You are here

Gene Therapy Fast-Tracked for Huntington’s Disease Treatment

First Patient Expected to Receive AMT-130 Therapy in 2019

uniQure N.V. has received a fast track designation from the FDA for AMT-130, a gene therapy candidate for the treatment of Huntington’s disease. The agent comprises a recombinant AAV5 vector with a DNA cassette encoding a microRNA that non-selectively reduces human huntingtin protein in patients with Huntington’s disease. AMT-130 is unique as it targets the highly toxic exon1 protein fragment, which is more toxic than the mutant huntingtin protein. 

Huntington’s disease affects an estimated three to seven per 100,000 people of European ancestry. Adult-onset Huntington’s disease, the most common form, usually appears during the ages of 30 to 40 years. The rarer juvenile form begins in childhood or adolescence, and typically progresses at a faster rate than the adult-onset form. Affected individuals usually live 10 to 15 years after signs and symptoms appear. There are no approved therapies to delay the onset or slow the progression of the disease.

uniQure is poised to begin the phase 1/2 study of AMT-130, which is the first, one-time administered adeno-associated viral gene therapy to enter clinical testing for Huntington’s disease. The company is on track to treat the first patient in the second half of 2019.

Source: uniQure N.V., April 8, 2019

Recent Headlines

Scenesse is new treatment for people with rare, painful light sensitivity disease
Humira, Rituxan top list of drugs that added $5.1 billion to nation's health care bill
Lower court rulings cleared the way for generic versions of the MS drug
Maryland man wins lawsuit that alleges that the company's antipsychotic caused his gynecomastia
Antidepressants, ADHD meds are also used to self-poison
U.S. maintains measles elimination status as NY outbreak ebbs
Study lists steps that could save close to $300 billion a year
While many victims used THC, the cause remains elusive
Descovy joins Truvada, another Gilead product, in the HIV prophylaxis market