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Breaking News

FDA Approves Gavreto for the Treatment of Adults with Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer
Incyte Announces Pivotal GEOMETRY mono-1 Study Results of Capmatinib in Patients with METex14 Metastatic Non-Small Cell Lung Cancer Published in NEJM
Natera Announces SMART Study Data for Aneuploidy and 22q Unblinded
Aimmune Agrees to be Acquired by Nestlé Health Science for $2 Billion
Twist Bioscience Announces Potent SARS-CoV-2 Neutralizing Data from COVID-19 Therapeutic Antibody Program
Final Analysis of the Observational GioTag Study: Sequential Afatinib and Osimertinib in Patients with EGFR Mutation-Positive NSCLC
Santhera Exercises Option to Obtain Worldwide Rights to Vamorolone in Duchenne Muscular Dystrophy and All Other Indications
FDA Approves Onureg, a New Oral Therapy, as Continued Treatment for Adults in First Remission with Acute Myeloid Leukemia
FDA Accepts Vertex’s sNDA for Trikafta, Symdeko and Kalydeco for Additional CFTR Mutations
Gilead Sciences and Jounce Therapeutics Announce Exclusive License Agreement for Novel Immunotherapy Program

Tag Archives: Mucopolysaccharidosis

January, 2018

25 January
FDA Grants Fast Track Status to Sobi’s Investigational MPS IIIA Drug

Swedish Orphan Biovitrum AB (publ) (Sobi™) announces that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug (IND) application for the drug candidate SOBI003. In addition, SOBI003 was granted Fast Track status by the FDA. SOBI003 is a …
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February, 2016

8 February
FDA Clears Sangamo Biosciences’ IND Application of SB-318 for Treatment of MPS I

RICHMOND, Calif., Feb. 8, 2016 /PRNewswire/ — Sangamo BioSciences, Inc. (NASDAQ: SGMO), the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type …
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