Specialty Pharma Journal Specialty, Precision & Technology
CAMBRIDGE, Mass., Nov. 30, 2018 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (NASDAQ: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for EDIT-101, an experimental CRISPR genome editing medicine being investigated for the treatment of …
Tag Archives: gene therapy
November, 2018
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28 November
Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy and Fast Track Designations for RP-L102 Gene Therapy for Fanconi Anemia
NEW YORK–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations to RP-L102, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia …
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26 November
Rocket Pharmaceuticals and REGENXBIO Announce New License Agreement for the Treatment of Danon Disease Using NAV AAV9 Vector
NEW YORK & ROCKVILLE, Md.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc. (Nasdaq:RCKT), a leading U.S.-based multi-platform gene therapy company, and REGENXBIO Inc. (Nasdaq:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced an exclusive, worldwide license agreement for …
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1 November
Ophthotech Expands Gene Therapy Pipeline with Exclusive Option for Novel Product Candidates to Treat Best Vitelliform Macular Dystrophy
NEW YORK–(BUSINESS WIRE)–Ophthotech Corporation (NASDAQ:OPHT) announced today that it has entered into an exclusive option agreement with the University of Pennsylvania (Penn) and the University of Florida Research Foundation (UFRF) for rights to negotiate to acquire an exclusive global license to develop and commercialize novel adeno-associated virus (AAV) gene therapy product …
September, 2018
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21 September
Amicus Acquires Gene Therapy Portfolio of Ten AAV Programs in Neurologic Lysosomal Storage Disorders
CRANBURY, N.J., Sept. 20, 2018 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD) today announced the signing of a definitive agreement in which Amicus Therapeuticswill receive worldwide development and commercial rights for ten gene therapy programs developed at The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital and The Ohio State University. The ten programs are licensed …
August, 2018
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29 August
Genprex Amends Options to License Additional Cancer Fighting Technologies from MD Anderson Cancer Center
AUSTIN, Texas & CAMBRIDGE, Mass.–(BUSINESS WIRE)–Genprex, Inc. (NASDAQ: GNPX), a clinical stage gene therapy company developing a new approach to treating cancer based upon a novel proprietary technology platform, announced today that it has entered into amendments with The University of Texas MD Anderson Cancer Center (MD Anderson) to extend 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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6 August
Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium, Imperial Innovations and Oxford Biomedica Partner to Develop Gene Therapy for Cystic Fibrosis
INGELHEIM, Germany–(BUSINESS WIRE)–Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium (GTC)—consisting of Imperial College London and the Universities of Oxford and Edinburgh—Imperial Innovations, and Oxford BioMedica (OXB) announce a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF). The new partnership brings together the …
July, 2018
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20 July
PTC Therapeutics to Acquire Agilis Biotherapeutics Expanding its Gene Therapy Pipeline
SOUTH PLAINFIELD, N.J., July 19, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that it has entered into an agreement to acquire Agilis Biotherapeutics, Inc., a biotechnology company advancing an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system (CNS). The transaction was approved by the Boards of …
April, 2018
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23 April
FDA Grants Fast Track Status to MeiraGTx’s AAV-RPGR for the Treatment of X-Linked Retinitis Pigmentosa Due to RPGR Deficiency
LONDON & NEW YORK–(BUSINESS WIRE)–MeiraGTx Limited, a vertically integrated, clinical stage gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for AAV-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP) due to defects in the retinitis pigmentosa GTPase regulator (RPGR) gene. “XLRP …