenmeldy (GlobalData Drug Pricing database). These pressures have shifted development activity toward the Asia–Pacific region, where CGT clinical trial counts reached 982 in 2025 compared with 904 in North America (GenScript Biotech Forum, 2025).

Current status in respiratory diseases

Progress in respiratory CGTs has been limited. As of 2025 there are no approved CGT therapies for respiratory diseases and only a single candidate has reached Phase III: TasDes-02 from Taskin Bioregeneration Co., being developed to treat acute respiratory distress syndrome (GlobalData Pharmaceutical Intelligence Center). The slow pace reflects scientific and clinical challenges, including incomplete understanding of disease drivers in disorders such as idiopathic pulmonary fibrosis and substantial patient heterogeneity in conditions like chronic obstructive pulmonary disease (COPD), which complicates the design of broadly effective therapies.

Notable pipeline programmes

One of the most advanced respiratory-focused CGT programmes is 4D Molecular Therapeutics' 4D-710 for cystic fibrosis (CF). 4D-710 is an inhaled gene therapy that uses a customized adeno-associated virus (AAV) vector engineered to penetrate viscous CF lung mucus and deliver an engineered CFTR construct (CFTRΔR) to airway cells. The goal is to restore functional CFTR protein production, correct the underlying defect, and normalize mucus clearance. In October 2025 4DMT announced up to $11m in support from the Cystic Fibrosis Foundation to advance late Phase II work and prepare for a Phase III trial planned to begin in H1 2026 (4DMT, 2025).

Manufacturing, regulation, and regional drivers

Manufacturing remains a critical bottleneck but innovations in automation and AI offer a pathway to scale and cost reduction. In October 2025 Made Scientific and Streamline Bio announced a collaboration to accelerate AI-driven robotic manufacturing after successful validation work; their approach is intended to be adaptable across equipment and workflows, which could improve consistency and lower production costs (Made Scientific, 2025).

Policy and regulatory developments are also shaping the field. U.S. regulators have signaled interest in alternative approval pathways for CGTs: the FDA's emerging 'plausible mechanism pathway' (PMP) aims to build on pioneering cases such as the CRISPR-based treatment used for a pediatric CPS1 deficiency, though detailed guidance is still forthcoming. Meanwhile, China has adopted measures to accelerate CGT research — including support for investigator-initiated trials that let researchers gather early safety and efficacy data quickly — combined with a large patient pool and substantial clinical capacity (Gao et al., 2025).

Outlook

Respiratory CGTs face steep scientific, clinical, and manufacturing hurdles, which help explain the slow progress to date. Nevertheless, targeted programmes like 4D-710, advances in AI-driven manufacturing, and evolving regulatory pathways offer realistic routes to accelerate development and reduce costs over time. Continued investment in basic disease biology, more efficient trial designs, and scalable production will be critical if CGTs are to reach broader patient groups in respiratory medicine.