Chiesi hands back gene therapy to uniQure | BioPharma Dive – BioPharma Dive

Dive Brief:

Even as gene therapies are being touted as the next wave of innovation that could offer cures for certain genetic conditions, it remains to be seen whether these products are actually commercially viable. There has yet to be a gene therapy approved in the U.S. (although Spark Therapeutics' application is pending), but two of the transformative drugs have been on the market in Europe.

Yet neither of those commercially available gene therapies have found much success. GlaxoSmithKline plc. said just last week it is looking to move away from its rare disease portfolio, including the gene therapy Strimvelis. Meanwhile, uniQureannounced back in April it would not renew the marketing authorization application in Europe for its already-approved gene therapy Glybera.

This latest move by Chiesifurther exemplifies the challenges gene therapy producers face. The announcement ends a deal which has been in place since 2013. Chiesisaid in a statement that the decision was "driven by recent changes in our strategic priorities."

uniQuretried to put brave face on the news, but partnership exits are rarely good news for a biotech.

"By regaining unencumbered, global rights to a late-stage program that has demonstrated significant clinical benefit for patients with hemophilia B, we believe uniQure is better positioned to accelerate the global clinical development plan, maximize shareholder return on our pipeline and take advantage of new potential opportunities related to the program," said CEO Matthew Kapusta.

The company recently announced positive developments in a Phase 1/2 trial of AMT-060, which supported further expansion of the eligibility of the adeno-associated virus 5 (AAV5) gene therapy to nearly all patients with hemophilia B. Meanwhile, investors are paying close attention to Spark's gene therapy for hemophilia B, which is also in early-to mid-stage development.

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Chiesi hands back gene therapy to uniQure | BioPharma Dive - BioPharma Dive