Tag Archives: genetic disorders

September, 2018

13 September
FDA Grants Fast Track Designation to ArQule’s Miransertib for the Treatment of PIK3CA-Related Overgrowth Spectrum

BURLINGTON, Mass.–(BUSINESS WIRE)–ArQule, Inc. (Nasdaq: ARQL) today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to miransertib (ARQ 092) for the treatment of PIK3CA-Related Overgrowth Spectrum (PROS), a group of related, ultra-rare genetic disorders characterized by excessive tissue growth in various parts of the …
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24 August
PTC Therapeutics Successfully Completes Acquisition of Agilis Biotherapeutics

SOUTH PLAINFIELD, N.J., Aug. 23, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ : PTCT ), today announced that it has successfully completed the acquisition of Agilis Biotherapeutics, Inc., a private biotechnology company focused on the advancement of innovative gene therapy programs for rare genetic disorders that affect the central nervous …
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13 October
New Research Increases Understanding of Duchenne Muscular Dystrophy

BINGHAMTON, NY – A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)–one of the most common lethal genetic disorders–and points to potential therapeutic approaches. “The findings suggest that the immune system has an important role in the …
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21 December
Bayer Partners with CRISPR Therapeutics to Develop New Gene Therapies

LEVERKUSEN, Germany and BASEL, Switzerland, Dec. 21, 2015 /PRNewswire/ — Bayer and CRISPR Therapeutics have entered into an agreement to create a joint venture (JV) to discover, develop and commercialize new breakthrough therapeutics to cure blood disorders, blindness, and congenital heart disease. CRISPR Therapeutics will contribute its proprietary CRISPR-Cas9 gene-editing technology …
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