Page 9 - Fall 2020 CMTA Report

therapy candidates. This allows a discussions with the CMTA to but may have value in treating
company interested in positioning lead evaluation of small-molecule CMT. Regenacy accessed our
a therapy for CMT to access the approaches already advancing for testing resource to evaluate the
infrastructure needed to evaluate different but related disease areas. candidate in several CMTs,
the therapy without committing including CMT1A. Regenacy
significant time and money l Ionis Pharmaceuticals was the is currently evaluating which
upfront. A number of companies first partner to demonstrate that efforts merit further study.
are engaged in testing for CMT1A a genetic modifier of the PMP22
with us. Of the four CMT types gene (anti-sense oligonucleotide l Acceleron is a Boston-based
currently in preclinical testing, or ASO) could effectively repair company working to develop
CMT1A has attracted the highest CMT1A defects in animal mod- a biological therapy aimed at
interest due to its prominence in els of the disease. Since then, maintaining muscle. Our testing
the CMT patient population. Ionis has been working to solve a resource was accessed for clinical
generally understood limitation trials to evaluate whether the
Current CMT1A alliance activity of its technology—delivery of an therapy prolonged patient
includes the following partners: ASO to the target cells. Recently, mobility in CMT1A animals as
they have acquired from us the the disease progresses. This clini-
l Sanofi was our first alliance CMT1A stem cell lines in the cal effort was discontinued after
partner for CMT1A and we New York Stem Cell Foundation it was shown that while the
are currently evaluating small repository for use in support of ACE-083 increase muscle size,
molecules that came from this continuing CMT effort. it did not improve muscle
joint program as potential new strength. The CMTA had a
alliance partners have expressed l Regenacy owns a drug candi- strong patient advocacy presence
interest in acquiring them. date that has been in human during these clinical trials.
In addition, Sanofi has entered testing for a different disease (continued on page 10)

FALL 2020 THE CMTA REPORT 9