Pompe is caused by mutations in the GAA gene, which provides instructions for an enzyme called alpha-glucosidase that is involved in breaking down glycogen. AskBio is using its adeno-associated virus technology platform to develop a gene therapy for Pompe, aimed at restoring the body’s ability to produce functional acid alpha-glucosidase enzyme on its own by providing cells with a functioning copy of the GAA gene.
In addition to supporting clinical trial participants, another AskFirst goal is to ensure accurate communication of the clinical-stage gene therapy company’s product safety information.
“AskBio was founded with a patient-focused mission to develop transformative genetic medicines for patients with serious diseases previously considered untreatable,” said Martin Childers, PhD, AskBio’s chief medical officer, in a press release.
“This patient-centered approach is at the heart of our AskFirst program that embraces the voice of patients and their families that puts their welfare first. We support the important work of patient advocacy organizations that play an essential role in advancing gene therapy.”
The effort will include patients and families in clinical study design, and bring together scientists, technicians, and clinical specialists who will engage patients during therapy development. In addition, AskFirst will create study protocols to ultimately speed novel treatment delivery, build sustainable relationships with advocacy organizations, and engage patient advocacy liaisons who have direct access to research teams to get answers when needed.
To lead AskFirst, AskBio has hired Matthew Alsante to direct patient advocacy. Alsante has more than a decade of experience with patient advocacy organizations, having served as former executive director of the Sarcoma Foundation of America and, most recently, as the CEO of the National Pancreas Foundation.
“AskBio has a long history of commitment to the patient community, and I’m honored to build upon that foundation and provide an external link to the important life-changing work that is happening here,” he said. “The patient voice is vital to everything we do. It’s my job to bring that into every aspect of gene therapy development, because we can’t do it without the incredible partnerships we have with patients and the community.”
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