Generation Bio backed with $100m financing round

pharmafile | February 28, 2018 | News story | Research and Development, Sales and Marketing Generation Bio, biotech, drugs, pharma, pharmaceutical 

At the start of year, Generation Bio was revealed to the public, announcing that it had been developing gene therapies after a $25 million financing round led by Atlas.

After a little less than two months of this announcement, Generation Bio has been backed with a further $100 million from investors clearly keen on the idea of a gene therapy alternative to viral vectors.

The 28-employee-strong biotech has developed “GeneWave” technology, which has been used to create gene therapies. In particular, this means using closed-ended DNA to deliver the gene therapy to cells rather than through the use of viral vectors.

Generation Bio has suggested that this will avoid the immunogenicity that has recently become associated with gene therapy vectors.

This latter method of treatment has been brought into question after leading researcher, James Wilson, urged caution when using high-doses of the gene therapy.

This came after a trial on animals, including pigs and rhesus macaque monkeys, found that there were strong adverse events after testing with an AAV9 gene therapy.

The monkeys were found to develop liver toxicity and the pigs suffered motor neuron damage. After the trial, Wilson had resigned from his advisor role on the board of Solid Biosciences prior to publication of this trial. Solid is a company looking into using high-dose therapies for the treatment of spinal muscular atrophy.

This explains why Generation Bio’s Round B financing has attracted such attention, especially when it claims that its method of delivery will also allow it to be re-dosed, as opposed to the viral vectors that are a one-shot only treatment.

“We are delighted to have the support of this group of expert, long-term investors who share our vision to create a generation of people living unaffected by genetic disease,” said Geoff McDonough, President and Chief Executive Officer of Generation Bio. “This will be a critically important year for us as we develop the GeneWave platform and work towards our first development candidates to address rare diseases of the liver. The round will also support the development of approaches to deliver ceDNA to other tissues such as the eye, the central nervous system and the lungs.”

Ben Hargreaves

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