First Patient Dosed in CHARISMA Trial of GB221 for SMA Type 1

First Patient Dosed in CHARISMA Trial of GB221 for SMA Type 1

By, Admin 27 February 2026

Gemma Biotherapeutics (GEMMABio) has achieved a major clinical milestone.
The company announced that the first patient has been dosed in the Phase 1/2 CHARISMA clinical trial evaluating GB221, its next-generation gene therapy for spinal muscular atrophy type 1 (SMA1).

A First-in-Human, Next-Generation Approach

The CHARISMA trial (NCT07070999) is the first clinical study of a next-generation SMA1 gene therapy delivered directly into the cerebrospinal fluid (CSF) via intracisterna magna (ICM) injection.

GB221 was developed using proprietary central nervous system (CNS) platform technology licensed from the University of Pennsylvania.

Key design features include:

  • Expression of a functional, codon-optimized SMN1 gene
  • A modified transgene cassette designed to reduce overexpression-related toxicity
  • Targeted delivery to motor neurons

Trial Design and Patient Population

The CHARISMA trial will evaluate safety, tolerability, and efficacy of GB221 in pediatric patients.

Key trial details

  • Phase 1/2, first-in-human study
  • Participants aged 2 weeks to <12 months
  • Includes symptomatic and presymptomatic infants
  • One-time gene therapy administered via CSF

The goal is to assess whether GB221 can safely restore SMN1 expression and alter disease progression in SMA1.

Brazil as a Strategic Clinical Hub

The SMA1 program is a core element of GEMMABio’s partnership with the Oswaldo Cruz Foundation (Fiocruz), announced in October 2024.

Brazil plays a central role:

  • Serves as a regional hub for Latin America
  • Clinical trial operations supported by Intrials, a Brazil-based CRO
  • Conducted in collaboration with leading public and academic institutions

Investigators at the Hospital de Clínicas de Porto Alegre are closely monitoring the first patient for early signs of therapeutic benefit and developmental progress.

Regulatory Momentum in the US

GB221 has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration.

If GB221 is approved in the future, GEMMABio may be eligible for a Priority Review Voucher (PRV) under the reauthorized Rare Pediatric Disease Priority Review Voucher Program, currently extended through September 2029.

About GB221

GB221 is an investigational, one-time gene therapy for SMA1.

  • Uses an AAVhu68 vector
  • Delivers a functional SMN1 gene to motor neurons
  • Designed to minimize sensory neurotoxicity
  • Administered via ICM injection into CSF

The absence of functional SMN1 protein is the root cause of SMA.

Why This Matters?

SMA1 is the most severe form of spinal muscular atrophy. Without treatment, infants typically do not survive beyond 18 months.

Dosing the first patient in the CHARISMA trial marks a critical step forward—not only for GEMMABio, but for the broader effort to develop safer, next-generation gene therapies for devastating rare pediatric diseases.

As the trial expands, results from CHARISMA will be closely watched by clinicians, regulators, and the global rare disease community.

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