Multiple Promising Compounds in Development for Rare Neurometabolic Disorders: NKH and UCD
Nonketotic Hyperglycinemia (NKH)
Nonketotic hyperglycinemia is a devastating genetic metabolic disorder caused by an inborn error of the glycine cleavage enzyme system (GCS).
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Urea Cycle Disorders (UCD)
Urea cycle disorders are eight genetic metabolic disorders caused by defects in one of the six enzymes or two transporters of the process that moves urea through the body.
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Our Pipeline for Rare Neurometabolic Disorders
CLINICAL CANDIDATE
INDICATION​
PRECLINICAL
PHASE 1
PHASE 2
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Basket Trial
(NKH, UCD, Other Rare Metabolic Disorders*​​)
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​Glycine Encephalopathy
(Potential Single Pivotal Trial Under NKH)
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​​Urea Cycle Disorder​
(Potential Single Pivotal Trial under UCD)
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LBX-100
LBX-200
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Glycine Encephalopathy
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Urea Cycle Disorder​​
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LBX-400
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Urea Cycle Disorder​
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LBX-300
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Glycine Encephalopathy
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​​Urea Cycle Disorder​​
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505(b)(1) New Chemical Entity - Combination Therapies
505(b)(1) New Chemical Entity - Monotherapies
LEAD CANDIDATE
LEAD CANDIDATE
LEAD CANDIDATE
* Includes X-linked adrenoleukodystrophy (X-ALD), metachromatic leukodystrophy (MLD), leukoencephalopathy, van der Knaap syndrome, and X-linked cobalamine disorder.
Our Leadership
Our team brings together leaders from emerging biopharma, academia, clinical practice, and life science companies.
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Mstone Healthcare Partners
Liberyx’s controlling shareholder is Mstone Healthcare Partners, an entrepreneurial incubator building aesthetics and healthcare, life sciences, and AI-related companies. Mstone founds, staffs, develops, and grows portfolio companies by building upon novel technology as well as human and investment capital.