Technology

Proprietary Platform

Genetix’ vectors, GMP manufacturing, and stem cell processing technologies are a proprietary platform for the development of therapeutic products for genetic diseases.  Using Genetix’ technology, somatic bone marrow stem cells are harvested from the patient, a healthy version of the causitive gene is inserted into the cells followed by transplantation back into the patient.  Through a single administration, the Genetix products achieve a lifetime cure.   To maximize our initial probability of success, it is currently being applied to single gene diseases which are treatable by allogeneic bone marrow transplantation.  Correcting the patients own cells enables Genetix to treat the patients who do not have a matched related donor (80%+) and avoids the graft vs. host disease/lack of engraftment which results in up to 15% mortality.  Based upon the results in clinical trials for Thalasemia and adrenoleukodystrophy (ALD), proof of principle has been achieved for the platform.

 

Lentiviral Gene Delivery

Lentiviral vector-based gene delivery calls for stem cells to be harvested from the patient, transduced ex vivo with a vector containing a healthy gene, and returned, with the incorporated corrected gene, to the patient to enable the re-constitution of bone marrow and a full cure.  Currently, bone marrow transplant is an effective long-term treatment for genetic  disorders, but there is no match for 70% of patients.  Further, in the absence of a match sibling donor, the mortality rate with matched unrelated donors can reach 15% due to graft versus host disease and lack of engraftment.  As such, matched unrelated donor allogeneic BMT is standard of care in ALD (as there is no other therapy and the disease is rapidly debilitating and deadly).  It is curative in both thalassemia and sickle cell disease but is not as common because these diseases can be managed by transfusion and chelation therapy ( thalassemia) and palliative morphine pain care (Sickle cell disease)  though the patients have a shortened lifespan due to iron build up organ failure in Thalassemia and ischemic events in Sickle cell Disease.  Because Genetix inserts a healthy gene in the patients’ own (autologous) cells, the mortality due to Graft versus host disease and lack of engraftment is avoided and the patient does not need to be on lifelong immunospression.  As such, we believe that Genetix therapies have the potential to become standard of care in all of the diseases we are treating.

 

LentiPak™

Genetix’ proprietary Lentipak™ vector backbone is based on ten years of research at Harvard and MIT.  Combined with Genetix’ somatic stem cell processing technologies, these vectors have achieved the highest lifetime levels of stem cells carrying the correct gene ever observed.  The vector backbone, GMP processes and Quality control  are designed to maximize safety and efficacy.  The company’s technology is compatible with stem cells harvested from the bone marrow, circulating blood, umbilical cord blood or other somatic sources.