uniqURE Acquires Life-Changing Gene Therapy to Combat SOD1-ALS

On Tuesday, uniQure announced an exciting collaboration with Apic Bio to develop and commercialize APB-102, a potential therapeutic treatment for a rare, genetic form of ALS. This partnership opens up a new opportunity to bring much-needed relief to those suffering from this debilitating disease.

APB-102 is a revolutionary gene therapy designed to bring hope to those living with familial ALS. This groundbreaking therapy is designed to treat the 20% of ALS patients who have the SOD1 gene mutation and the 2% of all ALS patients whose condition is caused by it. It is a one-time treatment with the potential to bring profound relief to those affected by this devastating disease.

Harnessing the power of the AAVrh10 vector, a novel therapy has been developed to combat the progression of ALS. This vector expresses a miRNA molecule, which has the ability to reduce the expression of SOD1 and thus slow or even reverse the debilitating effects of the condition.

Ricardo Dolmetsch, Ph.D., President of Research and Development, emphasized that the newly announced therapeutic, APB-102, is “highly complementary” to uniQure’s current gene therapy, AMT-161, which is currently in preclinical development to treat ALS caused by mutations in the c9orf72 gene. He further noted that the therapeutic is strategically aligned with the company’s pipeline to deliver the most effective treatments to those afflicted with the disease.

Dolmetsch recently announced that APB-102 will be re-branded as AMT-162 and uniQure is aiming to launch a Phase I/II trial for the gene therapy in the latter half of 2023. Get ready to experience the cutting-edge of gene therapy technology!

Targeting Familial ALS

AMT-161 may soon make history as the very first therapy to be approved for a genetic cause of ALS, but the race is on: Biogen and Ionis’ tofersen are hot on its heels. Will AMT-161 be the first to cross the finish line? Only time will tell!

The FDA’s Peripheral and Central Nervous System Drugs advisory committee is set to discuss a potential breakthrough in the fight against SOD1-ALS on March 22nd. Tofersen, an experimental therapy, could soon be available to those suffering from the rare and debilitating disorder. With any luck, this could be the first step towards a brighter future for those affected by SOD1-ALS.

The FDA’s AdComm is set to make a crucial decision on whether to grant accelerated approval for a promising new drug candidate. If approved, this would be a major milestone for the drug, advancing its path to becoming available to those who need it.

Biogen submitted for approval of tofersen, an anti-sense therapeutic aiming to reduce the production of SOD1 protein, largely based on neurofilament light chain (NfL) as a surrogate biomarker. This is in contrast to uniQure’s recently acquired candidate, which promises to provide a unique solution to a range of medical conditions.

The road to this milestone has been filled with challenges, hinting that the future of uniQure’s AMT-162 may be just as tumultuous.

In October 2021, Biogen made headlines when the FDA granted their New Drug Application for tofersen Priority Review, even though the Phase III VALOR trial had failed to meet its primary endpoint. Despite this, the company is forging ahead with their plans to make the drug available to the public.

In September 2022, Biogen released an extensive review of the VALOR trial and open-label extension study, which revealed that tofersen was effective in slowing the progression of the disease. These results provided a strong defense of the candidate’s potential efficacy.

The FDA is set to make their decision on tofersen on April 25 – a potential game-changer in the world of Alzheimer’s disease treatments. It’s a date many have been eagerly awaiting, as the drug could revolutionize the way we treat this debilitating disease.

In September 2022, Amylyx’s Relyvrio became the latest addition to the small but mighty group of FDA-approved treatments for ALS. With just three treatments available, this new hope for those living with the debilitating motor neuron disease is a welcome breakthrough.

Leave a Comment