Clinical Trial Updates & News

May 31, 2017 – (Novartis) – The results, which will be presented at the upcoming 53rd Annual Meeting of the American Society of Clinical Oncology (ASCO; abstract #7509; Monday, June 5, 1:15 PM CDT), that eight of nine evaluable patients had no signs of CLL in their bone marrow at three months.
May 18, 2017 – (Spark) – Voretigene neparvovec has the potential to be the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the U.S. 
April 10, 2017 – (Mesoblast) – Mesoblast Limited (ASX: MSB; Nasdaq: MESO) announced that the Phase 3 trial of its allogeneic mesenchymal precursor cell product candidate MPC-150-IM in patients with moderate to advanced chronic heart failure was successful in the pre-specified interim futility analysis of the efficacy endpoint in the trial's first 270 patients. It is expected that the trial will enroll in total approximately 600 patients. 
January 19, 2017 – (Abeona) – “This designation builds on our commercial portfolio of AAV gene therapies that have received FDA and EMA orphan drug designations, which is an important validation of the scientific and clinical translation of these products for severely underserved patient populations,” stated Timothy J. Miller, Ph.D., President & CEO of Abeona Therapeutics Inc. 
January 17, 2017 – (Pluristem) – The trial was recently cleared by the United States’ Food and Drug Administration and the United Kingdom’s Medicines & Healthcare products Regulatory Agency. 
January 12, 2017 – (MaxCyte) – Using MaxCyte’s patented Flow Electroporation Technology, researchers demonstrated that transfecting three molecules, a single-strand oligonucleotide correction template, along with messenger RNA encoding for CRISPR-Cas9 gene editing complex and selected guide RNA into HSC obtained from individuals with X-CGD, resulted in correction of mutation in the CYBB gene. 
January 11, 2017 – (Sangamo) – Using Sangamo's zinc finger nuclease genome editing technology, SB-318 is designed as a single treatment strategy intended to provide stable, continuous production of the IDUA enzyme for the lifetime of the patient.