SK pharmteco announced on September 22, 2026 an expanded manufacturing collaboration with Philadelphia-based Gemma Biotherapeutics (“GEMMABio”), supplying critical clinical trial materials for GEMMABio’s ongoing Phase 1/2 CHARISMA trial (NCT07070999) evaluating GB221, a next-generation gene therapy for Spinal Muscular Atrophy Type 1 (SMA1). CHARISMA is notable as the first clinical trial testing a next-generation SMA1 gene therapy delivered directly into the cerebrospinal fluid via intra-cisterna magna (ICM) injection — a targeted delivery route built on proprietary CNS platform technology licensed from the University of Pennsylvania, aimed at achieving high motor neuron transduction while minimizing systemic exposure and toxicity.
A Philly Gene Therapy Pioneer, Building
GEMMABio is led by CEO James M. Wilson, MD, PhD, said “Advancing complex, next-generation genetic therapies into the clinic requires not only rigorous science but also an exceptionally high standard of manufacturing precision and analytical expertise. SK pharmteco has demonstrated deep alignment with our core principles of safety, efficacy, and global accessibility. Their robust manufacturing infrastructure and technical agility have been vital to supplying high-quality materials for our CHARISMA trial. With the first patient already dosed and the trial progressing on schedule, this collaboration strengthens our ability to scale our programs efficiently from bench to bedside
Joerg Ahlgrimm, CEO of SK pharmteco, framed the partnership around SK pharmteco’s Rare Disease Advancement Initiative, which aims to help rare disease innovators bridge the gap between genetic science and clinical-grade manufacturing: “We are proud to support GEMMABio as they advance an important clinical development program for families affected by SMA1. Rare disease programs move with urgency, but they also require partners who can maintain the quality, consistency, and technical discipline needed to manufacture complex genetic medicines. This collaboration reflects our commitment to helping scientists and innovators move promising therapies into clinical trials and, ultimately, toward the patients who need them.”
Why SMA1 Is Such an Urgent Target
Spinal muscular atrophy is an autosomal recessive neurodegenerative disease caused by mutations in the SMN1 gene, affecting roughly 1 in 10,000 live births globally. SMA1 — the most severe, earliest-onset form, accounting for about 60% of global SMA cases — is typically diagnosed before six months of age and, left untreated, is fatal, with patients rarely surviving past 18 months. Beyond the current SMA1 trial, SK pharmteco and GEMMABio are already planning additional manufacturing collaborations across GEMMABio’s broader rare disease pipeline, including future programs expected to use SK pharmteco’s SKyvec™ viral vector platform.
About GEMMABio
GEMMABio is a Philadelphia-based, clinical-stage genetic medicines company focused on accelerating research and global access to advanced therapies for rare diseases, led by gene therapy pioneer Jim Wilson and a team with deep roots in academic gene therapy research. The University of Pennsylvania holds an equity interest in GEMMABio and has received or may receive future financial consideration tied to licensing Penn intellectual property to the company.
About SK pharmteco
SK pharmteco is a global CDMO with production, R&D, and analytical facilities across the U.S., Europe, and South Korea, specializing in small molecules, peptides, and viral vectors. The company is a subsidiary of SK Inc. (KRX: 034730), the strategic investment arm of SK Group, South Korea’s second-largest conglomerate.
