Helping patients with gene therapy
Bringing gene therapeutics to patients with diseases that have a high unmet medical need fuels our research and development pipeline. We have active preclinical and clinical programs for neuromuscular, metabolic, cardiovascular and central nervous system conditions, with particular attention on rare and more common diseases.
Lisa can’t get up from a chair on her own, but she is not letting limb-girdle muscular dystrophy type 2I/R9 stop her from being a working mother and loving wife.
“…the first time I was told I had a rare form of muscular dystrophy, it was a profound moment. I wanted to know more, but they said they had no more information than the diagnosis."
Read Lisa’s story
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Embracing patients
AskFirst™ is our collaborative program for advocacy groups, patients, and their families who may benefit from the latest advances in gene therapy research and potential curative therapies. Guided by the voice of patients, we strive to advance therapeutics, technology, regulatory requirements, and public policy in this area.
We “Ask” how we can help patients and their families and welcome your voice to help us treat genetic disease.
AskFirst is driven by our mission to improve patients’ quality of life and our desire to bring better genetic medicines to market.
- Include the patient and family voice in the design of our clinical studies
- Bring together scientists, technicians, and clinical specialists who engage patients during the therapeutic development process
- Create study protocols to identify investigational therapeutic agents that demonstrate safety and efficacy.
- Engage patient advocacy liaisons with direct access to research teams.
