Disease Focus: Bone or Cartilage Disease


A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients with Mucopolysaccharidosis Type 1

A Phase I Clinical Trial for a Lentiviral Gene Therapy Targeting the TCIRG1 Gene for Infantile Malignant Osteopetrosis (IMO)

Rocket Pharmaceuticals is conducting a clinical trial using a gene therapy for infantile malignant osteopetrosis (IMO), a rare and life-threatening disorder that develops in infancy.  IMO is caused by defective bone cell function, which results in blindness, deafness, bone marrow failure, and death very early in life.  The trial will use a gene therapy that targets IMO caused […]

The role of WNT and BMP signaling pathways in iPSC to iTenocyte step-wise differentiation for tendon repair

Research Objective Development-inspired differentiation will enable efficient and specific generation of tenocytes that can repair tendon injury, restore dysfunctional tissue, and prevent long term effects. Impact This study will eliminate heterogenous differentiation of pluripotent stem cells and will results in high yield and unified tenogenic phenotype. Major Proposed Activities Establish the mechanism by which Wnt […]

Pluripotent Stem Cells for Tendon Tissue Engineering

Research Objective We propose to develop a bio-tendon engineered from differentiated pluripotent stem cells for the repair of tendon injuries and degeneration. Impact Rotator cuff tears are the most common causes of shoulder pain that require surgery. However, failure rates range from 20% to 90%. A successful tendon repair will have a major impact on […]

Microgel encapsulated iPSC-derived notochordal cells to treat intervertebral disc degeneration and low back pain

Research Objective We aim to discover an injectable, rejuvenating treatment for painful intervertebral disc degeneration using microtissue-encapsulated iPSC-derived notochordal cells (iNCs) using large animal model Impact Our treatment candidate may allow for a non-invasive stem cell therapy, targeting the underlying pathogenesis of intervertebral disc degeneration, the leading cause of chronic back pain in adults. Major […]

Meniscal Repair and Regeneration

Research Objective Stem cells are seeded into fibers spun out of collagen to fabricate tissue that resembles the knee meniscus Impact Meniscal tears are very common but do not heal. The treatment is removal of the torn tissue, which leads to osteoarthritis. If successful, replacing the tissue will prevent osteoarthritis. Major Proposed Activities Establish the […]

A Novel Therapy for Articular Cartilage Autologous Cellular Repair by Paste Grafting

Research Objective Articular paste graft containing MSCs and an adhesive hydrogel that support cartilage growth will be combined for an effective and functional stem cell based cartilage repair procedure. Impact The proposed biologic cartilage repair therapy results in accessibility of an effective, low cost, one-step and functional biologic solution to those with cartilage injuries and […]

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 […]

IVD rejuvenation using iPSC-derived notochordal cells

Research Objective To identify a new therapeutic agent for disc regeneration using novel pluripotent stem cells and injectable beads that support differentiation and provide biomechanical strength. Impact If this study is successful, we will be able to bring completely new biologically and biomechanically relevant solution to degenerated intervertebral discs. Major Proposed Activities  To optimize stem […]

Noncoding RNA drug TY1 as a therapeutic candidate for scleroderma and systemic sclerosis

Translational Candidate Modified synthetic noncoding RNA molecule Area of Impact Systemic Sclerosis Mechanism of Action The mechanism of action of TY1 is alleviating cell stress and damage through enhancing genes that alleviate cell stress which, in turn, control inflammation and fibrosis in diseases tissue. Unmet Medical Need Systemic sclerosis is an incurable disease with no […]

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