Gemma Bio

Gemma Biotherapeutics (Gemma Bio) is a clinical-stage biotechnology company focused on developing gene therapies for rare neurodegenerative disorders. The company's lead asset, GB-101, is an AAV9-based gene therapy designed to treat GM1 gangliosidosis by delivering a functional copy of the GLB1 gene. Gemma Bio was established to advance programs originally developed at UT Southwestern Medical Center and previously held by Taysha Gene Therapies. The company leverages deep expertise in adeno-associated virus (AAV) vector design to address the underlying genetic causes of lysosomal storage diseases. By focusing on high unmet medical needs in the pediatric population, Gemma Bio aims to provide durable, transformative treatments for patients with limited options.