Rachel McMinn, Neurogene, Inc.

Rachel McMinn PhD

Founder & CEO, Neurogene Inc.

Dr. McMinn founded Neurogene in January 2018 with the mission of providing genetic medicines to improve the lives of neurologically-impaired and developmentally-delayed children and their families. Dr. McMinn currently serves as President and CEO of Neurogene. Prior to this, she spent 17 years in the biotechnology industry, most recently serving as the Chief Business and Strategy Officer of Intercept Pharmaceuticals, a company dedicated to patients with serious liver disease. Dr. McMinn was also an award-winning biotechnology analyst, with 13 years of experience at firms including Bank of America Merrill Lynch, Cowen, and Piper Jaffray. She earned her doctorate from The Scripps Research Institute and was awarded a Post-Doctoral Miller Fellowship at the University of California, Berkeley.

Rachel McMinn Neurogene TRNDS

TRNDS Speaker Spotlight: Rachel McMinn

Meet TRNDS speaker Rachel McMinn, CEO and President of Neurogene. Dr. McMinn founded Neurogene with a mission to bring life-changing medicines to patients and families affected by rare neurological disease.

Why are you participating in TRNDS 2019?

McMinn: I think it is a great opportunity to explain how companies and academia can work together to advance much needed therapies to patients and their families.

What are biggest opportunities for accelerating therapeutic development in the 21st century?

McMinn: Genetic medicines across the board—gene therapy but also related technologies that are based on understanding the genetic driver of disease. Also, more easily identifying patients through broad genetic testing for rare neurological disease prior to disease onset.

How is the increasing use of modern technologies changing the field of clinical research?

McMinn: Genetic testing is revolutionizing research. Machine learning is being used as an accepted tool for patient identification.

Share with us some of the most exciting developments in your organization.

McMinn: Pioneering a model to advance a broad pipeline of medicines for patients with rare disease that would be typically overlooked by pharma companies.

What is a critical topic you’d like to see discussed at TRNDS 2019?


  • How academics and industry can best work together to advance treatments for patients.
  • Discuss clinical trial patient selection for rare neurological diseases.
  • Debate the pros and cons of the appropriate treatment windows for these patient populations.
  • Greater sharing of data (foundations and institutions) to identify patients and understand the patient journey.

What challenges lie ahead in the rare disease space?


  • Regulatory challenges to streamline costs and regulations to develop therapies in an expeditious and affordable manner.
  • Biotech/gene therapy companies pricing for therapies aimed for rare diseases.
  • On the payer front, refining portability and value-based payment models. Fine tuning of these pricing models would ease the amount of risk payers take while increasing the likelihood that patients will have access to the potential curative therapies.

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