The alarm bell is ringing for companies developing adenoviral vectors for gene therapies, particularly those meant to treat liver disease. But every firm in the field is now on alert.
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With big pharma on board, disappointments like the threatened EU market removal of pioneering Glybera and the Celladon trial failure give the field of gene therapy a bumpy ride after some major highs.
Nucleonics, like other companies competing in the new field of RNAi, started out with big ambitions. Even before its Series A financing ran out, the company decided to focus on clinical goals. Nucleonics has just received a $41 million financing meant to see two drug candidates, presently still in preclinical testing, get through Phase II. Investors like its focus. Meanwhile, Alnylam still has big plans, which it's now hoping to finance through the public markets.
Targa Therapies marries gene therapy and monoclonal antibodies in its proprietary Antibody Delivery System, which it believes will help circumvent many of the flaws in current gene therapies.