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We are an innovative clinical stage biopharma combating rare pediatric neurometabolic disorders

Liberyx Therapeutics is focused on developing safe, patient-friendly treatments for neurometabolic disorders such as nonketotic hyperglycinemia (NKH) and urea cycle disorders (UCD).

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Multiple Promising Compounds in Development for Rare Neurometabolic Disorders: NKH, UCD and X-ALD

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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X-linked Adrenoleukodystrophy (X-ALD) 

X-linked adrenoleukodystrophy is an inherited metabolic disorder caused by mutation of gene that encodes membrane protein responsible for transporting very long-chain fatty acids.

 

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 Team

Our Executive Leadership

Our Board of Directors

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.

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