Heartbreak to Hope: A Mother’s Mission to Cure Angelman Syndrome | Dr Allyson Berent, CSO of FAST

In this episode, Allyson Berent, Chief Science Officer, Foundation For Angelman Syndrome Therapeutics (FAST), shares her remarkable journey from a career in veterinary medicine to becoming a leading advocate and innovator in the field of rare disease drug development, inspired by her daughter’s diagnosis with Angelman syndrome. She recounts the challenges of obtaining an early diagnosis, the emotional impact on her family, and her determination to find solutions where none existed. Allison describes how she immersed herself in scientific research, connected with experts, and joined the Foundation for Angelman Syndrome Therapeutics, eventually helping to launch and lead multiple initiatives aimed at accelerating the development of transformative therapies for Angelman syndrome and similar neurodevelopmental disorders.

Throughout the conversation, Allison emphasizes the importance of true patient-focused drug development, sharing how her personal experience shaped her approach to building companies, running clinical trials, and fostering collaboration across the biotech ecosystem. She offers practical advice for entrepreneurs and executives, urging them to listen to patients and families, remain humble, and surround themselves with experts to address knowledge gaps. This episode highlights the profound impact that new therapies are having on patients’ lives and underscores the value of mission-driven innovation in advancing treatments for rare diseases.

Time Stamps

00:02 – Introduction and Allison’s upbringing and early interest in veterinary medicine.

00:15 – Allison shares the story of how she decided to become a vet and her educational journey.

01:34 – She discusses her career as a vet, starting a family, and her daughter’s diagnosis with Angelman syndrome.

04:36 – Allison describes the early signs, medical consultations, and the process of getting a diagnosis for her daughter.

11:22 – Sam and Allison discuss the challenges of delayed diagnosis and the importance of proactive care.

13:01 – She shares stories from the community and changes in genetic testing practices.

0:14:35 – Allison explains how she and her family responded to the diagnosis and her drive to find solutions.

18:18 – She describes connecting with the foundation, joining the board, and developing a roadmap for a cure.

26:15 – The process of moving from mouse model research to human clinical trials, raising funds, and starting a company.

31:12 – Discussion on what true patient-driven development means and how it shaped Allison’s approach.

39:07 – Allison details the journey of the company, partnership with Ultragenyx, and the impact of the acquisition.

45:49 – Explanation of the accelerator model, portfolio companies, and collaborative approach to drug development.

51:20 – How the accelerator shares resources and knowledge across programs and with other companies.

52:24 – Quincy’s Progress and the Impact of New Therapies,participation in clinical trials, and the real-world impact on families.

56:05 – Sam and Allison discuss the life-changing effects of new treatments for Angelman syndrome.

58:56 – Allison offers advice on keeping the patient at the center of drug development and listening to families.

1:06:13 – She shares suggestions for integrating patient stories and perspectives into company culture and meetings.

1:09:14 – Final Words of Wisdom for Entrepreneurs: Allison emphasizes humility, gap analysis, teamwork, and resilience for those starting companies.

1:11:53 – Conclusion and Thanks