April 15, 2024

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Astellas Targets Pompe Diseases after Gene Therapy’s Frustrating

Astellas Targets Pompe Diseases after Gene Therapy’s Frustrating


Astellas Targets Pompe Diseases after Gene Therapy’s Frustrating.

On February 22 , at the 19th Annual WORLDSymposium in Orlando , Astellas commented on the ongoing phase I/II FORTIS clinical trial evaluating gene therapy AT845 in adults with late-onset Pompe disease ( LOPD ) Preliminary safety and efficacy data were updated.


Astellas Targets Pompe Diseases after Gene Therapy's Frustrating.

As of the data cutoff date of September 15 , 2022 , four participants received a one -time intravenous infusion of AT845 , two at 3×10 13 vg/kg and two at 6×10 13 v/kg. kg dose administration, safety follow-up in clinical trials up to 78 weeks.

Three of the four subjects discontinued enzyme replacement therapy ( ERT ) after AT845 application, and subjects ‘ measured functional outcomes remained stable at 19 , 44 and 51 weeks after ERT was discontinued.

Ongoing assessments of dosing participants, including those who discontinued ERT , showed continued stabilization of subjects’ functional disease endpoints, including forced vital capacity and 6 -minute walk tests. Patient-reported fatigue and daily activity results were also stable.


The infusion of AT845 was well tolerated by each subject , and most treatment-emergent adverse reactions were mild ( Grade 1 ) and considered unrelated to the study treatment.


Previously, a grade 2 peripheral sensory polyneuropathy event was reported in one subject in the 6 x 1013 v/kg dose cohort , leading to a clinical hold by the FDA in June 2022 . The FDA lifted this clinical restriction in January 2023 .


AT845 , which Astellas is developing , is a novel gene replacement therapy using an AAV8 vector under a muscle-specific promoter to deliver a functional copy of the GAA gene for the treatment of LOPD in adults .

AT845 is being studied to determine whether it can deliver a functional GAA gene that is efficiently transduced to directly express GAA in disease-affected tissues , including skeletal and cardiac muscle.


Dr Jordi Díaz Manera, Clinical Geneticist Professor of Neuromuscular Diseases, Newcastle Hospitals NHS Foundation Trust, Newcastle University, UK, said: “There is an urgent need for new treatments in progressive, debilitating, genetic disorders such as LOPD, where current treatments The standard regimen, enzyme replacement therapy (ERT), has not met the need. AAV gene therapy has the potential to be a safe and effective way to deliver a functional GAA gene directly to the muscle tissue of LOPD patients.”


Astellas has had a rocky ride in gene therapy development. Most notably, Astellas ’ gene therapy AT132 for X -linked myotubular myopathy ( XLMTM ) has resulted in multiple child deaths in clinical trials , which has also been repeatedly stopped by the FDA .





Astellas Targets Pompe Diseases after Gene Therapy’s Frustrating.

(source:internet, reference only)

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