Therapeutic/Technology: Gene Therapy (All)
Chimeric TGFB Signaling Receptor (CTSR) Enabled Anti-B7H3 CAR T-cell Therapy in Children and AYA with Recurrent Solid Tumors
Therapeutic Candidate or Device Engineered CAR T-cells are enhanced to allow their survival in solid tumors Indication Children, adolescents and young adults with variety of solid tumors but focus on sarcomas and neuroblastomas Therapeutic Mechanism Engineered T-cells are expanded and infused back to the patients. The additional mechanisms engineered in these cells allows them to […]
A 1XX-enhanced and fully non-viral BCMA CAR T cell therapy for Relapsed and Refractory Multiple Myeloma (UCCT-BCMA-1)
Therapeutic Candidate or Device Cryopreserved autologous TRAC locus 1XX BCMA-CAR T cells Indication Relapsed and Refractory Multiple Myeloma Therapeutic Mechanism Engineered T cells target and kill BCMA+ Myeloma cells Unmet Medical Need No durable treatments are available for RRMM and only ~30% of patients can access current BCMA CAR therapies. This product can improve product […]
Pre-Clinical To Clinical Gene Therapy Development For CMT4J
Therapeutic Candidate or Device AAV9 Gene Therapy For An Ultra-Rare Disease Called CMT4J Indication CMT4J or the FIG4 Gene Therapeutic Mechanism Gene Therapy through an Intrathecal One-Time Injection Unmet Medical Need CMT4J is an ultra-rare disorder that presently lacks any available treatment options and represents an underserved orphan population. Project Objective Successful Filing of an […]
Manufacturing of AS-202, an Antisense oligonucleotides for a Phase 1/2 Clinical Trial for Amyotrophic Lateral Sclerosis
Therapeutic Candidate or Device AS-202, an antisense oligonucleotide Indication Amyotrophic Lateral Sclerosis Therapeutic Mechanism AS-202 targets PIKFYVE, a lipid kinase. By inhibiting PIKFYVE activity, it induces exosomal secretion, which robustly clears misfolded proteins including C9ORF72 dipeptide repeat proteins (DPRs), TDP-43 and tau from neurons. Unmet Medical Need To date, therapeutic options for ALS have been […]
Neural stem cell delivered CRAd-S-pk7 oncolytic viro-immunotherapy for ovarian cancer
Therapeutic Candidate or Device A clinically tested tumor-tropic neural stem cell (NSC) platform for effective distribution of oncolytic virotherapy to ovarian cancer metastases. Indication Chemo-resistant, metastatic ovarian cancer. Therapeutic Mechanism CRAd-S-pk7 is a tumor specific replication-competent adenovirus driven by survivin, which is highly expressed in ovarian cancer cells. We will use our tumor-tropic NSC platform […]
TRX103 for prevention of GvHD in patients receiving HLA mismatched related or unrelated allogeneic HSCT for the treatment of hematologic malignancies.
Therapeutic Candidate or Device An allogenic, off the shelf, engineered regulatory T cell product that mimics the function of T regulatory Type 1 (Tr1) cells. Indication Prevention of acute and chronic Graft versus Host Disease (GvHD) in patients undergoing mismatched stem cell transplant. Therapeutic Mechanism GvHD is driven by the reaction of allogenic donor T […]
Ex vivo Engineering of Autologous Hematopoietic Stem Cells for the Treatment of Hypophosphatasia
Therapeutic Candidate or Device Hematopoietic stem/progenitor cells collected from patients with hypophosphatasia and genetically modified with a lentiviral vector to release TNALP Indication Hypophosphatasia (HPP) Therapeutic Mechanism We are proposing a cell-based enzyme replacement therapy for HPP: autologous gene-modified hematopoietic stem/progenitor cells (HSPCs) will be infused into the patient and will engraft into the bone […]
Hematopoetic stem cell gene therapy for the treatment of Tay-Sachs disease
Therapeutic Candidate or Device Autologous hematopoietic stem cells transduced with a HexA/HexB expressing lentiviral vector Indication Tay-Sachs disease Therapeutic Mechanism The transplanted gene modified autologous hematopoietic stem cells will engraft in the bone marrow and start producing HexA/HexB expressing immune progeny. Microglia, which establish residence in the brain, will deliver functional beta-hexosaminidase enzyme to affected […]
Development of TriLeukeVax, an Engineered Autologous Leukemia Vaccine for Stimulating Cytolytic Immune Responses to Residual Leukemic Stem Cells
Therapeutic Candidate or Device TriLeukeVax, an autologous AML vaccine designed to stimulate induction of anti-leukemic cytolytic activity and improve relapse free survival (RFS). Indication Older leukemia patients who achieve remission with chemotherapy and are at high risk of relapse, but are not eligible for allogeneic transplantation Therapeutic Mechanism Most older patients with acute myelogenous leukemia […]
Hematopoietic Stem Cell Gene Therapy for XCGD
Therapeutic Candidate or Device Hematopoietic stem and progenitor cells collected from X-CGD patients modified with a highly regulated lentiviral vector Indication X-linked Chronic Granulomatous Disease Therapeutic Mechanism Lentiviral vector (LV) modification of autologous hematopoietic stem and progenitor cells (HSPCs) to restore physiologic gp91phox expression. We have developed a next-generation LV designed by bioinformatic-guided screening of […]