Matt Wilsey, co-founder and CEO of Grace Science, shares a deeply personal journey after his daughter Grace was diagnosed with NGLY1 deficiency. He discusses the arduous path families face in rare disease diagnoses and the transformative advancements in genetic sequencing. Matt highlights innovative gene therapies, like AV9, aimed at treating NGLY1, and emphasizes the importance of strategic partnerships with scientists such as Nobel laureate Carolyn Bertozzi. The conversation also sheds light on navigating regulatory challenges in drug development.
Matt Wilsey's journey from a worried father to a biotech leader illustrates the emotional drive behind personal stakes in health innovations.
The collaborative efforts in Grace Science highlight the critical role of strategic partnerships and modern genetic testing in developing rare disease therapies.
Deep dives
The Devastating Beginning and Promise for a Cure
Matt Wilsey faced unimaginable fear when his daughter Grace was born in critical condition, leading him to promise to discover what was wrong. Grace was diagnosed with NGLY1 deficiency, a rare genetic disorder affecting only about 130 cases globally, compelling Matt to leverage his entrepreneurial background to create a solution. Despite having no scientific experience, Matt founded Grace Science to develop treatments for Grace and others battling rare diseases, symbolizing his transformation from a worried father to a committed leader in biotech. This journey reflects the emotional and professional drive that motivates many entrepreneurs, particularly in the health sector, where personal stakes are incredibly high.
The Diagnostic Odyssey: A Journey of Discovery
Matt described the challenging ordeal of seeking a diagnosis for Grace, often referred to as a 'diagnostic odyssey,' which took their family to numerous hospitals and experts specializing in genetic conditions. After extensive testing and consultations across multiple esteemed medical centers, they were eventually able to pinpoint the genetic anomaly responsible for Grace's condition. The advancements in genetic testing, such as whole genome and trio sequencing, played a crucial role in this discovery, demonstrating how technology has improved in the medical field over the last decade. Matt emphasized the importance of celebrating the milestone of receiving a diagnosis, as it represents both a stressful journey and a critical step towards potential therapies.
Innovation through Collaboration and Breakthroughs in Therapy
Grace Science's mission to find a treatment for NGLY1 deficiency highlights the dynamic interplay of innovation and collaboration in the biotech space. The company has progressed to clinical trials utilizing AAV9 gene therapy, which aims to deliver a healthy copy of the deficient gene into patients’ brains, demonstrating potential for both safety and efficacy. Matt's strategy of engaging leading scientists—like Carolyn Bertozzi, who contributed valuable insights into the gene's role—underscores the significance of teamwork in advancing research. As Matt navigates the complexities of regulatory processes, he views organizations like the FDA not as obstacles but as partners in the quest for effective treatments, emphasizing a collaborative approach to innovation.
In this episode, Matt Wilsey joins Jorge Conde to share the profound personal story of his daughter Grace's diagnosis with NGLY1 deficiency and how it catalyzed his journey to founding a biotech startup, Grace Science, aimed at developing a cure for his daughter’s condition.
Matt emphasizes the importance of strategic partnerships, like his collaboration with Nobel laureate Carolyn Bertozzi, and sheds light on the search for gene therapies for rare diseases.
Check out our previous episode with Carolyn Bertozzi here.
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