Expanded Collaboration Focuses on Discovery of Small-Molecule Drugs for
Spinal Muscular Atrophy
SOUTH PLAINFIELD, N.J., Dec. 6 /PRNewswire/ -- PTC Therapeutics, Inc.
(PTC), a biopharmaceutical company focused on the discovery, development,
and commercialization of small-molecule drugs targeting
post-transcriptional control mechanisms, today announced an expanded
research collaboration with the Spinal Muscular Atrophy (SMA) Foundation.
The collaboration builds on the existing research agreement to identify and
characterize compounds that have the potential to treat SMA by increasing
production of the survival motor neuron (SMN) protein, the absence of which
causes SMA. Under the terms of the expanded agreement, the SMA Foundation
will provide an additional $1.6 million in funding to PTC based upon
completion of certain milestones.
(Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
"We are pleased to expand this collaboration with the SMA Foundation,"
stated Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC
Therapeutics. "By applying our proprietary technologies, we have made
important progress in our efforts to identify treatments for SMA. This
program is another example of the breadth of our approach and we look
forward to our continued collaboration with the SMA Foundation as we seek
to develop treatments for disorders with high unmet medical need."
"PTC has been an ideal partner in the identification of molecules
targeting SMN," noted Meg Winberg, Director of Discovery Research for the
SMA Foundation. "We have been impressed with their progress and are pleased
to continue our work together to identify and advance potential treatments
for SMA."
About Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a genetic, motor neuron disease
characterized by the wasting of skeletal muscles. Caused by progressive
degeneration of nerve cells in the spinal cord, the disease leads to
increasing muscular weakness and atrophy. It is estimated that
approximately 1 in 6,000 to 1 in 10,000 infants are born annually worldwide
with SMA. SMA is often compared to polio because the same spinal cord cells
are attacked in each disease. Over time, patients afflicted by this disease
continue to lose muscle control and strength, leading to progressive
inability to walk, stand, sit up and breathe.
About the Spinal Muscular Atrophy Foundation
Founded in 2003, the Spinal Muscular Atrophy Foundation is a nonprofit
organization dedicated to accelerating progress towards a treatment and
cure for spinal muscular atrophy through targeted funding of clinical
research and novel drug development efforts. Since its inception, the
Foundation has awarded over $40 million in sponsored research agreements.
In addition, the Foundation is committed to raising awareness and
generating support for increased research efforts in SMA among the leaders
of industry and government. For more information on the Spinal Muscular
Atrophy Foundation, visit http://www.smafoundation.org or call (646) 253-7100.
About PTC Therapeutics, Inc.
PTC is a biopharmaceutical company focused on the discovery,
development and commercialization of orally administered, proprietary,
small-molecule drugs that target post-transcriptional control processes.
Post-transcriptional control processes regulate the rate and timing of
protein production and are of central importance to proper cellular
function. PTC's internally-discovered pipeline addresses multiple
therapeutic areas, including genetic disorders, oncology and infectious
diseases. In addition, PTC has developed proprietary technologies and
extensive knowledge of post-transcriptional control processes that it
applies in its drug discovery and development activities, including the
Gene Expression Modulation by Small-molecules (GEMS) technology platform,
which has been the basis for collaborations with leading pharmaceutical and
biotechnology companies such as Pfizer, Celgene, CV Therapeutics and
Schering- Plough.
SOURCE PTC Therapeutics, Inc.
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Related links:
http://www.ptcbio.com
http://www.smafoundation.org
Photo Notes:http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
CONTACT:
William Baird of PTC Therapeutics, Inc.,
+1-908-912-9159, wbaird@ptcbio.com; or Sheryl Seapy of Pure
Communications for PTC Therapeutics, Inc., +1-949-608-0841,
sheryl@purecommunicationsinc.com
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