AskFirst: A New Collaborative Program to Support Rare Disease Patients in Gene Therapy Clinical Trials


Asklepios BioPharmaceuticals (AskBio) was first founded in 2001. It is based out of North Carolina.  This company is dedicated to the research of gene therapies as potential therapeutic options for rare genetic diseases. Their cell manufacturing process is called Pro10. Additionally, they have AAV and a promoter library.

Some of the conditions they are currently researching are Pompe disease, congestive heart failure, hemophilialimb-girdle muscular dystrophy, and Duchenne muscular dystrophy (DMD).


AskFirst is a new program developed by AskBio. Its aim is to support patients and families who could potentially benefit from their research in gene therapy. It’s a patient-centered approach, keeping patient needs at the forefront of the mission. This program has three primary goals.

  1. Inform patients
  2. Accurately communicate safety information
  3. Provide support to clinical trial participants

A key participant in this new program is Mathew Alsante, the new Director of Patient Advocacy at AskBio. His career has been dedicated to being a patient advocate. He has previously served as the Director of the Sarcoma Foundation of America and the CEO of the National Pancreas Foundation.

He is determined to keep the patient and their experiences, needs, and desires, central. He explains that it’s vital to research at every stage.

If you have questions about this new program you can email using [email protected]. You can also sign up for their newsletter for more information.

You can read more about this new program here.

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