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PTC Therapeutics Announces $20 Million Strategic Investment From Celgene Corporation

   PTC Therapeutics logo. (PRNewsFoto/PTC Therapeutics, Inc.)

SOUTH PLAINFIELD, NJ UNITED STATES
- PTC Grants Celgene Option for Collaboration on Two Targets Utilizing GEMS
                                 Platform -

    SOUTH PLAINFIELD, N.J., Sept. 13 /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 that that Celgene
Corporation (Nasdaq: CELG) will make a $20 million equity investment in
PTC. At the same time, PTC will grant Celgene an option for an exclusive
research and option agreement on two oncology targets for the development
of orally bioavailable small molecules through the application of PTC's
GEMS (Gene Expression Modulation by Small-molecules) technology.
    (Logo: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO )
    Under the terms of the research and option agreement, if the Celgene
option is exercised, PTC may receive funding for research efforts and earn
substantial milestone payments per target for achieving certain discovery,
development, regulatory, and commercial objectives. Celgene will receive
exclusive worldwide rights and pay PTC royalties on worldwide net sales of
any products resulting from this collaboration.
    "Celgene is widely recognized for its scientific innovation and
commercial success in developing novel oral solutions for areas of high
unmet medical need. We are delighted to welcome them as an investor," said
Stuart Peltz, Ph.D., President and CEO of PTC Therapeutics. "We have
developed an innovative approach to drug discovery in our GEMS technology
and have demonstrated its potential through our multiple internal programs
and collaborations. This significant support from Celgene will strengthen
our continuing efforts to develop and commercialize products based on our
GEMS technology."
    "We are pleased to make this investment in PTC Therapeutics, a
demonstrated leader in the discovery and development of small molecules
targeting RNA biology," said Sol Barer, Ph.D., Chairman and Chief Executive
Officer of Celgene. "This investment, combined with the research option,
enables Celgene to participate with PTC in the development of innovative
therapies."
    About GEMS
    GEMS is PTC's novel and proprietary technology platform for the
identification of small-molecules that modulate post-transcriptional
control mechanisms. Compounds identified through the GEMS technology target
processes that act through the untranslated regions (UTRs) of messenger RNA
(mRNA) molecules. PTC has successfully employed the GEMS technology in drug
discovery programs in oncology, infectious diseases, cardiovascular
diseases, and neuromuscular disorders.
    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, CV Therapeutics and
Schering-Plough. For more information, visit the company's website,
http://www.ptcbio.com.


SOURCE PTC Therapeutics, Inc.




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Related links:
  • http://www.ptcbio.com
    Photo Notes:
    NewsCom: http://www.newscom.com/cgi-bin/prnh/20010919/PTCLOGO
    AP Archive: http://photoarchive.ap.org
    PRN Photo Desk, photodesk@prnewswire.com
    CONTACT:
    Jane Baj of PTC Therapeutics, Inc.,
    +1-908-222-7000, x167, jbaj@ptcbio.com; or Sheryl Seapy of Pure
    Communications, +1-949-608-0841, sheryl@purecommunicationsinc.com