Rewriting the future
for kids with ultra-rare diseases
Accelerating access to gene therapy for ultra-rare genetic disorders.
Since the discovery and development of new, innovative genetic medicines focus mainly on larger patient populations, we make sure that children with ultra-rare genetic conditions are afforded the same level of attention. Recognizing the potential for gene therapy to solve many of the ultra-rare genetic disorders these special children face, we bring our network of scientific experts, technology resources, and affordable manufacturing capacity to expand options with three core areas of focus:
- Providing financial support to accelerate the advancement of gene therapy clinical trials for ultra-rare genetic diseases.
- Supporting the open-source data model and standard protocols across the broader gene therapy community.
- Removing the burden for children and families to access to life-altering clinical trials around the world.
In 2017, through a collaboration between Columbus Venture Partners, Asklepios Biopharmaceutical, and Viralgen, Columbus Children’s Foundation began in Valencia, Spain. We are building a bridge for children with ultra-rare disorders to ensure no child is left behind by standard clinical processes that often only cater to homogenous populations with the highest potential for clinical success. Our chapter in Spain still operates as Fundación Columbus in Valencia. The US-based Columbus Children’s Foundation was started in 2018 and operates in Chapel Hill, NC with collaborations throughout the US and abroad.
We bridge the treatment gap for children with ultra-rare genetic diseases giving them hope to live their lives to the fullest.
We are deeply driven by the urgency to change the lives of children and their families, especially since we know help is in reach.
Meet our leadership team
R. Jude Samulski, Ph.D.
Co-founder, Chief Science Officer & Trustee
Dr. Samulski received his Ph.D. in medical microbiology and immunology from the University of Florida. His graduate work (1978-82) demonstrated the first use of AAV2 as a viral vector which culminated in the first U.S. FDA approved gene therapy drug for Leber Congenital Amaurosis (LCA). Dr. Samulski was hired at University of Pittsburgh where his lab demonstrated the first use of AAV for gene delivery to the Central Nervous System (CNS) which eventually led to the initial treatment of AAV for Canavan’s disease. In 1993, he was recruited to the University of North Carolina (UNC) to initiate, build and direct the UNC Gene Therapy Center. In this setting, development of self-complementary AAV vectors (currently used in approved SMA gene therapy drug), Chimeric AAV capsids (first example of synthetic vector used in humans – DMD trial), and critical research that enabled large scale production was carried out. Research in the Samulski Lab, headed by Dr. Xiao Xiao, Ph.D., demonstrated the first AAV-mediated long-term gene transduction in muscle (J. Virology, 1996). This finding precipitated efforts to develop 1st generation AAV vectors to treat Duchenne muscular dystrophy that is now in Phase 3 clinical trials.
In total, Dr. Samulski has worked with AAV for 40 years, and for 25 years, he was director of the University of North Carolina Gene Therapy Center. He was the scientific founder of Bamboo Therapeutics, Inc. and served as the Chief Scientific Officer and Executive Chairman of the company until its acquisition by Pfizer in 2016. Upon its acquisition, Dr. Samulski joined Pfizer, as VP Gene Therapy, to ensure the successful transition of Bamboo’s Duchenne muscular dystrophy therapeutic. Dr. Samulski is a former member of the Recombinant DNA Advisory Committee (RAC), a committee tasked with assisting the FDA with gene therapy clinical trial approvals in the U.S. He also frequently serves as a gene therapy consultant to the FDA. In 2008, Dr. Samulski was recognized by the American Society of Cell and Gene Therapy with their inaugural Outstanding Achievement Award for his work and later served as President of the Society. He was also invited by Pope Francis to the Vatican in recognition for his work in the treatment of Canavan’s disease.
Dr. Samulski has advanced therapeutics into human clinical trials for hemophilia, Duchenne muscular dystrophy, giant axonal neuropathy, Pompe disease and congestive heart failure, to name a few. He has published more than 350 articles and holds more than 300 patents related to AAV technology. Currently, Dr. Samulski is Alumni Distinguished Professor in the Department of Pharmacology at UNC, Co-founder & Chief Scientific Officer of Asklepios Biopharmaceutical (AskBio) and Co-founder and Chief Science Officer of Columbus Children’s Foundation that has focused on developing AAV gene therapies for “ultra-rare” disorders.
