Therapeutic/Technology: Small molecule therapy

Protein tyrosine phosphatase – sigma inhibitors for hematopoietic regeneration

Research Objective We propose to develop a lead small molecule inhibitor of PTPσ, a receptor expressed by human blood stem cells, for the purpose of promoting human hematopoietic regeneration. Impact Systemic administration of a PTPσ inhibitor can accelerate hematologic recovery in thousands of patients who have received myelosuppressive chemo- or radiotherapy. Major Proposed Activities Generate […]

Use of Human iPSC-derived Endothelial Cells for Calcific Aortic Valve Disease Therapeutics

Research Objective To develop drugs to treat Calcific Aortic Valve Disease (CAVD), the third leading cause of adult heart disease, by screening a stem cell-based platform based on CAVD patient-derived stem cells. Impact CAVD represents a major unmet medical need, with no treatments other than valve replacement. We will identify drugs, already proven to be […]

Gingival mesenchymal stem cells as a novel treatment modality for periodontal tissue regeneration

Research Objective To develop a novel regenerative and adhesive hydrogel encapsulating patient's' gingival stem cell which can potentially be used as an adhesive dental hydrogel for periodontal tissue regeneration. Impact Upon successful completion, this project will introduce a promising treatment approach for maxillofacial defects presenting an innovative treatment modality for periodontal tissue regeneration. Major Proposed […]

A new phenotypic screening platform that identifies biologically-relevant targets and lead compounds for the treatment of Parkinson’s disease

Research Objective Demonstrate that our HitFinder™ library can be screened for phenotypic changes in A53T-IPSC-derived dopaminergic neurons and use a secondary handle to identify the targets responsible. Impact This technology combines phenotypic screening and target-ID eliminating the need to bias assays and/or screening libraries permitting application directly in iPSC-derived cells. Major Proposed Activities Prepare screening […]

Human Pancreatic Cancer Stem Cells: Developing a Novel Drug for Cancer Eradication

Research Objective We will use human pancreatic cancer (PC) stem cells to show 1 inhibits proliferation, self-renewal and cell viability. This paradigm is transformational for anti-cancer drug discovery for patients. Impact Pancreatic cancer (PC) kills >40,000/yr in the US. PC is a major unmet medical need. Use of PC stem cells in development of 1 […]

Reverse transcriptase inhibitors as a novel therapeutic approach for neurological autoimmune disorders

Research Objective We found that approved anti-retroviral drugs could stop inflammation and block neurodegeneration. We propose to validate the re-purpose efficacy of these clinically-approved retroviral drugs. Impact We have identified an unexpected cause to a brain inflammation and a potential simple treatment. Our research could help millions of patients affected by a broad range neuro-immunological […]

Neurogenic hydrogel stimulation of stem cells to regenerate radiation-damaged salivary glands

Translational Candidate Ceviginate is a neuromimetic encapsulated in a hydrogel Area of Impact Dry mouth as a result of injury to the salivary glands by radiation therapy for head and neck cancer Mechanism of Action Regenerate damaged salivary gland tissue through neurogenic stimulation of stem cells Unmet Medical Need Current treatment options for dry mouth/xerostomia, […]

Therapeutic development of Oxy200, an oxysterol with bone anabolic and anti-resorptive properties for intervention in osteoporosis

Translational Candidate A novel oxysterol with bone anabolic and anti-resorptive activity that will effectively and safely treat osteoporosis better than current options. Area of Impact Osteoporosis that results in bone fractures. Mechanism of Action The proposed candidate will target Mesenchymal Stem Cells in the skeleton to stimulate their differentiation into bone forming osteoblasts that will […]

A Splicing Modulator Targeting Cancer Stem Cells in Acute Myeloid Leukemia

Translational Candidate 17S-FD-895 is a potent small molecule splicing modulator that inhibits aberrant splicing in CSCs that have deregulated SF3B1 expression. Area of Impact Development of 17S-FD-895 could address a major bottleneck to reducing AML mortality by targeting splicing deregulated-CSCs that fuel AML relapse. Mechanism of Action 17S-FD-895 will positively impact patients with AML by […]

Evaluation of the Safety and Tolerability of KA34 in a Phase 1, Double-Blind, Dose Escalation Trial in Patients with Knee Osteoarthritis

Therapeutic Candidate or Device KA34 is an intra-articularly delivered small molecule therapeutic candidate which directs the differentiation of endogenous stem and progenitor cells Indication Osteoarthritis Therapeutic Mechanism KA34 promotes the differentiation of cartilage endogenous stem cells through increased chondrogenic gene expression to generate healthy chondrocytes. KA34 will potentially limit the progression of and / or […]