Remodelins, a New Class of Compounds to Prevent Airway Remodeling and to Treat and Prevent Fibrosis in Multiple Organs

This technology includes a new class of compounds, called remodelins, which can be used to prevent airway remodeling and prevent lung fibrosis. Currently no effective therapies are available for lung fibrosis. This compound could also be employed as a treatment for asthma.

Remodelins, a New Class of Compounds to Prevent or Treat Cancer Metastasis or Glaucoma

This technology includes a series of small molecule organic compounds, called remodelins, that are synthetic derivative analogs of a parent compound discovered by screening of a Chembridge library. The novel synthetic derivative analogs were generated through multiple iterations of compounds directed by in vitro experiments. The invention also includes use of these or related molecules to treat cancer and/or glaucoma.

Murine Monoclonal Antibodies Effective To Treat Respiratory Syncytial Virus

Available for licensing through a Biological Materials License Agreement are the murine MAbs described in Beeler et al, "Neutralization epitopes of the F glycoprotein of respiratory syncytial virus: effect of mutation upon fusion function," J Virol. 1989 Jul;63(7):2941-2950 (PubMed abs). The MAbs that are available for licensing are the following: 1129, 1153, 1142, 1200, 1214, 1237, 1112, 1269, and 1243. One of these MAbs, 1129, is the basis for a humanized murine MAb (see U.S.

Systemic CRISPR Therapy for the Treatment of Inherited Diseases

This technology includes novel systemic adeno-associated virus (AAV)-mediated CRISPR gene therapy technology. While some diseases (e.g., retinal diseases) can be treated through local gene transfer, many diseases such as Duchenne Muscular Dystrophy (DMD) require systemic therapy. The CRISPR technology has two components, the Cas9 endonuclease, and the gRNA. To explore systemic CRISPR therapy, we co-delivered the AAV.Cas9 and AAV.gRNA vector to mdx mice, a mouse DMD model. Direct delivery to muscle yielded efficient gene correction.

Substituted Quinoline Analogs as Aldehyde Dehydrogenase 1A1 (ALDH1A1) Inhibitors

Aldehyde dehydrogenase enzymes (ALDHs) have a broad spectrum of biological activities through the oxidation of both endogenous and exogenous aldehydes. Unbalanced expression levels of ALDHs have been associated with a variety of disease states such as alcoholic liver disease, Parkinson’s disease, obesity, and multiple types of cancers. ALDH1A1 also plays a major role in preserving the tumor microenvironment via differentiation, self-protection, and proliferation of cancer stem cells.

Compounds and Methods for Blocking Transmission of Malarial Parasites

Malaria continues to be a life-threatening disease, causing roughly 241 million cases and an estimated 627,000 deaths in 2020, mostly among African children, although in 2020 nearly half of the world’s population was at risk of malaria. There is a big financial burden for antimalarial treatment; direct costs (for example, illness, treatment, premature death) have been estimated to be at least US $12 billion per year and the cost in lost economic growth is many times more than that.

Generation of Anti-TAT FXN Polyclonal and Monoclonal Antibodies to TAT Domain for Use in Quantitating or Detecting TATFrataxin (TAT-FXN) and Analogs

This technology includes a strategy to generate antibodies of rabbit origin, both polyclonal and monoclonal, which have strong affinity to the TAT sequence and which enable specific immunocapture or immunodetection of TAT containing frataxin and analogs for quantitative or qualitative assays. In addition, antibodies that react with the FXN region have also been generated with this strategy. The HIV virus encoded a translational activator protein containing a 12 amino acid domain which permits transmembrane delivery of any therapeutic protein containing the sequence.

Preparation of Benzene-1,4-disulfonamide Derivatives Useful as Therapeutic TRPML1 Receptor Modulators for the Treatment of Lysosomal Dysfunction and Membrane Repair Disorders

This technology includes a series of novel benzene-1,4-disulfonamides that activate TRPML1 receptor. The TRPML1 receptor is a lysosomal Ca2+ channel that has been shown to be involved in controlling lysosome functions, among then the maintenance of the integrity of the plasma membrane and the modulation of autophagosome-lysosome fusion. The improved ability of the receptor to deliver Ca2+ ions to the cytosol had been correlated with its capacity to modulate autophagy and lysosome exocytosis.