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.
Chairman, Chief Science Officer
Dr. Samulski co-founded leading gene-replacement therapy company AskBio and helped start Columbus Children’s Foundation in 2017. Dr. Samulski has 40 years of experience in the field of gene replacement therapy and was the first researcher to transform the adeno-associated virus (AAV) into a safe and effective vector for delivering corrected genes in vitro.
Today, most researchers use the AAV vector to develop potential gene-replacement therapy. Dr. Samulski helps identify the most promising targets worldwide and choose which ones the Foundation will sponsor.
Sheila Mikhail, J.D., MBA
Sheila Mikhail co-founded AskBio and is its current CEO. She previously served as CEO of NanoCor Therapeutics. Ms. Mikhail co-founded and was CEO of Bamboo Therapeutics which advanced a therapeutic for GAN[MH1] into the clinic. Bamboo was acquired by Pfizer in August 2016.
Trained as a lawyer, Ms. Mikhail practiced law for over 15 years, including at a law firm that she founded, Life Sciences Law, which serviced clients such as Bayer, Gilead, GSK, Sanofi, and Aventis. Ms. Mikhail also practiced with Ropes and Gray in Boston. Ms. Mikhail 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.
Krystof Bankiewicz, M.D., Ph.D.
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.
Javier Garcia, MBA
Javier García is the CEO of Viralgen in Spain and has spent almost 30 years working in healthcare and the biopharmaceutical industry. He is focused on life science venture capital investing.
He is a founder and board member of Aura Biosciences and AskBio. He also serves on the boards of the National Alliance for Hispanic Health and the Healthy Americas Foundation. Mr. Garcia is the founder of Fundación Columbus in Valencia, Spain.
Damià Tormo, Ph.D.
Damià Tormo has spent the last 12 years working in healthcare and the biopharmaceutical industry. He is focused on life science venture capital investing.
Dr. Tormo currently serves on the boards of the following companies: Artax Biopharma, Bioncotech Therapeutics (Chairman), Polymer Therapeutic Solutions, and Nubesis. He is also a board member of Fundación Columbus, the sister foundation of Columbus Children’s Foundation.
Matt Kane, MBA
Matt Kane is CEO and co-founder of Precision Biosciences, a genome editing company dedicated to improving life ($DTIL) through the proprietary genome editing platform ARCUS. Precision is actively developing product candidates in three innovative areas: allogeneic CAR T immunotherapy, in vivo gene correction, and food.
He has over 15 years of experience in the life sciences industry and was formerly with Suros Surgical Systems, later acquired by Hologic. When he isn’t working, you can find Matt enjoying time with his wife and daughters or hiking with his dog.
Robert Long is a partner at Piedmont Capital Investments, a family backed firm which makes investments in venture stage technology and life science companies.
He is also a partner of 36 North Capital, a privately held merchant bank providing select advisory services to family offices and private companies. Robert was formerly co-founder of A2 Access LLC, a financial services technology firm that was acquired by Dealogic, a Carlyle Group portfolio company.
Laura Brod Hameed
Laura Brod Hameed brings two decades of experience in the fields of bioscience management, non-profit leadership, higher education governance and state government to her role as Executive Director. Most recently, Ms. Hameed served as Chief Executive Officer of RoverMed BioSciences.
Prior to that, she was Chief Executive Officer of GeneSegues Therapeutics. Additionally, Ms. Hameed is an Aspen-Rodel Fellow with the Aspen Institute and serves on the board of Givvor, a cloud-based platform for online giving, and Drake Bank, which specializes in small-business lending.
Director of Outreach
Laurie brings three decades of service in federal and state government.
Laurie most recently served as Chief of Staff to a highly-regarded senior member of the House Ways and Means Committee. As Chief of Staff, she led the Congressman’s team as they navigated complex legislative challenges, including repeal of the medical device tax and comprehensive tax reform. She worked on regulatory issues with the FDA, CMS and other federal agencies on compliance, reimbursement and classification issues. She also worked with the House Rare Disease Caucus and Diabetes Caucus.
Before her federal service with the Congressman, she served in a variety of capacities in the Minnesota state government. She served as Chief of Staff for a Minnesota House Caucus. She also served as the Deputy Commissioner for the Minnesota Department of Commerce, which regulates financial institutions.
Robert Armstrong, Ph.D.
Gloria Gonzalez-Aseguinolaza, Ph.D.
Philippe Moullier, MD, Ph.D.
Sharon Hesterlee, Ph.D.