PTC Therapeutics

American pharmaceutical company

PTC Therapeutics logo

PTC Therapeutics, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. The company is headquartered in South Plainfield, New Jersey, and it operates globally.

History

PTC Therapeutics was founded in 1998 by Stuart W. Peltz, Ph.D., and Allan Jacobson, Ph.D. The company was established with the aim of developing small molecule drugs that target post-transcriptional control processes. Over the years, PTC Therapeutics has grown through strategic partnerships, acquisitions, and the development of its proprietary technologies.

Research and Development

PTC Therapeutics is known for its innovative approach to drug discovery and development. The company utilizes its proprietary technologies, including the Gene Expression Modulation by Small-molecules (GEMS) platform, to identify and develop new therapies. This platform allows the company to modulate gene expression and address the underlying causes of various genetic disorders.

Key Products

PTC Therapeutics has developed several key products, including:

• Translarna (ataluren): A treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna is designed to enable the ribosome to read through premature stop codons, allowing for the production of functional proteins.

• Evrysdi (risdiplam): A treatment for spinal muscular atrophy (SMA). Evrysdi is an oral medication that increases the production of the survival motor neuron (SMN) protein, which is deficient in SMA patients.

Strategic Collaborations

PTC Therapeutics has established several strategic collaborations with leading pharmaceutical companies and research institutions. These collaborations have been instrumental in advancing the company's research and development efforts. Notable partnerships include collaborations with Roche and SMA Foundation for the development of Evrysdi.

Acquisitions

In its pursuit of expanding its therapeutic pipeline, PTC Therapeutics has acquired several companies and technologies. These acquisitions have allowed the company to enhance its research capabilities and broaden its portfolio of treatments for rare diseases.

Corporate Social Responsibility

PTC Therapeutics is committed to corporate social responsibility and actively engages in initiatives that support patients, communities, and the environment. The company focuses on improving patient access to treatments and supporting rare disease communities through various programs and partnerships.

Related pages

• Biopharmaceutical company
• Duchenne muscular dystrophy
• Spinal muscular atrophy
• Gene therapy