From bench to bedside faster and with greater global access…

With international coalitions to support the rare disease community…

Welcome to the future of gene therapy.

About Us


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.

James M. Wilson, MD, PhD, Founder of GEMMABio

Meet Our Founder


James M. Wilson, MD, PhD

Having started his work in gene therapy nearly 40 years ago, Dr. Wilson was recruited to the University of Pennsylvania in 1993 and created the first and largest academic-based program in gene therapy. His laboratory discovered a family of viruses from primates called adeno-associated viruses (AAV) that could be engineered to be very effective gene transfer vehicles. These "vectors" have become the technology platform of choice and have set the stage for the recent resurgence of the field of gene therapy.

Dr. Wilson has also been active in facilitating the commercial development of these new gene therapy platforms through the establishment of eight biotechnology companies. His research has been focused on rare inherited diseases, ranging from cystic fibrosis to dyslipidemias to a variety of neurologic disorders and liver metabolic diseases, and on addressing these unmet needs for patients in marginalized populations. To date, his team’s accomplishments include 3 FDA-approved AAV-based gene therapies, 40+ active programs in development, 95 patents on gene therapy-related technologies, and first-in-patient studies in 15 different diseases.

Dr. Wilson serves as President and Chief Executive Officer of GEMMABio.

Meet Our Chief Medical Officer


May Orfali, MD, MBA

Dr. Orfali is an experienced medical executive with nearly 30 years of experience in clinical development and medical affairs across multiple therapeutic areas, including oncology, infectious diseases, and rare and orphan diseases.

Having started her career in international health affairs before transitioning into pharmaceuticals and life sciences, she brings a well-rounded approach to overseeing GEMMABio’s clinical strategy and operations.

Dr. Orfali earned her Medical Degree from the University of Baghdad and a Pharmaceutical Master of Business Administration from Cambridge University.

Launched October 1, 2024


Based in Greater Philadelphia

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