Clinical Edge

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Gene therapy in severe hemophilia B

Reiss UM et al. ASH 2018, Abstract 491

Key clinical point: With a median follow-up of 6.7 years in 10 patients aged 18-64 years who were treated with scAAV2/8-LP1-hFIX–comediated gene therapy, factor IX expression has been persistent and stable.

Major finding: Factor IX expression averaged 1.9%-2.3% at the lower doses, and 5.1% at the high dose at up to 8.6 years.

Study details: An interim follow-up data for 10 patients in a phase 1/2 study.

Disclosures: Dr. Reiss reported having no disclosures.

Read the article here.


Reiss UM et al. ASH 2018, Abstract 491.