Our
pipeline

Innovation in progress

We are advancing clinical development programs across multiple rare disease therapeutic areas and drug modalities.

Biologic

Description:
Phase:
  • Preclinical
  • IND
  • 1
  • 2
  • 3
  • Approved

UX143 is an investigational, fully human monoclonal antibody that inhibits sclerostin, a protein that acts on a key bone-signaling pathway and inhibits the activity of bone-forming cells.

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Small molecule

Description:
Phase:
  • Preclinical
  • IND
  • 1
  • 2
  • 3
  • Approved

Gene therapy

Description:
Phase:
  • Preclinical
  • IND
  • 1
  • 2
  • 3
  • Approved

UX111 (formerly ABO-102) is an investigational novel gene therapy under evaluation in the ongoing pivotal Transpher A trial in patients with Sanfilippo syndrome type A (MPS IIIA).

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DTX401 is an investigational adeno-associated virus serotype 8 (AAV8) gene therapy designed to deliver stable expression and activity of glucose-6-phosphatase-alpha (G6Pase-α) using a single intravenous infusion.

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DTX301 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of the OTC gene using a single intravenous infusion.

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UX701 is an investigational AAV9 gene therapy designed to deliver a modified form of the ATP7B gene, which is otherwise too large to package in an AAV vector.

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UX055 is an investigational gene therapy in preclinical development for CDD that aims to deliver a functional copy of the human CDKL5 gene to the brain using an AAV9.

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ASO/mRNA

Description:
Phase:
  • Preclinical
  • IND
  • 1
  • 2
  • 3
  • Approved

GTX-102 is an investigational antisense oligonucleotide (ASO) therapy designed to inhibit expression of UBE3A-ATS in order to prevent silencing of the paternally inherited allele of the UBE3A gene and reactivate expression of the deficient protein.

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Clinical Pipeline

Clinical study information

If you are considering participating in a clinical study, you should discuss it with your physician.

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