Novel aminoglycoside antibiotic based on new understanding of ototoxicity. Leads show broad-spectrum antimicrobial activity and substantially less ototoxicity and nephrotoxicity in animals.
sk06050 Leishmaniasis & Chagas Disease (Global Health) Peptide therapeutics against pathogen-specific targets in Leishmania spp and T. cruzi. Animal models show a reduction in parasitemia and increased survival after treatment.
Novel small molecule broad-spectrum antivirals targeting host processes. Expect reduced emergence of viral resistance. Testing in animal models of Dengue, West Nile and Ebola viruses, as well as other flavi- and filoviruses.
Novel broadly-protective influenza vaccine. Currently optimizing protein folding and developing a cold-chain independent production scheme. Plan ferret testing within a year.
Small molecule non-antibiotic treatment for Clostridium difficile colitis that preserves normal gut microbiota. Protective in mouse model of C. diff infection. Human safety data available.
sk09111 Novel target to treat heart failure through agonist activity. Proof-of-concept animal studies supported link between target signaling and cardiac function.
sk06056 Microneedle delivery of protein replacement to areas at risk of damage and wound margins in patients with dystrophic epidermolysis bullosa (orphan disease). Uses novel expression method to boost protein production yield.
Small molecule inducer of melanin production/tanning response as therapy for xeroderma pigmentosum (XP), vitiligo, erythropoietic protoporphyria (EPP).
Innovative platform to enhance Adeno-associated virus (AAV)-based gene therapy. Identified a host factor to improve AAV infection for all serotypes and cell types.
sk05034 Inhibitor of IgE-induced allergic response for severe asthma and other type I hypersensitivity responses. Developing biologic leads and conducting fragment-based screen for small molecule inhibitors.
Combination therapeutic monoclonal antibody plus engineered immune cells to treat acute gastrointestinal graft versus host disease (GVHD) (orphan disease).
Blocking monoclonal antibody against a novel immune target to prevent graft versus host disease (GVHD) (orphan disease). Evaluating two targets, one of which may also improve engraftment of donor cells.
Novel transdermal formulation of a repurposed small molecule to treat or prevent diabetic skin ulcers. Animal studies showed improved wound healing and histology.
sk08096 Small molecule inducer of beta cell regeneration for treatment of type 2 diabetes. Lead compound optimization on-going.
sk08091 Novel targets for induction of brown fat from white fat to treat obesity. Preparing assay for high-throughput screening to identify small molecule inhibitors.
sk06052 Novel biologic to promote stem cell differentiation into skeletal muscle. Also increases insulin sensitivity. Application in a variety of myopathy/sarcopenia disorders (several orphan diseases).
Novel biologic to promote stem cell differentiation into skeletal muscle. Also increases insulin sensitivity. Application in a variety of myopathy/sarcopenia disorders (several orphan diseases).
Novel pathway to control Huntington’s disease progression. Target knockdown showed decreased severity and delayed onset of disease in an aggressive mouse model of Huntington’s disease. Exploring SAR analysis around a repurposed molecule.
sk09097 Novelsmall moleculemodulators to improve learning and memory in Alzheimer’s patients. First generation repurposed molecule showed improvement in mouse models, will enter clinical trial in 2016.
sk09104 Novelsmall moleculeto treat neurodegenerative diseases by modulating microglia. Animal efficacy in Alzheimer’s, Parkinson’s, and Amyotrophic Lateral Sclerosis (ALS) animal models of disease.
sk09114 Novel small molecule plus companion imaging agent to detect and treat Alzheimer’s Disease by regulating toxic neuroinflammation. Developing high-throughput screening for small molecule inhibitor and PET imaging agent in animal studies.
sk10126 Novel small molecule biased agonists of the mu opioid receptor. Animal studies show lead has reduced abuse potential and fewer respiratory and GI side effects.
Repurposed oral small molecule as a treatment/ radiation sensitizer for prostate cancer. Positive human pilot study with IV route; enrolling in Phase-Ib oral trial. Not approved in USA.
Novel small molecule treatment for mixed-lineage leukemia (orphan disease) with large therapeutic window in patient samples and significant lifespan extension in animal model of disease. Identified by phenotypic screen; target has been identified.
sk06054 Peptide targeting a novel cancer scaffold protein. Promising proof-of-concept in multiple animal cancer models. Application in numerous cancers, including pancreatic. Currently developing lead and testing mechanisms of resistance.
sk08060 Novel small molecule inhibitor of an oncogene target for acute myeloid leukemia (AML) (orphan disease). Target biochemically validated in animal model and patient cells.
sk08077 Novel target of the mTOR pathway to treat a wide range of cancers. First indication: lymphangioleiomyomatosis (LAM) (orphan disease).
sk09105 Repurposed small molecule to prevent cancer emergence in patients with inherited BRCA-1 mutants (orphan disease). Establishing biomarkers and in consultation with FDA about trial endpoints. Good drug safety record, but never approved in USA.
sk09109 Next generation therapeutic vaccine for glioblastoma (orphan disease). Have identified several candidates with superior activity to the first generation vaccine currently in clinical trials.
sk10127 Novel small molecules to treat relapsed Estrogen-Receptor-positive breast and colorectal cancer. Hits discovered with a novel assay of a known cancer target. Could also be combined with cancer immunotherapies.
sk10128 Topical formulation of a small molecule plant extract to prevent radiation dermatitis during cancer treatment. Currently in preclinical testing.
sk04024 Blood test for diagnosis of allergies and for monitoring response to desensitization therapy. Has undergone clinical validation study.
sk08078 Diagnostic predictor for risk of Kawasaki Disease in children for use in combination with in silico tool. Validating biomarkers in patient samples.
sk09113 Diagnostic combining biomarkers from blood and stool to predict feeding intolerance in neonates and to diagnose necrotizing enterocolitis (NEC). Stool diagnostic may also be predictive of irritable bowel syndrome in adults.
sk06064 Novel, low cost diagnostic to rapidly detect Mycobacterium tuberculosis from sputum using a novel cell imprinting technique and traditional staining protocols. Preparing for clinical validation trial.
Inexpensive, point-of-care diagnostic to measure chemotherapy-induced hyperammonemia (“chemo brain”) and inborn errors of ammonia metabolism.