An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children with limb-girdle muscular dystrophy type R5 (LGMDR5). Long-term data from the first two treated patients suggest the therapy is working as expected, with no serious side effects, according to developer Atamyo Therapeutics.
“These initial results are very encouraging and demonstrate the potential of our product with biological data rarely seen in neuromuscular diseases and at such an early stage of the trial,” Angela Columbano, CEO of Atamyo, said in a company press release.
LGMDR5, formerly known as LGMD2C, is a genetic disorder caused by mutations in the SGCG gene. This gene provides instructions for making the protein gamma-sarcoglycan, which is normally needed to maintain muscle health and is part of a complex found in skeletal muscle. Lacking a working version of this gene, muscles accumulate excessive damage, leading to symptoms such as muscle weakness and eventual loss of walking ability. Symptoms of LGMDR5 typically manifest during early childhood.
How ATA-200 targets the root cause
ATA-200 is a gene therapy designed to deliver a healthy version of the SGCG gene to muscle cells, thereby restoring gamma-sarcoglycan protein production and addressing the underlying cause of the disease. The therapy delivers its genetic payload using a viral vector, which is a virus engineered to deliver a therapeutic gene rather than cause infection. The vector is derived from adeno-associated virus, which is often used as a platform for gene therapy because it’s good at delivering genetic material into human cells but generally doesn’t cause serious illness.
Atamyo is sponsoring a Phase 1 clinical trial (NCT05973630) testing the safety of ATA-200, given as a one-time infusion into the bloodstream, in four children with LGMDR5, ages 6 to 13. According to the company, safety data have so far been positive, with no serious side effects of the gene therapy being reported in any of the four children who have been dosed.
The first two participants in the study have now been followed for more than a year since the ATA-200 treatment. Muscle biopsies, collected six months after treatment, indicate the therapy is working as intended: gamma-sarcoglycan protein was detected in more than 90% of muscle fibers from both patients (90.2% in the first patient and 92.1% in the second).
One-year data showed that ATA-200 led to a reduction in levels of CPK (creatine phosphokinase), a marker of muscle damage. The one-year data also showed benefits on tests of motor function, according to Atamyo. The company’s press release didn’t provide details on the motor function outcomes observed.
Atamyo said that further results from the study will be published in the coming months when new, longer-term data become available.
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