Translational Candidate Autologous blood stem cells edited to restore glucocerebrosidase expression Area of Impact Gaucher disease type 1 (non-neuronopathic) Mechanism of Action To treat Gaucher disease, autologous blood stem cells…
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…
Translational Candidate Injectable biomaterial derived from the natural scaffolding of pig hearts Area of Impact Improving the quality of life of patients with heart attacks Mechanism of Action The proposed…
Translational Candidate The translational candidate is Autologous Hematopoietic Stem and Progenitor Cells from CD3δ SCID Patients Corrected by Adenine Base Editing Area of Impact The candidate will provide treatment for…
Translational Candidate Autologous CD34+ hematopoietic stem and progenitor cells (HSPC) with BTK gene insertion for treatment of X-linked agammaglobulinemia (XLA). Area of Impact The candidate will provide improved outcomes for…
Translational Candidate The candidate is CD34+ HSPCs transduced ex vivo with a LAMP2 lentiviral vector. Area of Impact Danon Disease, Lysosomal Storage Diseases, Drug Development for Rare Disease Mechanism of…
Translational Candidate s15.BsAb.BCMA-CAR NK cells derived from CD34(+) umbilical cord blood hematopoietic stem cells Area of Impact patients with multiple myeloma Mechanism of Action s15.BsAb.BCMA-CAR NK cells are umbilical cord…
Translational Candidate Human hematopoietic stem cells that have been modified to express a functional WAS gene to treat patients with Wiskott Aldrich Syndrome (WAS) Area of Impact These studies will…
Translational Candidate Extracellular vesicles derived from stem cells for the treatment of corneal scars. Area of Impact The candidate aims to restoring vision by reducing the need for corneal transplant…
Translational Candidate A blood forming stem cell based therapy to treat HIV infection and enhance HIV immunity. Area of Impact We are seeking to develop a therapy to treat HIV…
Get the latest CIRM news, information on upcoming meetings, and funding opportunities delivered to your inbox.
