uniQure’s Historic Hemophilia B Approval Sets the Stage for Potential Treatments of Huntington’s and ALS

In an unprecedented year for uniQure, the Massachusetts and Amsterdam-based biotechnology company made history with the U.S. and European approval of the world’s first gene therapy for hemophilia B. On Monday, the company released its 2022 financial report which detailed the progress of its Huntington’s disease and ALS programs. With this groundbreaking development, uniQure is on the cutting edge of the medical landscape, revolutionizing the treatment and management of debilitating chronic diseases.

In November 2022, uniQure and CSL Behring made history when Hemgenix was approved in the United States. This momentous achievement marked a major milestone for both companies and the medical field, setting an exciting precedent for the future of health care.

Hemgenix, the world’s most expensive medicine, has recently been conditionally approved in Europe. UniQure, the company responsible for supplying the therapy, manufactures it in their Lexington, Mass. facility in partnership with CSL. This remedy offers hope to those who have been affected by the devastating disease it is designed to treat.

Upon its first U.S. sale, the company became eligible for a $100 million payout from CSL. To sweeten the deal, an additional $75 million will be awarded should a sale be made in one of five European countries before the summer is out.

UniQure’s next big target is a groundbreaking gene therapy for Huntington’s disease – and their recent cash infusion should be put to good use in pursuit of this cutting-edge treatment. Not only will this innovative therapy offer hope to those afflicted with the condition, it will also bring the company closer to achieving their ambitious goals.

Huntington’s has proven to be an incredibly daunting challenge for biopharma – with no known treatments to slow or even halt its progression, it remains an untreatable and ultimately fatal condition. Despite the difficulties, researchers strive to find a way to combat this devastating disease.

Earlier this month, Novartis had to abandon its program after the majority of its trial participants experienced neurotoxicity signals. This setback in the space was preceded by the shut down of Triplet Therapeutics last fall.

Twenty-six brave patients are participating in a pioneering Phase I/II study, where they are receiving a revolutionary AAV gene therapy approach developed by uniQure. Participants have been divided into two cohorts and are receiving either a lower or higher dose of the therapy. This groundbreaking study is leading the way in the field of gene therapy.

Preclinical trials have shown that a single dose of AMT-130, an AAV5 vector carrying mRNA designed to silence the huntingtin gene and inhibit the production of mutant protein mHTT, may have remarkable effects, including improved brain cell function, reversed neuropathology and partial reversal of hippocampus volume loss.

The FDA and EMA have granted the AMT-130 Orphan Drug and medicinal designations, cementing its status as a revolutionary and cutting-edge treatment option. This breakthrough development has the potential to revolutionize the way medical professionals approach treating certain rare diseases.

In January of this year, the biotech company made a bold move to expand its focus areas by picking up a gene therapy hopeful from Apic Bio that could potentially slow or reverse the progression of ALS. The one-time therapy uses a recombinant AAVrh10 vector to reduce the expression of superoxide dismutase 1 (SOD1), giving new hope to those living with the debilitating disease.

uniQure is looking forward to initiating a Phase I/II study of its new asset in the second half of 2023, a project which has already cleared the IND hurdle. This is an exciting development that could lead to a breakthrough in the field!

Leave a Comment