SK pharmteco expands rare disease manufacturing partnership for SMA treatment

RANCHO CORDOVA, Calif., Sept. 22, 2026 /CourierPR/ -- SK pharmteco and GEMMABio have expanded their collaboration in rare disease manufacturing, following a significant milestone in a clinical trial for Spinal Muscular Atrophy (SMA) Type 1. The CHARISMA trial, which is a Phase 1/2 clinical trial evaluating GB221, an investigational next-generation gene therapy for SMA1, marks a crucial step forward in the treatment of this severe and rare disease. SMA1 is a form of SMA that accounts for approximately 60% of global SMA cases, with a prevalence of 1 in 17,000 live births. Without treatment, patients with SMA1 typically do not survive beyond 18 months of age.

SK pharmteco, a global contract development and manufacturing organization (CDMO), has been working with GEMMABio, a clinical-stage global genetic medicines company, to supply critical clinical trial materials for GEMMABio’s CHARISMA trial. The CHARISMA trial is the first clinical trial to test a next-generation gene therapy for SMA1, delivered directly into the cerebrospinal fluid (CSF) via an intra-cisterna magna (ICM) injection. By using this targeted delivery route and leveraging a proprietary central nervous system (CNS) platform technology licensed from the University of Pennsylvania, GEMMABio aims to achieve high levels of motor neuron transduction while minimizing systemic exposure and associated toxicities in clinical trial patients with SMA.

The partnership reflects SK pharmteco’s commitment to its Rare Disease Advancement Initiative, which seeks to accelerate the development of novel, advanced therapeutics for ultra-rare and severe orphan diseases. Through this initiative, SK pharmteco is focused on helping rare disease innovators bridge the gap between promising genetic science and reliable clinical-grade product manufacturing.

SK pharmteco is a global contract development and manufacturing organization (CDMO) with production sites, research & development facilities, and analytical laboratories across the U.S., Europe, and South Korea. The company’s core capabilities center on small molecules, peptides, and viral vectors, providing the specialized expertise needed to bring complex therapies to market. Through these pillars, SK pharmteco supports biopharmaceutical partners of all sizes with comprehensive development and manufacturing solutions worldwide. SK pharmteco is a subsidiary of SK Inc. (KRX: 034730) (SK), the strategic investment company for SK Group, South Korea’s second-largest conglomerate.

GEMMABio is a clinical-stage therapeutics company focused on speeding the research of and global access to life-changing advanced therapies for those living with rare diseases. The company conducts research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably. GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia.