June 9, 2016
Cambridge, MA

Solid Grows Therapeutic Pipeline with New Disease Modifying Therapy Candidate

Solid is committed to building a robust pipeline of candidates that target the multiple facets of Duchenne muscular dystrophy. It is with this goal in mind that we are excited to announce a new collaboration with Dr. Elizabeth McNally, M.D, Ph.D., of Northwestern University to develop a novel anti-fibrotic candidate with the potential to support muscle repair.

Muscular dystrophy is characterized by the replacement of muscle with non-functional fibrotic tissue, which is caused by continuous inflammation and muscle degeneration. The goal of this program is to target a known mediator of fibrosis, or TGF-b,  and inhibit its activity. We believe that this goal can be accomplished by modulating a protein known as the latent TGF-b binding protein 4 (LTBP4).

LTBP4 has been identified as a genetic modifier of Duchenne muscular dystrophy and has been associated with a delayed loss of ambulation. If successful, we believe that a therapy utilizing this novel pathway has the potential to reduce fibrosis in Duchenne muscular dystrophy patients and slow functional decline.

The LTBP4 candidate is currently in early discovery.  Solid is excited to work with Dr. McNally and her team to unlock the potential of LTBP4 and hopefully begin to move it through development

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  • Jennifer Ziolkowski, CPA, joins as Chief Financial Officer.
  • Carl Morris, Ph.D., promoted to Chief Scientific Officer.
  • Joel Schneider, Ph.D., promoted to Chief Technology Officer, Head of Exploratory R&D.

Company to Use Proceeds to Advance Programs for Duchenne Muscular Dystrophy.


Solid Biosciences is proud to join the global Rare Disease community to recognize Rare Disease Day 2017.


As we move into 2017, I want to reflect on our journey since founding Solid three years ago and to outline what we hope to accomplish in the coming year.


The Duchenne Research Fund (DRF) provided Solid with a research grant of $600,000 to support its LTBP4 program to combat fibrosis and to support novel biomarker work in Duchenne muscular dystrophy (DMD).


Grant from Duchenne UK to fund research to better understand the potential benefits of statins in patients with Duchenne muscular dystrophy (DMD).


Biologic candidate has the potential to complement gene therapy by improving muscle function.


It has been an exciting and emotional time for our Duchenne muscular dystrophy (DMD) community.


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