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Project Catalyst

Initiated by Parent Project Muscular Dystrophy (PPMD) in 2003, Project Catalyst was born out of a belief that the present generation of young men with Duchenne muscular dystrophy deserves a decisive and positive response to the challenges they face.

Project Catalyst is a first-of-its-kind research initiative designed to identify new drugs to treat this generation of young men. Driving this initiative is the collaboration between PPMD and PTC Therapeutics, Inc. (PTC), a biopharmaceutical company focused on the discovery, development, and commercialization of small-molecule drugs.

PPMD is committed to its goal of identifying treatments for 100% of boys with Duchenne. Building on the positive working relationship developed through the research of PTC124, PPMD and PTC have collaborated to apply PTC’s proprietary drug discovery technology, GEMS (Gene Expression Modulation by Small-molecules), to identify and develop new therapies for Duchenne.

(For more information about PTC or the GEMS technology, please visit PTC’s website

The current goal of Project Catalyst is to identify and characterize compounds that modulate expression of five targets with the potential to treat patients with Duchenne. To accomplish this goal, PTC put the original five PPMD-designated targets (alpha-7 integrin, utrophin, muscle-specific isoform of insulin-like growth factor, myostatin, and phospholamban) through GEMS high-throughput screen. They then narrowed it down to the following three targets:

You can follow along with the progress of these targets by reading the quarterly reports provided by PTC Therapeutics.

Click here to read report for August 2009
Click here to read Ellen Welch's presentation from the 2009 Annual Connect Conference
Click here to read report for May 2009
Click here to read goals for 2009
Click to read report for Q4 2008
Click to read report for Q2 2008
Click to read report for Q1 2008

To learn more about these targets and about Project Catalyst, visit PTC Therapeutics: click here.

In 2007 PTC received a $15.4 million grant from NIH to further the research efforts of PPMD’s Project Catalyst program. What began as an initial investment of $3 million has to date leveraged another $3 million in private investment, in addition to the $15.4 million NIH award.

Award press release

Be a Part of Project Catalyst

Parent Project Muscular Dystrophy invites you to be a part of Project Catalyst. This research program continues to move forward, but not without support and funding. Your help is needed.

For more information about Project Catalyst, contact Kimberly Galberaith at 201-944-9985 or kimberly@parentprojectmd.org.