- SK pharmteco is supplying clinical trial materials to support GEMMABio’s ongoing Phase 1/2 CHARISMA trial evaluating GB221, an investigational gene therapy for Spinal Muscular Atrophy Type 1 (SMA1).
- The collaboration is also expected to support additional manufacturing programs across GEMMABio’s rare disease pipeline, including programs using SK pharmteco’s SKyvec viral vector platform.
SK pharmteco is working with clinical-stage genetic medicines company GEMMABio to supply clinical trial materials for its ongoing Phase 1/2 CHARISMA trial, which is evaluating GB221, an investigational next-generation gene therapy for Spinal Muscular Atrophy Type 1 (SMA1). The companies said the collaboration supports the clinical development of GB221 and SK pharmteco’s Rare Disease Advancement Initiative.
The CHARISMA trial is evaluating GB221 delivered directly into the cerebrospinal fluid (CSF) through an intra-cisterna magna (ICM) injection. According to the companies, the approach is intended to achieve high levels of motor neuron transduction while minimizing systemic exposure and associated toxicities. GB221 uses a proprietary central nervous system platform technology licensed from the University of Pennsylvania.
SMA is a progressive neurodegenerative disease caused by mutations in the SMN1 gene that damage motor neurons and skeletal muscles. SMA1 is described in the release as an early-onset form of the disease. The companies said the condition can severely impair breathing, eating and walking, and that untreated SMA1 is fatal, with patients rarely surviving beyond 18 months of age.
SK pharmteco said the partnership reflects its focus on supporting the development and manufacturing of advanced therapeutics for rare and severe orphan diseases. The company is providing manufacturing infrastructure and technical support for materials used in the CHARISMA trial. GEMMABio CEO James M. Wilson said the company has already dosed its first patient and that the trial is progressing on schedule.
Beyond the current SMA1 program, the companies are planning additional manufacturing collaborations across GEMMABio’s rare disease pipeline. Future programs are expected to leverage SK pharmteco’s SKyvec viral vector platform.
