Putting Patients at the Center: An Overview of Taysha Gene Therapies

Quick Overview

Taysha Gene Therapies is a company focused on finding potential treatments for patients with monogenic diseases of the central nervous system. They are using proven approaches, such as AAV9 gene delivery, to bridge the gap between innovation and treatments. Taysha has a partnership with UT Southwestern, a gene therapy powerhouse, and is building its own manufacturing facility. They are currently developing a gene therapy approach for Angelman Syndrome, using AAV9 capsids to deliver a short hairpin RNA (shRNA) to reactivate the silenced maternal gene. Taysha emphasizes the importance of collaboration, transparency, curiosity, empathy, and respect in their work, and they value the partnership with patient advocacy groups like FAST. They are seeking to understand the needs of the Angelman community and are committed to delivering impactful therapies for the disease.

Introduction

In this talk, Suyash Prasad, Chief Medical Officer and Head of Research and Development at Taysha Gene Therapies, provides an overview of the company’s mission and approach to treating monogenic diseases of the central nervous system. He is joined by Emily McGinnis, Chief Patient Officer, who discusses the importance of patient advocacy and collaboration in their work.

Taysha Gene Therapies: Bridging the Gap

Taysha Gene Therapies is dedicated to finding potential treatments for patients with monogenic diseases of the central nervous system. With a focus on pediatric neurological diseases, the company aims to bridge the gap between innovation and providing therapies for children with various neurological conditions. They have assembled a team of experienced professionals in gene therapy from around the world.

The Meaning Behind the Name

The name “Taysha” holds significant meaning for the company. It was suggested by the CEO’s nine-year-old daughter, who is a Texas history buff. In the Caddo Native American language, “Taysha” translates to ally or friend, as well as Texas or Tejas. This name reflects the company’s commitment to collaboration and forming lasting alliances with the biomedical and rare disease communities.

Building Blocks for Gene Therapy Development

Taysha Gene Therapies utilizes the AAV9 gene delivery approach, which has already proven its merits in treating diseases such as spinal muscular atrophy. This technology is well-tolerated, effective, and has a well-understood manufacturing process. The company has partnered with UT Southwestern, a gene therapy powerhouse, to access their world-class gene therapy facility and manufacturing platform. Additionally, Taysha is in the process of building its own commercial-scale manufacturing facility.

Treating Angelman Syndrome with Gene Therapy

Taysha’s approach to treating Angelman Syndrome involves administering a short hairpin RNA (shRNA) to reactivate the silenced maternal UBE3A gene. The shRNA is packaged in AAV9 capsids and delivered intrathecally, directly to the cerebrospinal fluid. This method allows for optimal distribution and cell transduction within the central nervous system. The company is actively seeking input from the Angelman community to understand the most important clinical features and desired improvements in treatment.

The Development Process and Patient Partnership

Taysha acknowledges that they are in the early stages of the development process. Preclinical work involves defining the gene therapy vector’s molecular structure and determining effective and safe doses. Once clinical trials are underway and sufficient data is gathered, Taysha will work with regulatory agencies to seek approval for their gene therapy. Throughout this process, the patient community is seen as partners, collaborators, and teachers. Taysha is committed to providing updates and learning from the community’s insights.

Patient Advocacy and Collaboration

Emily McGinnis, Chief Patient Officer, emphasizes Taysha’s patient-centric approach. The company’s guiding principles include collaboration, transparency, curiosity, empathy, and respect. Taysha recognizes the importance of strong advocacy and patient communities, as well as partnerships with academic and clinical research centers. They believe that an integrated approach, working closely with organizations like the Foundation for Angelman Syndrome Therapeutics (FAST), is crucial to advancing new therapies for Angelman Syndrome.

Conclusion

Taysha Gene Therapies is dedicated to putting patients at the center of their work. They strive to develop impactful and life-changing therapies for Angelman Syndrome and other monogenic diseases of the central nervous system. By collaborating with patients, caregivers, advocacy partners, clinicians, and government stakeholders, Taysha aims to deliver innovative treatments and improve the lives of those affected by these conditions.

To learn more about Taysha Gene Therapies, visit their website at www.tayshagtx.com.