One of the biggest challenges to the cell and gene therapy (CGT) landscape over the last couple of years has been the reduction in venture financing from $7.5 billion in 2021 to $3.3 billion in 2024. In the respiratory space, there has been very little development of CGT assets, with no approved therapy currently and only one product in an ongoing Phase III trial. Despite these challenges, an evolving CGT pipeline landscape for respiratory diseases, coupled along with ongoing research and development efforts within the wider CGT space, holds promise for this treatment modality, says GlobalData, a leading data and analytics company.   

According to GlobalData’s Pharmaceutical Intelligence Centre, the Phase III asset is TasDes-02 by Taskin Bioregeneration Co, which is being developed to treat acute respiratory distress syndrome. One promising pipeline asset is 4D-710 by 4D Molecular Therapeutics Inc (4DMT) to treat patients with cystic fibrosis (CF).

4D-710 is an aerosol gene therapy that works by using a customized adeno-associated virus (AAV) vector to deliver a healthy copy of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to lung cells. This process enables the lung cells to produce functional CFTR protein, correcting the underlying defect caused by the disease, and normalizing mucus production to ease CF symptoms. The vector is specifically designed to penetrate the sticky mucus in the lungs and efficiently deliver the gene, which is an artificial version called CFTRΔR.

In October 2025, 4DMT secured up to $11 million funding from the Cystic Fibrosis Foundation for the advancement of Phase II and readiness for Phase III, with the latter expected to begin in H1 2026.

Connor Daniels, Healthcare Analyst at GlobalData, comments: “There are many potential reasons for the slow development, including a lack of understanding of the underlying mechanisms driving different disease conditions, such as idiopathic pulmonary fibrosis, and patient heterogeneity, which makes it difficult to devise a CGT that works across multiple patients in the case of chronic obstructive pulmonary disease (COPD).”

Despite the challenges of previous years, there are still reasons to believe in CGTs. One such reason is the research and development efforts in automation and artificial intelligence (AI) from companies such as Made Scientific and Streamline Bio. In October 2025, the two companies announced a collaboration to accelerate AI-driven robotic manufacturing after recent validation work. What is particularly interesting is that this technology is designed to be flexible and work across equipment and methods.

Daniels adds: “These developments will help to overcome the manufacturing challenges CGTs currently face while also ensuring the quality of the product is maintained, ultimately driving down the cost of these therapies for patients and payers.”

Furthermore, the current US administration has signaled that it believes CGTs are aligned with their ‘Make America Healthy Again’ initiative, in a shared goal of targeting the underlying causes of diseases. As a result, the FDA recently outlined the ‘plausible mechanism pathway’ (PMP), which could offer a new way to market for CGTs.

The PMP looks to build on the success of the treatment of baby KJ, where CRISPR-based gene editing was used to treat CPS1 deficiency, by expanding this into a broader regulatory approach, but the details of this are still to be disclosed. Finally, China has introduced various initiatives to incentivize CGT research. For example, the country embraces investigator-initiated trials, which allow researchers to quickly obtain safety and efficacy data to ascertain the potential utility of CGT candidates.

Daniels concludes: “These initiatives help to accelerate the development of the CGT modality and, coupled with a high density of patients eligible for clinical trials and vast clinical resources, explains why the future for CGTs look promising.”