Date: 2018-10-22

Type of information: Licensing agreement

Compound: adeno-associated virus (AAV) vectors, including AAV9

Company: RegenXBio (USA - MD) Ultragenyx Pharmaceutical (USA - CA)

Therapeutic area: Rare diseases - Genetic diseases - Neurological diseases

Type agreement: licensing

Action mechanism: gene therapy.

Disease: CDKL5 deficiency disorder


  • • On October 22, 2018, Ultragenyx Pharmaceutical announced it has exercised its option with RegenXBio to develop a gene therapy to treat patients with CDD (CDKL5 Deficiency Disorder) utilizing RegenXBio's adeno-associated virus (AAV) vectors, including AAV9. CDD is a severe and debilitating neurological disorder that shares many features of Rett Syndrome, though the two disorders are now considered distinct from each other.
  • CDD is an X-linked genetic disorder that results in seizures that typically begin in the first few months of life and severe intellectual and gross motor impairment. The CDKL5 gene provides instructions for making a protein that is essential for normal brain development, with mutations causing a deficiency in the protein level. CDD is caused by a deficiency in an enzyme that may only be needed in small quantities for the brain to function normally. In the past, CDD was commonly diagnosed as an atypical Rett Syndrome until genetic diagnostics changed this view and improved accurate diagnosis.

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