GEMMA Biotherapeutics is focused on advancing gene therapy research to provide innovative treatments for individuals with rare diseases. The company collaborates with Franklin Biolabs to expedite the development and global distribution of these advanced therapies. Central to their approach is the use of adeno-associated viruses (AAV) as gene transfer vehicles, which have been engineered to enhance their effectiveness in delivering therapeutic genes. This technology has been pivotal in the resurgence of gene therapy, enabling GEMMA Biotherapeutics to work on a diverse range of inherited conditions, including cystic fibrosis and various metabolic and neurological disorders.

Under the leadership of Dr. James M. Wilson, a pioneer in the field of gene therapy, GEMMA Biotherapeutics has achieved significant milestones, including the development of three FDA-approved AAV-based therapies and over 40 active programs. The company holds an extensive portfolio of patents related to gene therapy technologies, reflecting its commitment to innovation in this space. With a strong emphasis on addressing unmet medical needs, particularly in marginalized populations, GEMMA Biotherapeutics aims to transform the landscape of treatment options available for rare diseases, ensuring faster access to life-changing therapies for patients worldwide.

United States

GEMMABio, a new therapeutics company, will serve as the research and innovation arm to speed the research of and global access to life-changing advanced therapies for those living with rare diseases. 

Alongside Franklin Biolabs, GEMMABio will advance gene therapy research and deliver therapeutics to patients around the world significantly faster.

GEMMA Biotherapeutics

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