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Chronic Lung Disease (COPD, Severe Asthma, Pulmonary Fibrosis and Cystic Fibrosis)

While there are increasing number of new inhalers introduced over several decades for treating COPD patients, there has been little innovation by the pharmaceutical industry in offering life-changing and novel therapies for patients suffering from chronic lung disease.  There are 3 class of inhaled agents (inhaled corticosteroids, anti-cholinergic bronchodilators and beta agonist bronchodilators), which have not changed for decades. While these inhalers may improve exercise tolerance and decrease COPD exacerbations, there is no evidence that these inhalers increase patient survival and reverse or repair the pathological changes of COPD. Also, inhalers have almost little activity in patients with emphysema.  Similarly, there are no regenerative medicine treatments for other lung diseases like pulmonary fibrosis and Cystic Fibrosis.

Conditions like emphysema, pulmonary fibrosis and acute respiratory distress syndrome (ARDS) primarily target alveoli and cause alveolar damage and failure.  Regenerative medicine is the only treatment modality that has the potential for protecting, repairing and replacing damage alveolar cells.  Unfortunately, the pharmaceutical industry has failed to take a leadership role in regenerative medicine directed towards respiratory diseases.  Also, there has been little effort at NIH and private foundations to accelerate regenerative medicine research for respiratory disease.  Thus, the Institute has considered respiratory disease as a major unmet medical need.

The major research focus for the Institute centers on how to protect, repair and regenerate alveoli, which is the fundamental unit where gas exchange occurs.  The Institute’s research program on alveolar regeneration is outlined below:

A. Alveolar protection and repair:

1. Creating pipelines of postnatal stem cells and microvesicles for in vivo testing.

2. Integrating synthetic biology modifications in stem cells to augment therapeutic efficacy


B. Alveolar cell replacement:

1. Creating clinical-grade virus-free and oncogene-free induced pluripotent stem cells (iPSC).

2. Synthetic biology to optimize immune-tolerance of iPSC.

3. CHO cell-based bioprocessing technology to produce growth factors for lung-specific differentiation.

4. Lung organoid and encasement technology.

C. In vivo testing:

  1. Animal safety and efficacy.

  2. Physiological testing.

  3. Advanced imaging analysis.

  4. Histological analysis.

The final goal of this research program is to obtain an Investigative New Drug (IND) license from the Food and Drug Administration to initiate Phase I/IIA clinical trials.

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