TAY-SACHS GENE THERAPY CONSORTIUM UPDATE

MOVING CLOSER TO A CURE The Tay-Sachs Gene Therapy Consortium is working diligently to begin human clinical trials in 2013! It is a complicated process but the committed, dedicated TSGT Consortium team with support from NTSAD and the family foundations are up to the task.

NEXT STEPS

What happens next?

TSGT developed a clinical trial plan and recently submitted to the Food and Drug Administration (FDA) an information package summarizing the development program for the Investigation New Drug (IND) application necessary to initiate the clinical trial. A meeting is schedule in February. The consortium is currently submitting an application to the NIH Rapid Drug Access to Investigation Drugs program to support the production of the clinical trial grade vectors necessary for the trial.

Additional Studies A better understanding of Juvenile Tay-Sachs and Sandhoff is necessary to move forward with the first steps of the clinical trial.

Challenges

Funding for the clinical trial and necessary additional studies have yet to be secured. TSGT will apply for NIH support and continue to work closely with NTSAD and the Tay-Sachs Family Foundations.

ANIMAL UPDATE

Cats The cat studies continue to be well ahead of schedule! Nine cats have received the gene therapy. The four oldest cats are currently more than twice as old as untreated cats! Although the oldest two have noticeable rear leg weakness they are still able to walk, eat and use the litter box independently. None have shown any adverse reaction to the gene vector. Other cat studies defining the actual target dose for the human clinical trials continues to progress well and ahead of schedule.

Sheep Two of the four affected sheep that received the gene therapy continue to do well. Two were left untreated as controls. These results are encouraging but a better understanding of the natural course and variability of the disease in sheep is necessary before drawing conclusions.