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New Generation Therapeutics (NGT) was a biotechnology company focused on developing next-generation gene therapies for complex neurological disorders. The company's core innovation was the EXACT (Expression Attenuation Capability Technology) platform, which allowed for the precise regulation of transgene expression to avoid the toxic effects of protein overexpression. NGT's pipeline primarily targeted rare genetic conditions such as Rett Syndrome and Batten disease, utilizing adeno-associated virus (AAV) vectors. In 2018, the company was acquired by Neurogene, which integrated NGT's technology and lead programs into its own clinical development efforts. Today, the assets originally developed by NGT represent the vanguard of Neurogene's efforts to treat severe CNS diseases.
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