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First Pompe patient dosed in FORTIS Trial of AT845.

Astellas Gene Therapies (formerly Audentes Therapeutics)  announced on 5th April 2021 that the first participant has been dosed in the FORTIS trial. This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).

Nate Bachtell, VP Clinical Development, Astellas Gene Therapies

“The only approved treatment for LOPD is to replace the enzyme through chronic, repeated injections, and lack of other therapeutic options represents a significant unmet need for these patients,” Nate Bachtell, MD, vice president of clinical development at Astellas Gene Therapies, said in a short video on Facebook. 

 Link to full Nate Bachtell video

Enrollment of approximately 8 participants is ongoing

The FORTIS trial, which is expected to enroll eight participants, will be conducted at eight sites across the U.S. and in Germany and the U.K. The first patient was dosed at a site in the U.S.  Further information about the clinical trial can be found via the link below.

 Clinical Trials website

Astellas Gene Therapies was created to be the Global Gene Therapy Center of Excellence by Astellas, after its aquisition of Audentes Therapeutics.  Its aim is to advance the development of gene therapies for rare neuromuscular diseases.

Kenji Yasukawa, President and CEO, Astellas

“The field of gene therapy has made significant strides to date, and we believe the future holds even greater opportunity to bring new treatments to a wide range of diseases for patients in need,” said Kenji Yasukawa, President and CEO, Astellas. “With Astellas Gene Therapies as a Center of Excellence, we can continue to deepen our knowledge in this ever-evolving space with the expertise and talent from within Audentes to lead us to the forefront of creating next-generation treatments.”