GermanyGermany

mRNA replacement therapy works

06.02.2011

Munich – For the first time, researchers have provided evidence that mRNAs can be used as drugs. By repairing genetic defects in knock-out mice, a team under Carsten Rudoph from Ludwig-Maximilians University in Munich demonstrated that transcript replacement therapy has an effect (Nat Biotechn., doi: 10.1038/nbt.1733).
Before treating mice with a hereditary lung defect, Rudolph replaced roughly a quarter of all uridine and cytidine moieties with 2-thiouridine and 5-methyl-cytidine as mRNA building blocks. “Modification of the mRNA drastically reduced TLR-mediated immunogenicity and at the same time significantly improved mRNA stability,” said Rudoph. Inhalation of an mRNA aerosol, which encoded the surfactant protein B (SF-B), significantly prolonged overall survival of the SF-B-deficient mice. They usually die after 3 days due to a lung defect. SF-B mRNA delivery twice a day over 4 weeks prolonged survival time to 26±2 days. Rudolph and his colleague Christian Plank, who co-founded Seefeld-based Ethris GmbH, now want to commercialise their SNIM® technology. “Our goal is to apply our SNIM® mRNAs locally in regenerative therapies, i.e. as a treatment for diabetes ulcers,” explained Ethris-CEO Rudolph. Other potential applications are local in vivo production of therapeutic proteins or mRNA therapies, which do not show the limitations of DNA-based gene therapies such as activation of oncogenes (cancer) or poor transfection rates. According to Rudolph, the technology is almost ready for market. He said he “hopes to start clinical trials in regmed settings in 2 or 3 years.”

Germany, FranceGermany

21.12.2011

Martinsried/Paris - Sanofi has done some Christmas shopping in Munich and has licensed Scil Technology’s programme for the regenerative treatment of osteoarthritis and cartilage disorders. Under the agreement, Scil could reap up...

GermanyGermany

20.12.2011

Monheim - It's nearly Christmas, and Bayer Crop Science gives out some presents. As the company announced, a $750 million settlement with U.S. rice farmers is now binding and can move forward. In summer the German firm had agreed...

GermanyGermany

16.12.2011

Bonn – Germany’s federal government is prolonging one of its most successful measures to date for stimulating the establishment of high-tech companies – the High-Tech-Gründerfonds (HTGF). The HTGF was launched in 2005 with €272m...

GermanyGermany

15.12.2011

Leverkusen – One short statement from the European Medicines Agency (EMA) means one giant step for German biotech company Biofrontera. The agency issued a favourable opinion on the firm’s marketing approval application for its...

GermanyGermany

15.12.2011

While the European sovereign debt crisis keeps capital markets in a choke-hold, with potential negative effects on the financing situation in many industries – including biotech – there was encouraging news for drug-developing...

GermanyGermany

08.12.2011

Heidelberg – There is still life in the European life science finance. EMBL Ventures has closed a €40m fund. The early stage investor and the venture capital arm of the European Molecular Biology Laboratory(EMBL) announced the...

GermanyGermany

03.11.2011

Düsseldorf – BIO-Europe 2011 in Düsseldorf has again broken all previous records in terms of participants and company numbers. More than 3,000 representatives from 1,800 companies registered for Europe‘s largest partnering event....

GermanyGermany

02.11.2011

Brussels/San Francisco – As the biggest biotech partnering event on the continent, BioEurope, draws to a close, the venture capital community sent ambivalent messages for life sciences. Dow Jones VentureSource, an analyst,...

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