top of page
ChatGPT Image May 8, 2026, 06_41_59 PM.png

Our Pipeline

Genosera is advancing a focused pipeline of next generation gene therapies through development toward the clinic with the goal of delivering life-changing treatments for patients and families.

OUR PIPELINE

Pipeline Overview

Our pipeline is advancing through the FDA development pathway. Hover or focus each program to see details. Progress bars will animate left to right in the live site.

Gemini_Generated_Image_r9qqi9r9qqi9r9qq_
Pre-IND meetings with the FDA for GA-001, GA-002 and GA-003 are planned for 2026. Clinical trials are planned to begin in 2027.

GA-001 GNE Myopathy

 A progressive adult-onset recessive genetic disorder caused by loss of function mutations in the GNE gene. Our therapy is designed to restore GNE function and restore muscle strength.

​

Learn More

GA-002 LGMDR9

Limb-girdle muscular dystrophy R9 caused by a defect in the FKRP gene, characterized by progressive muscle weakness. Our therapy is aimed at addressing muscle function and mobility.

Learn More

​ GA-003 LAL-D

Lysosomal Acid Lipase Deficiency, a severe rare lysosomal storage disorder caused by recessive mutations in the LIPA gene. Our therapy is designed to restore enzyme activity and prevent disease progression.

Learn More
ChatGPT Image May 8, 2026, 07_31_19 PM.png

Advancing Today. Transforming Tomorrow.

Every discovery brings us closer to delivering gene therapies that change lives. Together, we can build a healthier future. Button: Support Our Mission ♡

bottom of page