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Specialty Pharma Journal Specialty, Precision & Technology

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: Hurler syndrome

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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April, 2015

8 April
FDA Accepts ArmaGen’s IND for its Hurler Syndrome Candidate

Privately-held biotech ArmaGen, Inc. announced today that US health regulators have accepted for review its investigational New Drug (IND) application for its drug for a rare lysosomal storage disease. The company, focused on developing novel therapies to treat severe neurological disorders, said that the US Food and Drug Administration (FDA) …
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