Transforming Lives: Our Commitment to 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.
About AskFirst™
AskFirst™ is driven by our mission to improve patients’ quality of life and our desire to bring innovative genetic medicines to market.
Our unique outreach initiative is designed for advocacy groups, patients, and caregivers seeking the latest advancements in gene therapy research and potential curative therapies. We believe collaboration and education have the power to eradicate genetic diseases—and patients and their support systems are integral at every step of the process. Together, we’re guiding efforts to advance therapeutics, technology, regulatory requirements, and public policy for genetic medicine. From designing clinical studies to engaging with research teams, our commitment to patient advocacy remains at the heart of everything we do.
Patient voices are instrumental in driving the innovation and progress that define AskFirst™. We’re applying collaboration and compassion to eradicate genetic diseases.
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“We put a lot of eggs in this basket of hope. To those working on a treatment for LGMD, time is of the essence. We are desperate for anything that might improve our sons’ daily lives.”
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Ask Kara
“In the dynamic field of gene therapy for complex diseases, the voices and
experiences of individuals and their families drive our mission to innovate,
collaborate, and ultimately, transform lives.”
Kara Witcoff
Patient Advocacy & External Engagement
