Putting Patients at the Center
Taysha Gene Therapies, a company focused on developing gene therapy treatments for rare neurological diseases, presented their approach to treating Angelman syndrome at the 2021 FAST Science Summit. They have two programs in development, one involving gene replacement therapy and the other using a knockdown approach. Both programs utilize the AAV9 capsid, HEK-293 mammalian cell suspension, and intrathecal delivery. Taysha emphasizes collaboration with academic partners and patient advocacy groups to ensure their treatments meet the needs of patients and families. They are currently in the preclinical research phase and plan to have discussions with regulators in 2022. Taysha’s goal is to bring a gene therapy option to patients with Angelman syndrome.
In this talk, we will discuss Taysha Gene Therapies, a company focused on developing gene therapy treatments for rare and severe neurological diseases, particularly in children. We will provide an overview of Taysha and its approach, as well as discuss the specific program for Angelman syndrome. Additionally, we will touch on the company’s partnership with the University of Texas Southwestern and highlight the importance of collaboration with the patient community.
Taysha Gene Therapies
Taysha Gene Therapies is a gene therapy company that was founded close to two years ago. The company leverages proven gene therapy platforms, specifically the AAV9 capsid and the HEK-293 mammalian cell suspension, to develop treatments for a range of pediatric neurological diseases. Taysha has a collaborative partnership with the University of Texas Southwestern, where early proof of concept discovery work is conducted before transitioning to preclinical IND-enabling studies. The company’s executive team has extensive experience in rare and severe neurological diseases, as well as gene therapy.
Three Main Tenets
Taysha Gene Therapies focuses on three main tenets across its range of programs. These tenets provide a common foundation for the company’s approach. Firstly, the company exclusively uses the AAV9 capsid, which has extensive clinical experience and proven efficacy. Secondly, Taysha utilizes the HEK-293 mammalian cell suspension process for manufacturing gene therapies, as it has demonstrated success and scalability. Lastly, all drugs developed by Taysha are administered intrathecally, delivering the gene therapy directly to the brain and spinal cord. This approach has shown clinical precedent and effective delivery to the target areas.
Partnership with UT Southwestern
Taysha Gene Therapies has a strong collaborative partnership with the University of Texas Southwestern (UTSW). Led by gene therapy researcher Stephen Gray and pediatric neurology division chair Berge Minassian, UTSW conducts early proof of concept and discovery work for Taysha’s programs. The university’s expertise and resources contribute to the development of gene therapy treatments, which are then advanced by Taysha through preclinical IND-enabling studies and into clinical trials. The partnership allows for a seamless transition from research to clinical development.
Portfolio of Programs
Taysha Gene Therapies has a diverse portfolio of programs focused on different disease categories. The neurodegenerative disease franchise includes conditions characterized by ongoing progressive destruction and loss of neurons. The neurodevelopmental diseases franchise encompasses disorders with brain dysfunction that require correction to alleviate symptoms. The genetic epilepsies franchise focuses on early-onset seizures and developmental delays. While these franchises are distinct, they are interconnected, and Taysha’s platform approach allows for cross-learning and optimization across programs.
Angelman Syndrome Programs
Taysha Gene Therapies has two programs specifically targeting Angelman syndrome. One approach involves gene replacement therapy, where the UBE3A gene is replaced to restore proper protein expression. The other approach utilizes short hairpin RNA (shRNA) to knock down the antisense transcript and allow expression of the UBE3A gene through the paternal allele. Both programs utilize the AAV9 capsid, the HEK-293 mammalian cell suspension, and intrathecal delivery. These programs are currently in the preclinical stage, with proof of concept studies being conducted in animal models. Taysha plans to engage in discussions with regulators in 2022 to prepare for clinical trials.
Collaboration with Patient Advocacy Groups
Taysha Gene Therapies recognizes the importance of collaboration with patient advocacy groups and the patient community. The company has developed patient guiding principles that emphasize collaboration, transparent communication, curiosity, empathy, and respect. Taysha actively collaborates with advocacy partners, such as the Foundation for Angelman Syndrome Therapeutics (FAST), to understand unmet needs, identify educational gaps, gain disease expertise, and co-create educational resources. The insights gained from these partnerships inform the development of clinical trial programs and ensure that the needs of patients and families are met.
Taysha Gene Therapies is committed to putting patients at the center of its work. Through its collaborative partnerships with academic institutions and patient advocacy groups, the company aims to develop effective gene therapy treatments for rare and severe neurological diseases. The focus on common tenets, such as the AAV9 capsid, the HEK-293 mammalian cell suspension, and intrathecal delivery, allows for efficient development and optimization of therapies. Taysha’s programs for Angelman syndrome are progressing through preclinical studies, and the company plans to engage with regulators in 2022 to prepare for clinical trials. The collaboration with patient advocacy groups ensures that the needs and perspectives of patients and families are incorporated into the development process.
- Title: Putting Patients at the Center
- Author(s): Emily Mcginnis, Suyash Prasad
- Author(s)’ affiliation: Taysha Gene Therapies
- Publication date: 2022-01-04
- Collection: 2021 FAST Science Summit