Crigler-Najjar Syndrome - Healing Genes

Crigler-Najjar Syndrome

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Gene Transfer Clinical Study in Crigler-Najjar Syndrome (VALENS)

VALENS: A Phase 1/2, Randomized, Open-Label, Ascending-Dose, Delayed-Treatment Concurrent Control Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT342, an AAV8-Delivered Gene Transfer Therapy in Crigler-Najjar Syndrome Subjects Aged 1 Year and Older

Phase 1 / 2


Researchers at research centers in NY and PA as well as abroad are recruiting patients with  Crigler-Najjar Syndrome to trial of a new gene therapy to correct mutations in the UGT1A1 gene. This therapy is a virus engineered to transfer a functional UGT1A1 gene to the patient, so that their bodies can process bilirubin normally.

Participants will undergo a single IV infusion of the investigatory gene therapy, then be assessed at 12 weeks and 18 weeks, with a total observation period of 24 weeks.


  • Be 1 years of age or older
  • Have a diagnosis of Crigler-Najjar syndrome resulting from a confirmed mutation in the UGT1A1 gene as assessed by a Sponsor-approved testing facility
  • Be prescribed daily phototherapy for a minimum of 6 hours within a 24-hour period (daily illumination time).


  1. Screening to confirm eligibility.
  2. Participants receive a single infusion of the investigatory gene therapy.
  3. Phototherapy will continue for 12 weeks, when bilirubin levels will be assessed.
  4. Phototherapy will then discontinue for 4 weeks, when bilirubin levels will be assessed again.
  5. Total follow up will continue until 24 weeks post step (2).


The study sites include
Children’s Hospital at Montefiore in Bronx, NY. Map.
Clinic for Special Children in Strasburg, PA. Map.
Kim Trant, Director of Patient Advocacy  |  (415) 805-1049  |  [email protected]
Audentes Therapeutics
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