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Generation Bio Sinks on Preclinical Study of Hemophilia Gene Therapy

December 15, 2021

Generation Bio’s preclinical study of its nonviral gene therapy for hemophilia A showed impressive results in mice, but disappointing effects in non-human primates.

Photo: Matthew Stanton, chief scientific officer of Generation Bio

The update on its program sent shares in Generation Bio downward as the stock was trading at $5.76 this morning following a close at $13.90 on December 13 before the news. The shares are off a 52-week high of $41.86.

Generation Bio, in an update of its hemophilia A program December 14, reported that it was able to generate peak mean human factor VIII expression of 205 percent of normal at 2.0 mg/kg in mouse studies.

Generation Bio Sinks on Preclinical Study of Hemophilia Gene Therapy

Generation Bio’s preclinical study of its nonviral gene therapy for hemophilia A showed impressive results in mice, but disappointing effects in non-human primates.

The update on its program sent shares in Generation Bio downward as the stock was trading at $5.76 this morning following a close at $13.90 on December 13 before the news. The shares are off a 52-week high of $41.86.

Generation Bio, in an update of its hemophilia A program December 14, reported that it was able to generate peak mean human factor VIII expression of 205 percent of normal at 2.0 mg/kg in mouse studies.

Findings from companion studies in non-human primates demonstrated human factor VIII expression of up to 2 percent of normal at 2.0 mg/kg, with higher-than-expected variability in both factor VIII expression and tolerability within and across studies.

The company said additional optimization is needed to translate the improvement in potency and reduction in variability observed in mice to non-human primates (NHPs) and to support nomination of a development candidate for the company’s hemophilia A program.

“Over the course of the year, we have made significant progress in the development of our platform,” said Matthew Stanton, chief scientific officer of Generation Bio. “We are working to translate the improved potency and decreased variability that we have observed in mice to NHPs. By applying novel analytical methods to these preclinical studies in both species, we have gained important insights that we believe will allow us to advance additional novel proprietary [cell-targeted lipid nanoparticles] to meet our target profile for a hemophilia A development candidate.”

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