Amir SohailFirst patient dosed marks an important step for the SMA community and highlights a growing rare disease manufacturing partnership, with future GEMMABio programs expected to leverage SK pharmteco’s SKyvec™ viral vector platform
RANCHO CORDOVA, Calif., Sept. 22, 2026 (ZM NEWSWIRE) — SK pharmteco announced today it has been working with Gemma Biotherapeutics (“GEMMABio”), a clinical-stage global genetic medicines company, to supply critical clinical trial materials to support GEMMABio’s ongoing Phase 1/2 clinical trial, CHARISMA, (NCT07070999), evaluating GB221, an investigational next-generation gene therapy for Spinal Muscular Atrophy Type 1 (SMA1).
The CHARISMA trial marks a milestone as the first clinical trial testing a next-generation gene therapy for SMA1 delivered directly into the cerebrospinal fluid (CSF) via an intra-cisterna magna (ICM) injection in patients. 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 affirms 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 the initiative, SK pharmteco is focused on helping rare disease innovators bridge the gap between promising genetic science and reliable clinical-grade product manufacturing.
“We are proud to support GEMMABio as they advance an important clinical development program for families affected by SMA1,” said Joerg Ahlgrimm, Chief Executive Officer of SK pharmteco. “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.”
“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,” said James M. Wilson, MD, PhD, Chief Executive Officer of GEMMABio. “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.”
Spinal muscular atrophy (SMA) is an autosomal recessive, progressive neurodegenerative disease caused by mutations in the SMN1 gene that damage motor neurons and skeletal muscles, severely impairing functions such as breathing, eating, and walking. The global incidence of SMA is approximately 1:10,000 live births, while Spinal Muscular Atrophy Type 1 (SMA1) is an exceptionally severe, early-onset form accounting for approximately 60% of global SMA cases (1:17,000 live births). SMA1 is typically diagnosed before six months of age in infants with only 1–2 copies of the modifying SMN2 gene. Without treatment, SMA1 is fatal, with patients rarely surviving beyond 18 months of age.
Beyond the ongoing SMA1 trial, SK pharmteco and GEMMABio are planning additional manufacturing collaborations across GEMMABio’s rare disease pipeline, including future programs expected to leverage SK pharmteco’s SKyvec™ viral vector platform.
About SK pharmteco
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.
About Gemma Biotherapeutics (GEMMABio)
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. Learn more about GEMMABio at gemmabiotx.com and follow us on LinkedIn.
Disclosure: The University of Pennsylvania (“Penn”) owns an equity interest in GEMMABio and has received, or may receive in the future, financial consideration related to the licensing of certain Penn intellectual property to GEMMABio.
Contact:
Keith Bowermaster, APR, CCMP
Communications Consultant
keith.bowermaster@skpt.com
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