FDA lifts clinical suspension of two gene therapies for hemophilia and Huntington's disease

The FDA has successively lifted two clinical suspensions of gene therapy, involving hemophilia and Huntington's disease.

A few days ago, UniQure announced that the FDA has lifted the suspension of clinical trials of the gene therapy AMT-061 for hemophilia B.

In December 2020, after a patient treated with the research gene therapy AMT-061 was diagnosed with hepatocellular carcinoma (HCC), the FDA asked UniQure to temporarily shelve the HOPE-B clinical trial to further investigate whether the AAV virus itself caused it.

Last month, UniQure announced the results of its investigation into the HCC incident.

In addition, the aforementioned AMT-061 subjects with liver cancer have a variety of risk factors related to HCC, including a 25-year history of hepatitis C and hepatitis B.

The report also showed that "analysis of tumor gene expression and adjacent tissues showed that the subject's tumor is in a precancerous state, which is consistent with the conclusion that the patient has risk factors for HCC.

UniQure's gene therapy candidate drug AMT-061 is a gene therapy based on adeno-associated virus 5 (AAV-5).

HOPE-B is an open-label, single-dose, single-arm, global clinical trial.

Previous trial data showed that AMT-061 promoted the production of FIX in patients in almost all trial participants.

Huntington's disease gene therapy VY-HTT01 clinical trial

After lifting the suspension of uniQure's B-type gene therapy clinical trial, the FDA also lifted the clinical suspension of Voyager Therapeutics's Huntington's disease (HD) gene therapy VY-HTT01.

The suspension of the VY-HTT01 clinical trial project was due to chemistry, manufacturing and control (CMC) issues.

But now, the FDA has lifted the restrictions on VY-HTT01.

Huntington's disease is a progressive brain disease caused by defective genes, usually fatal after about 20 years.

Just last month, a once promising HD project from Singapore genetic medicine company Wave Life Sciences was abandoned.

A few days ago, Roche and Ionis also stopped the research work of tominersen (also known as IONIS-HTTRx, RG6042), which is an antisense RNA drug designed to reduce the production of all types of huntingtin proteins (HTT, including mHTT).

Reference source:

1.
FDA Officially Lifts Hold on UniQure's Promising Hemophilia B Therapy

2.
FDA says Voyager can trek on as it removes trial hold for Huntington's gene therapy test