Novo Biosciences based of Bar Harbor, Maine is a regenerative medicine company
developing small molecule therapies that reactivate and stimulate innate healing abilities in humans and animals.
Some research efforts are closer to clinical implementation, including developing improvements in clinical management of rare diseases and trials of
small molecule therapies for inherited and mosaic genetic disorders.
A fruit fly model of Duchenne muscular dystrophy allowed Ruohola - Baker's lab to rapidly
score small molecule therapy candidates for raising the level of sphingosine 1 - phosphate.
Regenerative
medicine small molecule therapies offer many advantages over other therapeutic approaches including reduced complexity, ease of administration, ready reversibility, fewer regulatory hurdles and the absence of ethical concerns like those that have challenged the stem cell field.
20 - 30 % of patients with acute myeloid leukemia have mutations of the FLT3 biomarker that make them eligible for several FLT3 -
targeted small molecule therapies currently in late stage clinical trials.
«I expect that both arms of immunotherapy — cell therapies and
small molecule therapies — will move forward.