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Seek A Miracle (SAM) was a clinical-stage biotechnology company focused on the development of gene therapies for rare, life-threatening genetic diseases. The company's primary focus was on Sanfilippo Syndrome, a lysosomal storage disorder with no existing curative treatments. Utilizing adeno-associated virus (AAV) vector technology developed at Nationwide Children's Hospital, Seek A Miracle aimed to deliver functional genes to the central nervous system to restore enzyme activity. In 2015, the company was acquired by PlasmaTech Biopharmaceuticals, which subsequently changed its name to Abeona Therapeutics. This acquisition integrated Seek A Miracle's lead programs, ABO-101 and ABO-102, into a broader rare disease pipeline. The company's work laid the foundation for some of the first clinical trials involving systemic gene therapy for Mucopolysaccharidosis Type III.
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