Cell and gene therapy in respiratory diseases: current landscape and future prospects

The cell and gene therapy (CGT) sector has faced a notable decline in venture capital investment over the past few years, dropping from $35.9 billion in 2021 to $23.3 billion in 2024 (GlobalData Webinar - Cell & Gene Therapies: Current & Future Landscape, 2025). This downturn is largely due to two key issues: the low success rates in clinical trials and the production challenges that arise once therapies gain approval. As a result, CGTs remain extremely costly on the market, ranging from $462,000 for the single-dose CAR-T therapy Tecartus to $4.25 million for Lenmeldy, a one-time treatment for metachromatic leukodystrophy, according to GlobalDatas Drug Pricing (POLI) database.

The shift in CGT development is moving away from the US and EU toward the Asia-Pacific region, where the number of clinical trials (982) surpassed North America (904) in 2025 (GenScript Biotech Forum, 2025). In the respiratory therapy domain, progress has been minimal, with no approved CGTs currently and only one therapy in Phase III trials. TasDes-02 from Taskin Bioregeneration Co is the sole asset under investigation for acute respiratory distress syndrome.

Slow advancement in respiratory CGTs can be attributed to multiple factors, such as limited understanding of disease mechanisms like idiopathic pulmonary fibrosis and the variability among patients, which complicates the creation of broadly effective therapies for conditions like chronic obstructive pulmonary disease (COPD). Among emerging candidates, 4D-710 from 4D Molecular Therapeutics Inc (4DMT) shows promise for cystic fibrosis (CF). This aerosol gene therapy employs a tailored adeno-associated virus (AAV) vector to deliver a functional copy of the CFTR gene to lung cells, restoring proper CFTR protein production and improving mucus clearance. The vector is engineered to penetrate thick lung mucus, delivering an artificial gene variant called CFTRR. In October 2025, 4DMT secured up to $11 million from the Cystic Fibrosis Foundation to support Phase II progress and prepare for Phase III, scheduled for H1 2026 (4DMT, 2025).

Despite recent challenges, optimism remains for CGTs, driven by advances in automation and artificial intelligence. Companies like Made Scientific and Streamline Bio announced a collaboration in October 2025 to accelerate AI-enabled robotic manufacturing, with technology adaptable to different equipment and methods (Made Scientific, 2025). These innovations aim to streamline production, maintain product quality, and reduce therapy costs.

Government support also plays a role. The US administration has emphasized that CGTs align with the Make America Healthy Again initiative, focusing on addressing disease causes. Consequently, the FDA has introduced the plausible mechanism pathway (PMP), inspired by the CRISPR-based treatment of baby KJ with CPS1 deficiency, offering a potential new regulatory route for CGTs, though specifics are still pending.

China has implemented policies to promote CGT research, including investigator-initiated trials, enabling rapid acquisition of safety and efficacy data to evaluate candidate therapies (Gao et al., 2025). Coupled with a high patient population and extensive clinical resources, this approach positions China as a central hub for CGT development.

Analysis of the Current State of the Cell and Gene Therapy Sector

The cell and gene therapy (CGT) sector is currently experiencing significant challenges, primarily driven by the declining investment and increasing complexity of development. Venture capital funding has sharply fallen from $35.9 billion in 2021 to $23.3 billion in 2024, reflecting investor concerns over the sector’s viability. This decline can be attributed to two main issues: the relatively low success rates of clinical trials and the expensive production costs of therapies once they gain approval. The high costs of CGTs, such as Tecartus priced at $462,000 and Lenmeldy at $4.25 million, highlight the financial burden associated with these treatments, making them unaffordable for many patients despite their potential benefits.

Moreover, the geographical landscape of CGT development is shifting, with increasing clinical trials taking place in the Asia-Pacific region. As of 2025, this region has surpassed North America in the number of trials, signaling a shift in focus toward areas with less regulatory pressure and potentially more favorable market conditions. However, the respiratory therapy segment has seen minimal progress, with only one therapy under Phase III trials and none currently approved. The slow pace of development can be attributed to limited understanding of disease mechanisms and the challenge of developing therapies that work across a wide range of patient profiles.

Nonetheless, there are reasons for cautious optimism. Innovations in automation and artificial intelligence (AI) are emerging as key drivers to overcome production challenges. Collaborations like the one between Made Scientific and Streamline Bio aim to streamline manufacturing processes through AI-enabled robotic systems, potentially lowering costs and increasing production efficiency. Furthermore, government support in the form of new regulatory pathways, such as the FDA's “plausible mechanism pathway” (PMP), could accelerate the approval process for CGTs, though the details of these mechanisms are still developing.

In addition, China’s proactive approach to CGT research, with policies supporting investigator-initiated trials and rapid data acquisition, positions the country as an important hub for future developments. The combination of a large patient population, government backing, and extensive clinical resources strengthens China’s role in advancing CGT therapies.

In conclusion, while the CGT sector is currently facing challenges, including declining investments and slow therapeutic progress, innovation in manufacturing technologies and supportive government policies could provide a pathway to overcoming these obstacles. The sector's long-term viability will depend on successfully addressing the high costs of therapies and improving clinical trial success rates, alongside leveraging new opportunities in emerging markets.

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• Cell and gene therapy landscape in respiratory diseases and future outlook