To disrupt current treatment practices for severe leukodystrophies by the development of a therapy that tackles the root of the disease and has the potential to be curative.
SynaptixBio was founded in 2021 to enable translation of ground-breaking research in TUBB4a Leukodystrophy into a transformative therapy. Therapeutic development is based onto novel science conceived in the laboratory of Dr Adeline Vanderver. Adeline Vanderver with a team of scientists at the Children’s Hospital of Philadelphia (CHOP) discovered the causative mutation for H-ABC. Further research has identified potential molecules to control the toxic mutation. A partnership has been established between CHOP and SynaptixBio to take a TUBB4a therapy to the clinic and beyond.
We are experienced leaders driven by the goal of finding new and effective treatments for this type of rare and underserved disease.
Dan Williams Ph.D.
Chief Executive Officer, Executive Director and Co-founder
Dan Williams is an accomplished biopharmaceuticals leader with two decades of industrial experience from bench to operational and scientific executive roles. During his career Dan has served as Vice President of Research Operations at Adaptimmune PLC. and Chief Product Officer at Meatable B.V
Michelle Teng, Ph.D.
Chief Scientific Officer, Executive Director and Co-Founder
Michelle is a science entrepreneur and rare disease advocate. She is the Founder CEO of Oxford based technology start-up, Etcembly. Michelle also co-founded the H-ABC Foundation, a charity, following the diagnosis of H-ABC in her daughter, to raise awareness of the disease and accelerate gene therapy treatments for H-ABC.
Head of Research and Preclinical Development
Laure is an experienced preclinical leader with extensive research and translational background in the biotechnology industry, passionate about helping bring drugs to patients. Before joining SynaptixBio, Laure was Head of Pipeline Biology at Immunocore, where she supported the regulatory submissions of several oncology and infectious diseases drug candidates for first-in-human studies as well as a biologics license application for the first TCR therapeutic to be approved.
Bent Jakobsen Ph.D.
Executive Director and Founder
Bent founded Avidex Ltd., Adaptimmune Ltd. and lmmunocore Ltd. where he served as Chief Scientific Officer and as an Executive Board Director. In these roles Bent was responsible for all aspects of their lead discovery and optimisation and translational programmes. He is currently the Chief Executive Officer of Accession Therapeutic Ltd. and Continuum Lifesciences Ltd. and holds non-executive director positions in several Biotechnology companies.
Nick Cross M.B.A
Chairman and Non-executive Director
Nick is a serial entrepreneur and investor with considerable Board experience in both biotech and high-tech sectors. He has co-founded and served as the Chairman of several companies including Immunocore PLC, Adaptimmune PLC, Oxford Asymmetry International PLC (now part of Evotec), Oxford Semiconductor Ltd (now part of Toshiba).
Scientific Advisory Board
We collaborate closely with leading specialists in their field.
Dr Adeline Vanderver
Children’s Hospital of Philadelphia
Dr Adeline Vanderver, MD, is an attending physician in the Division of Neurology, Program Director of the Leukodystrophy Centre, and Jacob A. Kamens Endowed Chair in Neurologic Disorders and Translational Neurotherapeutics at Children’s Hospital of Philadelphia. Under Dr. Vanderver’s leadership, the multidisciplinary Leukodystrophy Center team is focused on creating new standards of care for children with leukodystrophies by advancing leukodystrophy gene discovery, creating new therapies, and supporting and advocating for patients and their families.
Dr Tim Yu
Boston Children’s Hospital
Dr Tim Yu, MD, PhD is an Associate Professor at Harvard Medical School and Staff Physician and Group Leader at Boston Children's Hospital. His research lies at the intersection of genetics, neurobiology, and bioinformatics, which he applies to understand and treat rare paediatric neurologic diseases.
H-ABC Foundation UK
The H-ABC UK foundation was founded in Sept 2019 by Michelle Teng and Amy Sheridan-Hill, mothers to Sofia and Frankie respectively who were both diagnosed with the disease when they were toddlers. Michelle and Amy were told by clinicians that nothing could be done and just to enjoy the time with their respective children. Unwilling to accept their children’s genetic fates and frustrated by the lack of education about the disease, Michelle and Amy set up the foundation to raise funds to find a treatment and heighten the awareness of H-ABC. The foundation raised more than £100,000 during lockdown, an effort that was featured in the BBC News.
Children’s Hospital of Philadelphia
The Leukodystrophy Center at the Children's Hospital of Philadelphia (CHOP) provides comprehensive clinical care, diagnostic testing and the most advanced treatments available to infants, children and adolescents living with these inherited brain white matter diseases. Here, children have access to doctors and nurses who are experienced in caring for individuals with leukodystrophies, and who are at the forefront of ongoing research to find new treatments.