Gene-Based Therapies for Neurodevelopmental Disorders: A Joint Effort Between Biotech, Academia and Patient Groups
Speaker: Yael Weiss, MD, PhD, Chief Executive Officer, Mahzi Therapeutics
Date: September 24, 2024, noon-1 p.m.
Location: Roy Blunt NextGen Precision Health Building, Atkins Family Seminar Room
Recording
Description
"Just one change in one nucleotide can change the trajectory of a patient's life and their family’s life."
Yael Weiss, M.D., Ph.D.
Up until about ten years ago, if a child was diagnosed with a seizure disorder, autism or other intellectual disability, they were sorted into broad categories that weren’t well understood. In the last ten years, more than 700 genes have been identified which cause those disorders. Effectively, this means there are actually hundreds of conditions which can be studied in a more precise way, giving rise to advocacy organizations built around each of them.
In her lecture, Dr. Weiss will discuss her role as a mediator between patient groups, biotech industry and academic scientists who are working together to find solutions for these narrow neurodevelopmental disorders. To demonstrate how this work is done, she’ll also explain the different approaches available for treating genetic disorders (gene therapy, antisense oligonucleotide, or small molecule) and how scientists determine which new treatments show the most potential.
In Her Own Words
With over 10,000 genes known to be associated with genetic disorders - hundreds of which were identified in the last 5-10 years - gene-specific patient advocacy groups are rising to the challenge of developing therapies for themselves and their loved ones. Due to lack of experience and resources, many of these efforts are not productive.
In my talk, I will lay out the various therapeutic options for genetic disorders and the advantages and challenges of each. I will also address the topic of collaborating with patients and their families to streamline and improve the chances of successful clinical trials and commercializing products.
About the Speaker
Dr. Weiss Is currently CEO of Mahzi Therapeutics, a company focused on the development of therapies for ultra-rare genetic neurodevelopmental disorders. Mahzi works closely with patient foundations to support their journey towards drug development and bring programs into Mahzi once pre-clinical proof of concept is established.
She completed her MD at Hadassah Medical School at the Hebrew University in Jerusalem and her PhD at the Weizmann Institute of Science in Rehovot, Israel. She has over 20 years of industry experience in medical/clinical and business development roles at Genzyme, Merck and Ultragenyx. Dr. Weiss is a member of ASGCT and AES translational committee, N=1 collaborative and is a 2022 Termeer Fellow. Board member/advisor to Everlum and ADNP, FOXG1 KCNT1 and Team Telomere foundations.
About the Discovery Series
The NextGen Precision Health Discovery Series provides learning opportunities for UM System faculty and staff across disciplines, the statewide community and our other partners to learn about the scope of precision health research and identify potential collaborative opportunities. The series consists of monthly lectures geared toward a broad multidisciplinary audience so all can participate and appreciate the spectrum of precision health efforts.
For questions about this event or any others in the Discovery Series, please reach out to Mackenzie Lynch at lynchmm@health.missouri.edu.