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Taysha Gene Therapies Joins the Rare Disease Company Coalition

DALLAS–()–Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced that it has joined the newly formed Rare Disease Company Coalition, a first-of-its-kind alignment of life sciences companies committed to discovering, developing and delivering rare disease treatments for the patients they collectively serve.

“Taysha is honored to be a founding member of the Rare Disease Company Coalition,” said RA Session II, President, Founder and Chief Executive Officer of Taysha. “As we grow to become a leader in the development of gene therapies for the treatment of rare diseases, we have a responsibility to our community, especially to our patients and their families, to advocate for more effective policies and regulations that will encourage innovation around potentially life-saving therapies. We look forward to working with this impressive group of companies to drive awareness about the specific challenges of rare disease research and development and promote solutions that will support timely delivery of valuable treatments to patients.”

As an education and advocacy-focused coalition of companies, the shared goal of the Coalition is to inform policymakers of the unique circumstances of the rare disease company business model, specifically focusing on the unique circumstances involved in developing and bringing to market therapies for small and differentiated patient population sizes. The Coalition will advocate for policy and regulatory frameworks that account for and recognize the distinct considerations of life science companies operating in the rare disease space and the impact it has on delivering innovative therapies to the patients they serve.

Founding members of the Coalition include:

  • Acceleron Pharma
  • Aeglea BioTherapeutics
  • Agios Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Harmony Biosciences
  • Orchard Therapeutics
  • Orphazyme US, Inc.
  • Sarepta Therapeutics
  • Taysha Gene Therapies
  • Ultragenyx Pharmaceutical

In the coming month, the Coalition will engage with policy stakeholders on potentially impactful drug and healthcare policies and regulations currently under discussion, including prescription drug pricing, to highlight the consequences that blanket legislation can have on continued innovation for rare disease treatments.

About the Rare Disease Company Coalition

The Rare Disease Company Coalition represents life science companies committed to discovering, developing and delivering rare disease treatments for the patients we serve. As an education and advocacy-focused coalition of companies, our goal is to inform policymakers of the unique challenges and promises of rare disease drug discovery, development and manufacturing for small population sizes in order for critical innovation to continue. To achieve this goal, we will use our unified voice to advocate for long-term, consistent, equitable and sustainable government policies that enable life science companies to continue to bring hope and provide access to approved treatments to people living with rare diseases. For more information, please visit

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our team’s proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platform—an engine for potential new cures—with a goal of dramatically improving patients’ lives. More information is available at


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