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 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…
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…
Translational Candidate We propose to develop a universal, off-the-shelf treatment for articular cartilage repair based on pluripotent stem cell (PSC)-derived chondrospheres Area of Impact The proposed therapy could treat the…
Translational Candidate An autologous somatic stem cell therapy for the treatment of osteonecrosis. Area of Impact Osteonecrosis is a painful, progressive disease for which there is no treatment, save replacing…
Therapeutic Candidate or Device B cells will be isolated from patients suffering MPSI. These will be transformed with a normal copy of the gene and re-introduced into the patient Indication…
Therapeutic Candidate or Device GNSC-001 is an adeno-associated vector (AAV) expressing an optimized form of IL-1Ra, a naturally occurring protein that blocks IL-1 signaling. Indication Osteoarthritis of the knee Therapeutic…
Therapeutic Candidate or Device The therapeutic candidate is an ex-vivo autologous gene therapy approach for Infantile Malignant Osteopetrosis (IMO). Indication The target clinical indication is Infantile Malignant Osteopetrosis (IMO), a…
