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conferenceseries.com
October 26-27, 2016 Chicago, USA
Annual Congress on
Rare Diseases & Orphan Drugs
Volume 7, Issue 5 (Suppl) J Genet Syndr Gene Ther
ISSN: 2157-7412 JGSGT, an open access journal Rare Diseases 2016
October 26-27, 2016
Cellular therapies for rare diseases
Stephen Shrewsbury
Fortuna Fix, Canada
C linical programs directed at rare diseases present many unique challenges for clinical research. Most druggable targets have
been identified and exploited and the sciences of drug (be they small molecules including oligonucleotides or biologics) or
device development has dramatically advanced in recent decades. With that advance, the opportunity to develop products for large
populations of common diseases has largely disappeared. At the same time the phenotypes for many common diseases have been
split into multiple smaller populations and even genotypes focusing more and more programs on smaller populations. Planning to
complete increasingly complex studies in smaller but more homogeneous patient groups has become increasingly competitive and
costly. Against this clinical research landscape, cellular therapies as the third major branch of clinical research have arrived. Initially
working with fetal, embryonic, bone marrow, adipose or cord blood derived stem cells, the field has been less regulated than drugs
and devices, leading to a proliferation of clinics and claims that have not all been through a rigorous and appropriate review. Attention
is now evolving from the ethically and immunologically challenging programs involving allogeneic stem cells to autologous, organ
specific stem cells. This can be best achieved by generating directly reprogrammed precursor cells for that organ. These promise
greater safety and easier production, for a very complex product and will allow this third branch of medical research to start to
tackle many rare diseases where cellular regeneration may be required for clinical benefit. To capitalize on this timely opportunity,
time and cost efficient direct reprogramming to lineage-specific precursor cells is vital. With this advance, cellular therapies will take
their rightful place in the physicians’armamentarium against injury, disease and degeneration, just as healthcare costs in advanced
countries look set to spiral completely out of control.
Biography
Stephen Shrewsbury is currently an Executive Vice President of Development and Chief Medical Officer at Fortuna Fix, leading their novel cellular technology
into multiple clinical programs focused on regenerative medicine, several of which are rare diseases. He moved to lead inhaled antibiotic programs at Chiron
Corporation (in Cystic Fibrosis and other rare diseases) before becoming CMO sequentially to MAP Pharmaceuticals, Adamas Pharmaceuticals, AVI BioPharma
(now Sarepta Therapeutics; where he opened their first IND for Eteplirsen in Duchenne muscular dystrophy and planned their oligomer programs against Ebola
and Marburg Hemorrhagic Fevers) and Aquinox Pharmaceuticals. He is the author of Defy Your DNA and over 70 scientific papers, holds several patents and
contributed to many awarded grants, mostly for work on rare diseases.
Stephen Shrewsbury, J Genet Syndr Gene Ther 2016, 7:5 (Suppl)