Uniqure’s gene therapy, a new ray of hope for patients

Announcement

The uniqueness of gene therapy for Huntington’s disease’s pivotal results is a new ray of hope for patients disappointed in the past few years. Though one expert is concerned about the communication of the results, which are building false expectations. It is said that few therapeutic areas claiming hope as confined to the short supply. This is the reason why, when Dutch biotech declared super positive three-year data from its gene therapy for Huntington’s disease last month, the mainstream media and the company were so thrilled. Trump and others are also excited, as it brings hope for patients and families suffering the adverse impact and loud stress of the most extreme diseases.

The director of the University College London Huntington’s disease centre and professor of clinical neurology, Sarah Tabrizi, said, “We didn’t expect around 75% steadiness of the clinical progression.”

One of the analysts at Stifel said, “In the three years, a single high dose of AMT-130 proved to benefit 80% of the patients. This percentage has been observed for two years in the same trial. This result raised the expectation bar.”

Uniqure’s potential driving business and science with the social appearance

The Uniqure’s shares have accelerated by 248% on 24^th September on the proud announcement of Huntington’s green signal for its treatment for the first time. The CEO at uniqure, Matt Kapusta, has been following a reaction trend to the social media announcement in Gen-G way via the eyes of his 16-year-old daughter. He cheerfully said, ‘My daughter has been forwarding me the videos of family members discussing Huntington's progress (update) to their loved ones and capturing their reaction. It’s really very humble and cheerful.’

The future with this new light in the spark

The new light is so soothing after the long-standing disappointment. It’s a hope that the whole Huntington community was aware of. The past five years have witnessed clinical failures from Roche, Sage Therapeutics and Wave Life Sciences. Alongside, the wrap-up of Huntington’s aimed triplet therapeutics, as a drug developer faced challenges to exercise a fragile stability of specifically mitigating the mHTT protein simultaneously preserving the neuroprotective wild-type protein.

Now with the uniqure gene therapy, there will be a relief and release from Huntington's, a neurodegenerative disease that has struck patients during the peak of their lives (mainly in their 30s and 40s) and later transferred to half of the next generation.