SPARK Projects

Anti-infective

  • sk05029
    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.
  • sk09098
    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.
  • sk10121
    Novel broadly-protective influenza vaccine. Currently optimizing protein folding and developing a cold-chain independent production scheme. Plan ferret testing within a year.
  • sk06046 Gastroenterology 
    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.

Cardiovascular

  • sk09111
    Novel target to treat heart failure through agonist activity. Proof-of-concept animal studies supported link between target signaling and cardiac function.

Dermatology

  • 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.
  • sk07081
    Small molecule inducer of melanin production/tanning response as therapy for xeroderma pigmentosum (XP), vitiligo, erythropoietic protoporphyria (EPP).  

Gene therapy

  • sk10134
    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.

Immunosuppression/
Allergy

  • 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.

Immunology/
Hematology

  • sk08095
    Combination therapeutic monoclonal antibody plus engineered immune cells to treat acute gastrointestinal graft versus host disease (GVHD) (orphan disease).
  • sk10125
    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.

Metabolic

  • sk04019
    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.

Muscle Atrophy

  • 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).

Neurology

  • 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).
  • sk07065
    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
    Novel small molecule modulators 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
    Novel small molecule to 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.

Oncology

  • sk03014
    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.
  • sk04021
    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.