uniQure Gets Back Gene Therapy After Chiesi Ends Partnership

uniQure will regain the rights for their hemophilia B gene therapy as the Chiesi Group backs out from working partnership with the genetics company.


uniQure (NASDAQ:QURE) will regain the rights for their hemophilia B gene therapy as the Chiesi Group backs out from working partnership with the genetics company. 

“We are grateful for the substantial investments that Chiesi has made in AMT-060, and we have been fortunate to have them as a collaboration partner over the years,” Matthew Kapusta CEO of uniQure said in a press release published on Monday (July 31).

Chiesi’s CEO Ugo Di Francesco said despite the potential results of the gene therapy, recent changes in their “strategic priorities” forced the company to drop the partnership. “[We] sincerely wish [uniQure] the best as they advance this potentially exciting gene therapy to patients.”

Shift in strategy leaves the promising gene therapy back with uniQure

Prior to that, the company announced on July 21 that it had completed a reproducible and scaled-up manufacturing process for their hemophilia B gene therapy program.

“This progress includes the validation of analytical methods required to manufacture and test product in accordance with Good Manufacturing Practices (GMP),” the company revealed in the July press release.

According to the National Hemophilia Foundation, uniQure’s development seeks to help treat a genetic disorder that is present in close to 20,000 people in the US. This ailment takes place in approximately in one out of 5,000 births.

Reporting from BioPharma DIVE indicates uniQure will now have to face all of the program’s future development costs, part of which includes $3 million 2017 that before was planned to be a shared expense.

In April of this year, uniQure dropped the production of Glybera due to very small adoption rate, since that gene therapy was focused on rare diseases.

At the time, Kapusta said “Glybera’s usage had been “extremely limited” and the company did not see patient demand increasing in the future.

Investor Takeaway

“This latest move by Chiesi further exemplifies the challenges gene therapy producers face. The announcement ends a deal which has been in place since 2013,” BioPharma DIVE wrote.

Despite the potential for gene therapies the arduous work can often lead to a lack of results and in this case force companies to refocus their business.

Since the day of the announcement uniQure’s stock has dropped 0.25 percent. During after hours trading on August 3, QURE was valued at $8.07.

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Securities Disclosure: I, Bryan Mc Govern, hold no direct investment interest in any company mentioned in this article.

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