| PTC Therapeutics, Inc. | |
| Type: | Public |
| Traded As: | NASDAQ: Russell 2000 Component |
| Industry: | Pharmaceuticals |
| Location City: | South Plainfield, New Jersey |
| Location Country: | U.S. |
PTC Therapeutics, Inc. is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases.[1] [2]
In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases.[3] PTC acquired the Bio-e platform in 2019.[4]
In 2017, PTC acquired Emflaza (deflazacort) from Marathon Pharmaceuticals.[5] PTC also owns Translarna, (Ataluren) marketed for nonsense mutation Duchenne muscular dystrophy.[6] Together, the two products generated revenues of 174 million dollars and 260 million dollars in 2017 and 2018 respectively.[7]
PTC has the commercialization rights for WAYLIVRA (volanesorsen) in Latin America.[8]
In 2018, PTC acquired Agilis Biotherapeutics and a gene therapy candidate, GT-AADC, with its compelling clinical data in treating aromatic L-amino acid decarboxylase (AADC) deficiency.[9] AADC deficiency is a rare CNS disorder arising from reductions in the enzyme AADC that result from mutations in the dopa decarboxylase (DDC) gene.
In 2020, PTC acquired Censa Pharmaceuticals, Inc., a biopharmaceutical company focused on the development of CNSA-001 (sepiapterin), a clinical-stage investigational therapy for orphan metabolic diseases, including phenylketonuria (PKU) and other diseases associated with defects in the tetrahydrobiopterin (BH4) biochemical pathways diagnosed at birth.[10]
In 2020, PTC announced the FDA approval of Evrysdi (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older.[11]
https://www.ptcbio.com/our-science/expertise/bio-e-electron-transfer/