In 2022, the 25th Annual Meeting of the American Society of Gene & Cell Therapy took place from May 16th-19th. During the meeting, researchers and other stakeholders discussed the latest trends and advances in gene and cell therapy research and technology. According to a news release in Yahoo! Finance, one presentation centered around a clinical study evaluating MB-107 for infants with X-linked severe combined immunodeficiency (XSCID or X-linked SCID). The study centered around the lentiviral vector from MB-107, which has been developed for XSCID, in infants under two years old. Ultimately, the study found that the treatment helped improve immune recovery and strength without causing any other malignancies. 

About MB-107

Developed by biopharmaceutical company Mustang Bio, MB-107 is an ex-vivo lentiviral gene therapy designed to treat X-linked SCID. Within this particular Phase 1/2 study, researchers evaluated the lentiviral vector in babies with XSCID. Altogether, 23 infants were enrolled in the study. Researchers, following treatment, also followed up with the infants for a median of 2.6 years. Findings from the study include:

• MB-107 was shown to be relatively safe and well-tolerated. Although three infants experienced severe reactions, all infants recovered without any issues. 
• Prior to treatment, 74% of the infants had ongoing infections. MB-107 helped to clear up all of these infections. 
• Following MB-107 treatment, 65% of the infants were able to stop intravenous immunoglobulin use. 61% were also able to receive immunization. 
• 100% of the infants from the trial remain alive with functioning immune systems.

Researchers believe this shows promise for the future and shows that early treatment could reverse or at least address the disease. Moving forward, Mustang Bio hopes to launch a Phase 2 study by the end of 2022. 

What is Severe Combined Immunodeficiency (SCID)?

Multiple gene mutations are associated with severe combined immunodeficiency (SCID), a group of diseases that cause life-threatening immune problems. These gene mutations affect T and B cells, which normally play a role in immune response. However, in those with SCID, these cells do not function properly, causing difficulty in fighting infections. There are multiple forms of SCID, some of which are inherited in an autosomal recessive pattern and others inherited in an X-linked pattern. SCID manifests during infancy. It is most common in those of Apache, Navajo, or Turkish ancestry. Without treatment, SCID can be fatal within one year of birth. Symptoms associated with SCID can include:

• Failure to thrive
• Chronic diarrhea
• Oral thrush
• Recurrent infections which respond poorly to medication, such as
  • Pneumonia
  • Meningitis
  • Blood infections
  • Skin infections
  • Hepatitis
  • Ear infections