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It was a good day for Novartis: A second drug with huge selling potential has been approved for the US market. The FDA gave the go ahead for breast cancer drug Piqray (alpelisib), the first and only treatment developed specifically for patients with a PIK3CA mutation in HR+/HER2-advanced breast cancer.

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The FDA has approved Novartis-subsidiary AveXis Inc’s spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec-xioi). The company wants to set the price tag at $2,125m per dose.

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At BioEquity 2019, Spanish Bioindustry Association ASEBIO has given a sneak peek into its 2018 Annual Report, that each year describes the news and trends from the Spanish Biotech Sector.

Structure model of idebenone. 123rf.com/ molekuul

Chiesi Farmaceutici has licenced commercialiation rights for LHON treatment Raxone (idebenone) from Santhera Pharmaceuticals for CHF50m (€44m) in cash plus sales milestones.

Using targeted mutation methods, a break in the DNA strand is induced at a specific site and subsequently is repaired by the cell's own DNA repair system. When DNA mismatches occur, the appropriate gene in no longer functional. © 123rf.com/Andrii Panchyk

EU agriculture ministers want to exclude crops created by targeted mutation methods from EU GMO legislation.

Seattle headquarters of Just Biotherapeutics. ©Just Biotherapeutics, Inc
Evotec SE is set to acquire biologics manufacturing specialist Just Biotherapeutics, Inc in a deal that could amount up to $90m.

The codon-specific elongation model (COSEM) simulates protein synthesis. © Scientific Reports

Researchers at German regulatory authority Paul-Ehrlich Institute (PEI) have patented a method to accurately forecast biotech-based protein synthesis in host cells.

© DNA Script

In an oversubscribed Series B round led by LSP, Paris-based DNA Script raised US$38.5. Bpifrance and existing investors Illumina Ventures, M. Ventures, Sofinnova Partners, Kurma Partners and Idinvest Partners contributed.

SBD 2019 Plenary Session. Picture: Deirdre O'Keeffe

Although the Swiss biotech sector continues to grow and the annual Swiss Biotech Day is as sought-after as never before, panelists and attendees remain cautious with their predictions. A reoccurring theme at the event in Basel was the plea for change. 

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Researchers from Universities Nijmegen and Naples have presented a retinal AAV-based gene therapy delivery system that can express huge genetic elements by assembling protein fragments from several vectors.