Many research companies around the world are researching treatments for Duchenne, one of the most devastating muscle diseases. One of these is Solid Biosciences’ SGT-003 gene therapy, which is currently in development and uses new generation technologies. In this article, we will explore frequently asked questions about Solid Biosciences’ SGT-003 gene therapy.
Things to Know About SGT-003 Gene Therapy
What is SGT-003 Gene Therapy?
AAV-SLB101, a proprietary, next-generation capsid that was logically created to target integrin receptors, and a differentiated microdystrophin construct are both components of SGT-003, an investigational gene therapy that has demonstrated improved transduction in the heart and skeletal muscles while reducing liver targeting in nonclinical studies.
According to these design elements, SGT-003 may be the finest experimental gene therapy available for treating Duchenne.
Who is Eligible for SGT-003 Gene Therapy?
According to the clinical trial launched by Solid Biosciences in May 2024, participants are selected between the ages of 4 and 11. [NCT06138639]
When considering the Inclusion Criteria, the following situations are taken into consideration for the participants:
- Participants who are ambulatory. Ambulatory as defined as “being able to walk without the use of an assistive device.”
- Established clinical diagnosis of DMD and documented dystrophin gene mutation predictive of DMD phenotype confirmed by Sponsor genetic testing.
- Negative for AAV antibodies.
- On a stable dose of at least 0.5 mg/kg/day of oral daily prednisone or 0.75 mg/kg/day deflazacort for ≥12 weeks prior to entering the study.
- Meet 10-meter walk/run time criteria
- Meet time to rise from supine criteria
- Participant has body weight: ≤50 kg
Who is Not Eligible for SGT-003 Gene Therapy?
Considering the exclusion criteria, it is stated that patients with some exon deletions cannot participate in this study.
- Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing.
This may mean that Solid Biosciences SGT-003 gene therapy cannot be used for these exon deletions.
Interim Clinical Trial Results of SGT-003 Gene Therapy
Solid Biosciences announced interim results for its SGT-003 gene therapy on February 18, 2025. [Read More]
- Mean vector copies per nucleus: 18.7 (N=3),
- Mean microdystrophin expression: 110% (N=3), as measured by western blot,
- Mean microdystrophin expression: 108% (N=3), as measured by mass spectrometry,
- Mean percent dystrophin positive fibers: 78% (N=3), as measured by immunofluorescence,
- Mean beta sarcoglycan percent positive fibers: 70% (N=3),
- Mean nNOS (neuronal nitric oxide synthase) percent positive fibers: 42% (N=3),
- Improvements in 7 additional muscle integrity biomarkers (N=3), and
- Early mean improvement in left ventricular ejection fraction (LVEF) of 8% from baseline at Day 180 (N=2).
In mid-2025, the Company plans to request a meeting with the FDA to discuss potential accelerated approval pathways for SGT-003. [Read More]
SGT-003 and Elevidys Comparison
Promising interim trial results of the SGT-003 gene therapy have drawn attention to this research, and patients and their families have begun to research whether SGT-003 or Elevidys is better.
When clinical data are examined, it can be seen that SGT-003 has higher microdystrophin expression. [Read More]
Price of SGT-003 Gene Therapy
Since SGT-003 is still in the research phase, Solid Biosciences has not yet announced the price of the gene therapy.
Hopefully SGT-003 will work, gain approval, and be available on the pharmaceutical market at a price that every family can afford.
Conclusion
Many research companies around the world continue their work to treat Duchenne muscular dystrophy. New generation gene therapies are still spreading hope for patients. Solid Biosciences’ SGT-003 gene therapy could rival Elevidys and pave the way for easier access for patients in terms of cost.
Learn More: Potential Upcoming New Gene Therapies for Duchenne Muscular Dystrophy
