According to a publication from Fierce Biotech, Biotech company Sangamo recently updated investors on three trials it has underway testing experimental forms of gene and cell therapies. The trials are testing treatments for three rare diseases: beta thalassemia, mucopolysaccharidosis, and hemophilia.

About ST-400

ST-400 is a experimental cell therapy for beta thalassemia, a rare blood disease. People with beta thalassemia have reduced levels of hemoglobin, the ferrous protein in blood that transports oxygen around the body. This results in poor oxygen distribution throughout the body to various muscles, tissues, and organs.

Sangamo’s ST-400 study involved only one individual, which is hardly ideal. Additionally, when the trial finally began, Sangamo’s one patient had an allergic reaction to a cryoprotectant (medical antifreeze) that was present in the drug. It was a shocking and discouraging start to the trial.

However, the patient quickly stabilized. Within just a few weeks of treatment, the patient displayed significant recovery in levels of neutrophils and platelets, two of the most important blood cells that are frequently in low supply in beta thalassemia patients.

Almost 2 months after receiving the drug infusion, the trial patient has continued to enjoy stable hemoglobin levels. Since many beta thalassemia patients have to undergo frequent blood transfusions to manage their condition, the development of a drug that could effectively stabilize their hemoglobin and platelet levels would be a huge breakthrough.

More patients will need to be involved and studied before Sangamo can claim any definitive results about the drug. However, the results are exciting and undeniably encouraging.

About SB-913

SB-913 had the unfortunate distinction of having some some bad press going into April. Sangamo claimed the experimental mucopolysaccharidosis II (MPS II) drug would increase levels of an enzyme that allows healthy individuals to break down the complex sugars (called GAGs) that can accumulate in tissues and organs of MPS patients. However, data published earlier this year showed that the drug had made little or any difference in the GAG levels of studied individuals, even when administered over 24 weeks.

The treatment uses CRISPR-rivalling ZFN technology to splice in a “working” form of the gene that codes for an enzyme that helps break down GAGs. Sangamo announced that they would continue to collect data for the trial in the second half of the year, in part to determine whether or not to proceed with phase 3 testing.

About SB-525

SP-525 is an experimental hemophilia A gene therapy being jointly developed by Sangamo and Pfizer.

A phase 1/2 clinical study is currently underway, and interim data from eight participants is showing encouraging results. It seems to be well tolerated, and the early data suggests that it might improve the condition of the patients – the degree of response seemed correlated to the amount of treatment received. Further studies of the treatment are likely as enrollment for additional cohorts is already underway.

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