Page 5 - 2019 Fall CMTA Report

NEW GENE THERAPY DEVELOPMENT PROGRAM

FOR CMT2A ANNOUNCED

he CMTA and Passage Bio by tackling the underlying genetic
VIRUS VECTOR
announced September 9 the VIRUS VECTOR WITH HEALTHY GENE cause. Passage Bio will develop this
T licensing of a gene therapy experimental therapy, designed to
development program for CMT2A restore the normal function of the
under the company’s research, MFN2 gene, which is mutated in
HEALTHY GENE
collaboration and license agreement INJECTED INTO CELLS patients with CMT2A, and we look
with the University of Pennsylvania. forward to initiating a clinical trial in
Passage Bio is a privately held, the near future.”
fully integrated genetic medicines CMTA Board Chair Gilles
company with a mission to develop GENE THERAPY DELIVERY Bouchard said, “Just one year after
a portfolio of life-transforming AAV we formally launched our gene ther-
(Adeno-associated virus)-delivered ther- apy program, we are witnessing two
apeutics for the treatment of rare monogenic central nervous major players in the field working col-
system diseases. The company is based in Philadelphia and laboratively to develop potential treatments for one of the
has a research, collaboration and license agreement with more common types of CMT. We are delighted to partner
the University of Pennsylvania and its Gene Therapy Pro- with Passage Bio and Penn in this effort and to contribute
gram (GTP), as well as the Orphan Disease Center at Penn. key elements of the Strategy to Accelerate Research (STAR)
“CMT2A affects almost all of the severe dominant CMT2 program, such as pre-clinical and clinical assets, access to
cases and patients suffering from this rare disease experience top CMT experts and engaging the CMT community.”
progressive muscle atrophy of legs and arms, with no FDA- The clinical trial is anticipated to be a global, open
approved curative or symptomatic medications available,” label, multicenter, dose escalation study to evaluate the
said Dr. Stephen Squinto, co-founder and interim chief execu- safety, tolerability and exploratory efficacy endpoints in
tive officer at Passage Bio. “The Gene Therapy Program at subjects with CMT2A. h
Penn has developed AAV vectors and delivery methods to tar-
get the nerve cells that are affected in CMT2A, raising the
possibility of slowing or preventing progression of the disease

CMTA Board Approves Extension of CMT2E Project

he CMTA Board of Directors voted June 18 to award $96,803 in second-phase funding to
T Ron Liem, PhD, for continued testing of an FDA library of compounds aimed at reducing
the neurofilament aggregation of CMT2E. The money will be used to screen the remaining
35 percent of an FDA-approved library (360 compounds) and to test four HDAC6 inhibitors.
Screening a library of compounds pre-approved for other purposes can expedite the drug
discovery process.
Researchers at Liem’s Columbia University lab are looking for compounds that reduce the
neurofilamentous swellings observed in a mouse model of CMT2E. Their strategy is twofold:
to identify potential drug targets for treatment of CMT2E, and perhaps other CMTs, and to
identify FDA-approved drugs that can be repurposed for treatment of CMT.
The CMTA’s reviewers said the proposed experiments are a natural progression of
Liem’s initial work and are necessary to move leads further in the development pipeline.

Ronald Liem CMTA CEO Amy Gray said the newly approved project “aligns nicely with the
CMTA’s Strategy to Accelerate Research (STAR).”

FALL 2019 THE CMTA REPORT 5