the CURE

What is gene therapy?

Gene therapy treats disease by putting working genetic code into cells. It’s like getting an instruction manual delivered to your doorstep, showing you how to fix that broken sink that’s flooding the kitchen.

This is a cure for GMPPA because it tells the cells how to make a functional enzyme. Here’s how it works:

Design GMPPA gene therapy - researchers will design 3 different combinations of genetic code to fix the defects.

Model study & FDA application - the gene therapy combinations will be tested in GMPPA disease models & the best one selected; then we will open our FDA application.

Safety studies - based on FDA guidance, deeper studies into the safety of the GMPPA gene therapy will be done.

Manufacture treatment - the final medical grade version of the cure is produced for patient use.

Clinical Trial/FDA approval - CURE given to patients!

*While our ultimate goal is the development of a gene therapy, bloom for a cure will simultaneously invest in drug research for GMPPA.

Milestones

  • Design GMPPA gene therapy

  • Model study & FDA application

  • Safety studies

  • Manufacture treatment

  • Clinical Trial/FDA approval - the cure is given to Rosie and other children with GMPPA