Abstract: This application describes methods of treating alpha-1 antitrypsin deficiency (AATD) comprising administering Compound I, a deuterated derivative thereof, and/or a pharmaceutically acceptable salt thereof. The application also describes pharmaceutical compositions comprising Compound I, a deuterated derivative thereof, and/or a pharmaceutically acceptable salt thereof.

Abstract: Modulators of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR), their pharmaceutical compositions, and methods of treating cystic fibrosis in patients with residual function mutations.

Abstract: Devices, compositions, and methods for achieving non-surgical lung volume reduction (e.g., bronchoscopic lung volume reduction (BLVR)) are described. BLVR can be carried out by collapsing a region of the lung, adhering one portion of the collapsed region to another, and promoting fibrosis in or around the adherent tissue.

Abstract: One aspect of the present invention relates to compositions and methods comprising polyelectrolyte molecules for treating patients who have certain diseases. Aspects of the invention relate to using certain polyelectrolyte compositions in therapy. According to the invention polyelectrolyte compositions may be used, for example, to slow or stop cell growth, kill cells (e.g., via necrotic or apoptotic pathways), promote fibrosis, or a combination thereof. In one aspect of the invention, certain toxic (e.g., cytotoxic) properties of polyelectrolytes are exploited for therapeutic purposes. In certain embodiments, compositions and methods of the invention are used to target polyelectrolyte toxicity to predetermined regions within a subject, while minimizing undesirable toxicity at other regions with the subject. In certain embodiments, the present invention relates to lung-volume-reduction therapy using a polyelectrolyte composition.

Abstract: The invention includes methods for performing non-surgical lung volume reduction in a patient by (a) administering, through the patient's trachea, a composition comprising an enzyme (e.g., a protease, such as a serine protease (e.g., trypsin, chymotrypsin, elastase, or an MMP), and/or a detergent, and/or a polycation; and (b) collapsing a region of the lung, at least a portion of which was contacted by the composition administered in step (a). The patient can have COPD (e.g., emphysema) or their lung can be damaged by a traumatic event. The tissue in the targeted area can also include an abscess or fistula. One can similarly treat other tissues (i.e., non-lung tissues) by exposing those tissues to an enzyme-containing composition (or other composition described herein). These tissues may be those that are obscured from a therapeutic agent by epithelial cells or that will contact an implantable device.

Abstract: Devices, compositions, and methods for achieving non-surgical lung volume reduction (e.g., bronchoscopic lung volume reduction (BLVR)) are described. BLVR can be carried out by collapsing a region of the lung, adhering one portion of the collapsed region to another, and promoting fibrosis in or around the adherent tissue.

Abstract: The invention includes methods for performing non-surgical lung volume reduction in a patient by (a) administering, through the patient's trachea, a composition comprising an enzyme (e.g., a protease, such as a serine protease (e.g., trypsin, chymotrypsin, elastase, or an MMP); and (b) collapsing a region of the lung, at least a portion of which was contacted by the composition administered in step (a). The patient can have COPD (e.g., emphysema) or their lung can be damaged by a traumatic event. The tissue in the targeted area can also include an abscess or fistula. One can similarly treat other tissues (i.e., non-lung tissues) by exposing those tissues to an enzyme-containing composition (or other composition described herein). These tissues may be those that are obscured from a therapeutic agent by epithelial cells or that will contact an implantable device.

Abstract: The present invention features compositions and methods for treating emphysema by reducing the amount of force the fibers in the lung (e.g., the collagen and elastin fibers in the walls of the alveoli) must bear. More particularly, in one embodiment, the invention features a pharmaceutically acceptable composition comprising a lipid that, when applied to an enlarged alveolus (e.g., an alveolus having a diameter substantially larger than (e.g., 5, 10, 20, 50, or 100% or more than) the average alveoli in a healthy patient (i.e., a patient with no discernable lung disease), exerts a surface tension within the alveolus that substantially reduces the stress on fibers within the alveolus when inflated by a normal inspiration. The composition can display a &ggr;* of about 30 to about 70 dynes/cm.