Krystof Bankiewicz, M.D., Ph.D.
Co-founder, Chief Medical Officer
Dr. Bankiewicz holds an M.D. from Jagiellonian University in Krakow, Poland, and a Ph.D., D.Sc., from the Institute of Neurology and Psychiatry in Warsaw and trained at National Institutes of Health in Bethesda, MD. Dr. Bankiewicz is a world-renowned expert in neuro-restorative medicine, with successful translation of multiple gene therapies to the clinic.
Previous positions included 20 years tenure as Kinetics Foundation Chair in Translational Research and Professor in Residence of Neurological Surgery and Neurology at the University of California at San Francisco and, currently, Professor of Neurosurgery and Gilbert and Kathryn Mitchell Endowed Chair at The Ohio State University. Dr. Bankiewicz has both industry and academic experience, is an inventor on numerous patents and has published more than 230 peer-reviewed research articles and co-founded 3 companies.
Sheila Mikhail, J.D., MBA
Co-founder, Executive Director & Trustee
Sheila Mikhail has over 20 years of biopharmaceutical leadership experience and is a co-founder of Asklepios Biopharmaceutical (AskBio), a company she started in 2001. She served as the Chief Executive Officer of AskBio until March 2023 and currently serves in the role of Advisor/Co-Founder. During her tenure as CEO, she built three GMP manufacturing facilities in Spain and AskBio’s excess capacity is sold under the tradename Viralgen for CDMO services and TAAV for plasmid supply. With $235m in Series A funding, she built the company to over 349 employees operating in five countries. Under her leadership, AskBio has advanced programs into the clinic for Pompe, late-stage Heart Failure, Parkinson’s, Multiple System Atrophy, and Huntington’s. In December 2020, Ms. Mikhail led the negotiation of the $4b AskBio acquisition by Bayer AG and has since grown the company to over 800 employees worldwide. Ms. Mikhail co-founded and now serves as Executive Director of Columbus Children’s Foundation focused on providing medical treatment for children with ultra-rare diseases world-wide.
Prior to her role at AskBio, she served as Chief Executive Officer and co-founder of Bamboo Therapeutics which, in 20 months, raised $50m, advanced a therapeutic for GAN into the clinic, completed pre-IND studies for a Duchenne muscular dystrophy therapeutic, and built a GMP manufacturing facility. Bamboo was acquired by Pfizer in August 2016 in a deal valued at $827m. Prior to that, she was part of the management team at Chatham Therapeutics which was acquired by Baxter in 2014. Ms. Mikhail practiced law for more than 15 years. During that time, she founded Life Sciences Law which serviced clients including Bayer, Gilead, GSK, Sanofi and Aventis. She also practiced with Ropes and Gray in Boston. Ms. Mikhail was recognized as the Ernst & Young 2022 Overall National Entrepreneur of the Year. She earned a JD with honors from Northwestern University, a finance MBA with honors from the University of Chicago, and a BS with highest honors from the University of Illinois at Urbana-Champaign.
Javier Garcia, MBA
Trustee
Javier García, Co-Founder and General Partner of Columbus Venture Partners, worked for 25 years at Eli Lilly and Company where he held various executive positions in Business Development and Information Systems. Since 2009, he has focused on venture capital investment, particularly in private companies in the biopharmaceutical, life sciences, and medical technology sectors.
He serves on the board of several biotechnology companies, including Highlight Therapeutics, Vivet Therapeutics, Viralgen Vector Core (as Chairman), Cocoon Therapeutics, Syngoi, and NeRRE.
He is also the Founder and Secretary of the Columbus Foundation and Trustee of Columbus Children’s Foundation, which are dedicated to the development of advanced therapies for ultra-rare diseases. He is a trustee of the National Alliance for Hispanic Health and the Healthy Americas Foundation, both based in Washington DC. Additionally, he is a trustee of the Querer Foundation.
Javier García has a Bachelor’s degree in Mathematics (Statistics and Operations Research) from the Complutense University of Madrid and studied an MBA at ICADE (Spain). He is the Tambor de Oro 2023 of the City of San Sebastian.
Scientific Advisors
Robert Armstrong, Ph.D.
Gloria Gonzalez-Aseguinolaza, Ph.D.
Philippe Moullier, MD, Ph.D.
Sharon Hesterlee, Ph.D.