Project Objective: Pre-IND/IDE or Equivalent

Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells for Pulmonary Arterial Hypertension

Translational Candidate Autologous MPO Knock-Out Hematopoietic Stem and Progenitor Cells Area of Impact Pulmonary Arterial Hypertension (PAH), initially associated with Scleroderma (Systemic Sclerosis -SSC), and then applied to other causes of PAH Mechanism of Action Myeloperoxidase (MPO) protein produced by neutrophils plays a critical role in the development of PAH. Disrupting the MPO gene in […]

Optogenetic therapy for treating retinitis pigmentosa and other inherited retinal diseases

Translational Candidate Ray-001 is an AAV gene therapy delivering a light sensitive gene to treat patients with advanced Retinitis Pigmentosa. Area of Impact Retinitis pigmentosa (RP) is a genetic disease that causes retinal degeneration leading to near or complete blindness for most patients. Mechanism of Action Ray Therapeutics' therapy (Ray-001) delivers a potent transgene with […]

A human neural stem cell therapeutic candidate for the treatment of chronic cervical spinal cord injury

Translational Candidate The therapeutic candidate is a central nervous system tissue-derived GMP line developed under DISC2-10753 with an established GMP qualified seed bank. Area of Impact The target is chronic cervical spinal cord injury, which represents approximately 59% of clinical spinal cord injury cases. Mechanism of Action Integration of transplanted human neural stem cells is […]

CD72 nanoCARs for the treatment of refractory pediatric B-cell acute lymphoblastic leukemia

Translational Candidate CD72-targeting chimeric antigen receptor (CAR) T cells incorporating fully synthetic nanobodies Area of Impact Pediatric B-cell acute lymphoblastic leukemia refractory to currently available treatments without other potentially curative options Mechanism of Action The proposed candidate functions as Chimeric Antigen Receptor (CAR) T cell. When the CAR-T cell recognizes tumor cell expressing the designed […]

Development of MyoDys45-55, a gene editing therapy for Duchenne muscular dystrophy

Translational Candidate A gene editing therapy for Duchenne muscular dystrophy that permanently removes a hotspot region of patient mutations to restore dystrophin. Area of Impact Duchenne muscular dystrophy (DMD), a fatal muscle wasting disease with no cure. Mechanism of Action Our therapy uses CRISPR/Cas9 gene editing to permanently remove a hotspot region of DMD patient […]

MPS II: Plasma cell delivery of iduronate sulfatase

Translational Candidate The patient’s own B cells will be engineered to express the therapeutic enzyme needed for care in Mucopolysaccharidosis type II (MPS II) patients Area of Impact MPS II, a rare genetic disease causing multi-organ symptoms and death by age 15, if not treated. Current treatment does not address major symptoms. Mechanism of Action […]

IND-enabling Studies of Wearable Evolve-FSTL1 for Cardiac Regeneration after MI

Translational Candidate The therapeutic candidate is the Regencor’s proprietary Cardio-Regenerative Factor (FSTL1.37) formulated in the Wearable Injector Evolve-FSTL1. Area of Impact The targeted area of impact is to restore cardiac function and reduce progression to heart failure in patients after myocardial Infarction Mechanism of Action FSTL1.37 activates the controlled proliferation of progenitor heart cells within […]

Development of novel synNotch CART cell therapy in patients with recurrent EGFRvIII+ glioblastoma

Translational Candidate Human T cells transduced with a lentiviral vector encoding anti-EGFRvIII synNotch-primed anti-EphA2/IL-13Rα2 chimeric antigen receptor. Area of Impact Glioblastoma is the most common malignant brain tumor, affecting approximately 3 out of 100,000 people/year in the USA with extremely poor prognosis. Mechanism of Action In our proposed system, the first antigen EGFRvIII, which is […]

Hematopoietic Stem Cell Gene Therapy for IPEX Syndrome

Translational Candidate Human hematopoietic stem cells that have been modified to express a functional FOXP3 gene to treat patients with IPEX Syndrome Area of Impact These studies will bring stem cell gene therapy for IPEX closer to the clinic especially for those without an HLA match or disease too severe for HSCT Mechanism of Action […]

Targeting stromal progenitors to prevent the development of heart failure

Translational Candidate Monoclonal antibody targeting Ectonucleotide phosphodiesterase/pyrophosphatase 1 (ENPP1) Area of Impact Heart disease: To prevent the development of heart failure after heart attacks Mechanism of Action After myocardial infarction, myofibroblast progenitors express ENPP1. ENPP1 is a type II transmembrane protein that hydrolyzes extracellular ATP and hydrolytic products generated by ENPP1 initiate an inflammatory cascade […]